Decisions based on the pharmaceutical market
Baltic States pharma market review
SoftDent is providing quarter and annual Lithuanian, Latvian, and Estonian pharma markets' secondary sales analysis based on the data collected by PharmaZOOM project.
Summarized Lithuania, Latvia and Estonia pharma market overview reports for last quarter is already generated with the forecast for next year.
PharmaZOOM News
2024 04 23
New LT data
Data for the period April 15-21, 2024 is uploaded to PharmaZOOM LT.
2024 04 10
New LV data
Data for March 2024 is uploaded to PharmaZOOM LV.
2020 07 17
Integration with Power BI tool
With the help of Power BI, SoftDent prepares individualized interactive reports according to specific customer needs.
This solution includes the integration of pharmaceutical market data with the PowerBI business analytics tool, which allows you to effectively monitor pharmaceutical market changes and analyze data using interactive visuals.
2020 01 07
Additional data sources
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Every month together with regularly updated wholesalers sales, we are importing VVKT sales of packages which sales we did not have before.
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Information on Procurement Contract Data has been integrated into the PharmaZOOM LT system. Every month together with updated wholesalers sales it is possible to analyze data of the concluded contracts.
Pharmaceutical Market News
2024 04 09
Cheap drugs, inspections, and a future war – what’s driving drug shortages?
Drug shortages are worsening globally, leaving patients without access to essential medications, and healthcare providers struggling to find alternative treatments. Many governments are now intervening to prevent escalating shortages by stockpiling certain essential drugs, investing in domestic pharma manufacturing, or allowing price increases for generics to encourage their production.
Drug shortages are increasing for yet another year, according to the Pharmaceutical Group of the EU, which represents pharmacists. The shortages are impacting both high and low-value drugs (such as antibiotics) alike, the group stated in an annual report.
EU-level policy to combat the problem is unlikely to make much difference in 2024, GlobalData analysts say.
As demand for obesity-related treatments surges, the supply of glucagon-like peptide-1 receptor (GLP-1R) agonists is not expected to return to normal until at least the end of 2024. The US Food and Drug Administration (FDA) and European Medicines Agency have reported shortages of these drugs, and Australia’s Therapeutic Goods Administration and Health Canada are advising doctors not to start new patients on them due to the global shortage.
US generic shortages and political interventions
In the US, generic drug shortages are affecting patient access to treatment, particularly for cancer patients, and healthcare providers are having to choose who to treat.
During a hearing on chronic drug shortages in February 2024, the US House Ways and Means Committee highlighted poor manufacturing quality as the leading driver of chronic shortages.
Jeromie Ballreich, a research professor at John Hopkins Bloomberg School of Public Health (Baltimore, US), proposed new legislation to require transparency in drug manufacturing; the withheld information makes it harder to identify supply chain vulnerabilities, he said. Ballreich also proposed paying manufacturers to stockpile drugs, and boosting domestic active pharmaceutical ingredient (API) manufacturing.
The House Committee suggested that government intervention is required for generic prices to correct the market to “its natural competitive state.”
The current economic model leads to outsourcing manufacturing to cheaper countries to reduce costs and does not encourage domestic production, claimed the House Committee.
Further, during the meeting, politicians suggested policies to incentivise generic manufacturers to enter and stay in the market. For example, some suggested higher payments for manufacturers who have resilient and transparent supply chains, or higher payments for complex generic drugs production.
The American Society of Clinical Oncology (ASCO) (Alexandria, US) and some pharma manufacturers have suggested that the low prices of generics are a reason for the drug shortages, by forcing generic manufacturers to discontinue production and exit the market.
The ASCO also suggested that Congress implement incentives to boost US manufacturing and encourage advanced technologies such as continuous manufacturing.
In the US, increased demand, shipping delays, and shortages of API account for 88% of current drug shortages where a cause is known. Demand is especially high for anaesthetics and oncology drugs.
Japan, France, and Germany prepare for shortages
Japan’s Ministry of Health, Labour and Welfare will launch a drug shortage reporting scheme in April this year, according to the newspaper Yomiuri. Manufacturers should report potential drug shortages within six months for antibiotics and immunosuppressants.
At the same time, France’s National Assembly (the lower house of its parliament) has voted to increase the financial penalties for shortages that can be imposed on pharmaceutical companies and suppliers.
The potential for wider regional conflicts may also exacerbate a drug shortage crisis in the future.
Germany’s Health Minister Karl Lauterbach recently announced plans with the country’s Ministry of Defense for a law to prepare Germany’s healthcare sector for the possibility of an armed conflict involving Nato allies. In a press release, Lauterbach stated that it is vital to prepare the healthcare system for scenarios long considered unthinkable, emphasising that this was an important lesson of the Covid-19 pandemic. Lauterbach stated that there needs to be preparedness at every level of the healthcare system in the case of a large number of injured people needing to be treated in German hospitals.
2024 03 01
Spotlight on Promising New Medicines and Leading Companies in 2024
Every year, we tap Evaluate's annual preview report to assess the biggest potential new drugs of the year ahead. Typically, major pharmaceutical and biotechnology companies are featured, each presenting potential blockbuster launches. In 2024, however, things are set to play out a little differently. The most anticipated drug comes from a biotech that many of us may have been unaware of, Karuna Therapeutics. Its drug is not for traditionally well-known diseases like obesity or cancer; instead, it focuses on schizophrenia, a condition with few effective treatments and limited media attention. That all changed when Bristol Myers Squibb snapped up the biotech in late 2023 for a major $14 billion, with the jewel in the crown of that deal being KarXT, a new type of drug to treat schizophrenia. Evaluate, which estimates sales for each drug by 2028, projects $2.8 billion in sales for this particular drug, making it the highest estimate among its top 10 most anticipated drug approvals for the year. The top 10 list is based on Evaluate’s 2024 preview, where the analysts assess what they see as the biggest-selling drugs that will be approved this year in terms of sales by 2028.
1. KarXT (Karuna Therapeutics), est. 2028 sales: $2.8 billion
Karuna Therapeutics' name began to be mentioned in the media at the end of 2023 when the pharmaceutical company Bristol Myers Squibb concluded a deal to acquire it for USD 14 billion. The company's drug, KarXT, is expected to be launched this year and to become the most profitable new product by 2028. "KarXT is a first-in-class M1/M4 muscarinic receptor agonist, which is being sought for approval for the treatment of schizophrenia. "Karuna Therapeutics believes that it has two clear advantages in this market - the efficacy and safety of the drug. The first results of the trials showed a reduction of almost 10 points on the schizophrenia positive and negative syndrome scale, which is an indicator of the severity of the disease, in treated patients. Another important advantage is that the drug did not have the severe side effects that are seen with current treatments for schizophrenia, such as lethargy and weight gain.
2. Donanemab (Eli Lilly), est. 2028 sales: $2.2 billion
The experimental drug donanemab is the most anticipated treatment for Alzheimer's disease. In 2022, it was projected to reach sales of USD 6 billion by 2026. In a new review, Evaluate forecasts sales of $2.2 billion by 2028. Early optimism was considerably dampened when the drug was rejected by the US Food and Drug Administration in January 2023 due to the low number of patients studied. However, Eli Lilly has continued its work, and a new assessment should be published in the first quarter of 2024. The failure of Biogen's Aduhelm, when it was launched but later withdrawn from the market due to high cost, safety, and efficacy issues, also contributed to the lowered expectations. Studies published in May 2023 showed that Eli Lilly's donanemab slowed cognitive decline by 35% and reduced the risk of progression to the next stage of the disease by 39% compared to placebo.
3. Resmetirom (Madrigal Pharmaceuticals), est. 2028 sales: $2.1 billion
Madrigal Pharmaceuticals hopes to succeed in areas where many major pharmaceutical and biotechnology companies have failed. The company aims to develop a safe and effective treatment for non-alcoholic steatohepatitis (NASH). This disease, which can go unnoticed for years with very few symptoms, is caused by fatty deposits in the liver. A proper diet and physical activity can help reduce the amount of fat accumulated in the liver, but the pharmaceutical industry is seeking pharmaceutical solutions. Over the last five years, dozens of biopharmaceutical companies have conducted numerous trials using different mechanisms of action to reduce fat in the liver. Although almost all trials have been unsuccessful, analysts see the NASH market as potential for significant gains. Clinical trials of Madrigal Pharmaceuticals' drug resmethimethrom at the end of 2022 showed positive results in the treatment of NASH. Twenty-six percent of patients on the 80 mg dose and thirty percent of patients on the 100 mg dose achieved the primary endpoint of a two or more-point reduction in the NASH activity score, as well as no worsening of fibrosis. Evaluate forecasts that sales of the drug will reach USD 2.1 billion by 2028.
4. Sotatercept (Merck & Co), est. 2028 sales: $2 billion
Merck's experimental drug sotatercept is for the treatment of pulmonary arterial hypertension (PAH). Sotatercept is of great interest because it works in a different way compared to drugs currently on the market that act as vasodilators. As a type IIA-Fc activin receptor fusion protein, it has a direct effect on the key problematic proteins that cause the disease. If approved, the drug will be the first in its class to treat PAH. Merck hopes this will give it an advantage over other drugs such as Johnson & Johnson's Uptravi and Opsumit. Sotatercept will also be important for Merck as it aims to develop a counterweight to Keytruda. The latter generated more than USD 18 billion in 2022. However, a decade has passed since the drug was approved, so it is inevitable that the end of the patent on the drug is being considered. Merck has long been under pressure from investors to reduce its reliance on Keytruda, even though the PD-1 inhibitor is still expanding its leadership position. About half of Merck's sales in 2026 are projected to come from Keytruda, but this will decrease if sotatercept is successfully launched this year.
5. Datopotamab deruxtecan (Daiichi Sankyo/ AstraZeneca), est. 2028 sales: $1.8 billion
AstraZeneca and Daiichi Sankyo are collaborating in oncology research to develop an experimental cancer treatment, datopotamab deruxecan (Dato-DXd), which functions as an antibody-drug conjugate (ADC). The companies entered into a multi-billion dollar deal several years ago for ADC research and aim to license Dato-DXd for the treatment of non-small cell lung cancer and breast cancer. They anticipate submitting an application to the US Food and Drug Administration for approval in these areas soon, with marketing expected to commence later this year. However, the path to eventual approval will be challenging, as the death of a patient in one clinical trial raised questions about the drug's safety. AstraZeneca and Daiichi Sankyo stated that despite this, the drug's benefit/risk ratio remains positive. Subsequently, additional data were published demonstrating that the drug improved progression-free survival in HR-positive and HER2-negative breast cancer. Evaluate predicts that if the drug is approved this year, it will achieve sales of USD 1.8 billion in 2028.
6. Acoramidis (BridgeBio Pharma), est. 2028 sales: $1 billion
Data from Phase 3 clinical trials have demonstrated that BridgeBio's Acoramidis reduces cardiovascular-related deaths and hospitalizations. Analysts foresee significant market potential for the biotechnology company, projecting robust sales. Late last year, BridgeBio submitted a drug approval application for transthyretin to treat amyloid cardiomyopathy. However, analysts anticipate that Pfizer's tafamidis, marketed as Vyndaqel and Vyndamax, will dominate approximately two-thirds of this market in the coming years. This prediction reflects the perception that tafamidis is already well-established and may be challenging to surpass. Nevertheless, given the rapid growth of the transthyretin amyloid cardiomyopathy market, there remains potential for a second-place player to develop a successful product.
7. mRNA-1345 (Moderna), est. 2028 sales: $913 million
Moderna aims to enter the adult respiratory syncytial virus (RSV) vaccine market, competing against rivals GSK and Pfizer. If the company succeeds in obtaining approval within the next six months for use in adults aged 60 years and over, it will be positioned to vie for market share in the RSV season of 2024-2025. The success of GSK and Pfizer's vaccines quickly validated the hypothesis that RSV vaccines hold promising prospects. GSK's Arexvy vaccine generated £709 million in the third quarter and is projected to reach £1 billion for the full year. Pfizer's Abrysvo vaccine had a slower but still successful start, generating $375 million in the third quarter. At a conference in January, Moderna's Chief Financial Officer, James Mock, discussed the company's strategy for addressing challenges and expressed confidence in its ability to establish a presence in the market. Mr. Mock emphasized the appeal of Moderna's vaccine, citing its efficiency, strong benchmarks, remarkable safety and tolerability, and ease of use with pre-filled syringes—a feature he considers advantageous for productivity in retail pharmacies and doctors' surgeries. When asked about Moderna's competitive strategy in the RSV space, Mr. Mock highlighted that mRNA-1345 boasts best-in-class or near-best-in-class efficacy. Notably, after 37,000 vaccine administrations, no cases of Guillain-Barré syndrome—an adverse event associated with competitors' vaccines—have been reported.
2024 01 10
Financial Factors to Support and Challenge Pharma Next Year
While constraints such as inflation and drug pricing pressures will impact pharma’s growth in 2024, patent expiration of biologics is expected to support the industry over the next few years, research suggests.
According to results from a survey by GlobalData, the top challenges for the pharmaceutical industry in 2024 will be inflation, geopolitical conflict and drug pricing pressures. Data outcomes revealed that most respondents from Pharma considered that drug pricing and reimbursement constraints will have a negative impact in 2024.
The second biggest negative impact was thought to be geopolitical conflicts and inflation. “Geopolitical tensions and conflicts bring uncertainty to the economic outlook as deteriorating relationships are often accompanied by numerous repercussions ranging from reduced cooperation, IPO market disruptions, to economic sanctions,” noted Urte Jakimaviciute, Senior Director of Market Research at GlobalData.
Drug pricing was considered as the most negative emerging regulatory and macroeconomic trend, impeding growth for the industry, based on GlobalData’s 2019, 2020, 2021, and 2022 industry outlooks.
“Price controls – which apply to medicines in most major markets – mean that drug prices are not typically allowed to rise at the same rate as general inflation. Nevertheless, this may limit the revenue growth for pharma, with production costs on the rise: for example, due to suppliers rising the costs and employees expecting pay rises, drug production costs may increase well above inflation rates,” Jakimaviciute asserted.
However, GlobalData summarised that based on the report, influences including Environmental, Social, and Corporate governance (ESG) factors and patent expiration of biologics are predicted to positively impact the pharmaceutical industry in the subsequent years, with the latter helping to streamline operations and generate growth.
The key emerging industry trends in the biopharmaceutical industry are genomics, immuno-oncology drug development, personalized/precision medicine, real-world evidence, and remote patient monitoring among others. Genomics has been identified as the top emerging pharmaceutical industry trend for 2024.
The growth of the genomics market is attributed to the increasing prevalence of chronic diseases, plummeting DNA sequencing costs, and increasing funding for genomics. In addition, genomics has helped in tracking and understanding the spread of COVID-19, which further heightened its popularity and increased investments by the biopharmaceutical industry in this trend.
The key emerging technologies influencing the biopharmaceutical industry are AI, big data, cybersecurity, digital media, social media, virtual and augmented reality, and IoT among others. Across all geographies, AI and big data are expected to garner the highest impact on the pharmaceutical industry, in 2024.
Since the analysis of big data is best powered by AI, these technologies can be used together to achieve the desired results. Competence in emerging technologies is critical for healthcare companies to provide improved patient care at a micro level. Furthermore, using these technologies will allow companies to obtain higher levels of data insights, which will benefit operational and clinical efficiency.
The pharmaceutical industry will need to innovate and increase resilience to accelerate market expansion, the company highlighted. The report recommened that to gain a competitive edge in the market, focusing on modernising R&D models or implementing emerging technologies could support this endeavour.
The top therapy areas for planned clinical trials to be initiated, in 2024, in the biopharmaceutical industry, are oncology, central nervous system, infectious disease, cardiovascular, gastrointestinal, and metabolic disorders among others. Oncology has the highest number of trials to be initiated in 2024.
Lastly, according to the research, two other emerging industry trends in the biopharmaceutical industry are immuno-oncology drug development and real-world evidence (RWE).
2023 12 12
Clinical trials that will shape medicine in 2024
Pharma’s ongoing financial woes have continued in 2023, which has left insiders wondering when and how the field will rebound. Despite industry-wide belt-tightening, clinical trials continue even amid lingering shortages of staff — and drugs. With so many rollercoaster years since the start of the COVID-19 pandemic, it is impossible to predict exactly what the biomedical world will deliver in 2024. Nature Medicine asks leading researchers to name their top clinical trial for 2024, from base editing and a vaccine against HIV to artificial intelligence tools for lung cancer and patient triage.
Base editing for hypercholesterolemia
Amit Khera: Around 1 in every 300 people is born with heterozygous familial hypercholesterolemia, which makes it one of the most common inherited genetic conditions. Although statins can reduce the risk of cardiovascular disease in these patients, most are unable to achieve optimal LDL cholesterol levels on chronic therapy.
The heart-1 trial is a global first-in-human study of in vivo DNA base editing and has the potential to demonstrate proof of concept for PCSK9-targeted base-editing treatment approaches for durably lowering of LDL cholesterol.
VERVE-101 is an investigational, in vivo, base-editing medicine, designed to be a single-course treatment, that inactivates PCSK9 in the liver to durably decrease disease-driving LDL cholesterol. The components of VERVE-101 are an mRNA that encodes an adenine base editor, plus a guide RNA, that are packaged within a lipid nanoparticle and are delivered by intravenous infusion. Interim results were presented at the American Heart Association’s Scientific Sessions 2023.
Amit Khera is Vice President of Genomic Medicine at Verve Therapeutics, a cardiologist at Brigham and Women’s Hospital and a lecturer at Harvard Medical School, Boston, MA, USA.
AI for early lung cancer diagnosis
David Baldwin: Diagnosing lung cancer early saves lives. Although nearly three quarters of lung cancers are diagnosed late, at stage 3 or 4, earlier diagnosis at any stage allows better and more-effective treatment. A chest X-ray is most often the first test to suggest lung cancer and, if followed promptly by a computed tomography (CT) scan, can bring the diagnosis forward.
Our ongoing randomized control trial of 150,000 patients in six UK hospitals tests if artificial intelligence (AI) applied to chest X-rays as soon as they are taken shortens the time to a CT scan and time to diagnosis. We have previously shown that immediate reporting of chest X-rays by radiographers can make a substantial difference, almost halving the time to diagnosis, from 63 days to 32 days. Patients referred from primary care for chest X-ray are included for analysis by the AI. We will complete recruitment in July 2024, and we hope to have results in 2024. Our hypothesis, based on our previous research, is that we may identify lung cancer earlier, and reduce time to diagnosis as much as 50%.
AI is being implemented in hospitals often without adequate analysis of clinical impacts, something that we are concerned about. If our trial finds a significant improvement in time to diagnosis, it will probably lead to an immediate change in the standard of care to include AI at the time of chest X-ray.
David Baldwin is a respiratory physician at Nottingham University Hospitals and an honorary professor of medicine at the University of Nottingham, Nottingham, UK.
T cell vaccine for HIV
Carey Hwang: The purpose of our trial is to evaluate the safety, reactogenicity and immunogenicity of VIR-1388, a vaccine for the prevention of infection with human immunodeficiency virus (HIV). This phase 1, randomized, double-blind, placebo-controlled, multicenter study comprises adults 18–55 years of age in overall good health and without HIV who will receive one of three dose levels of VIR-1388 or placebo.
VIR-1388 is a cytomegalovirus (CMV) vector vaccine that induces strong, unique and sustained T cell responses that can potentially prevent acquisition of HIV. This follows a proof-of-concept trial of VIR-1111 that demonstrated its safety, although without a strong immune response; VIR-1388 is less attenuated than VIR-1111, and we believe it should be more immunogenic.
We recently dosed the first participants in the study. The trial is being conducted by the HIV Vaccine Trials Network at ten sites in the USA and at two sites in South Africa, with support from the US National Institute of Allergy and Infectious Diseases and the Bill & Melinda Gates Foundation. From a public health perspective, having a vaccine against HIV would obviously have tremendous impact.
Carey Hwang is a senior vice president and head of clinical research at Vir Biotechnology in San Francisco, CA, USA.
App therapy for perinatal depression
Atif Rahman: Many parts of the world face challenges in providing adequate, effective mental healthcare, but the lack of trained professionals is especially acute in low- and middle-income countries. Funded by the Research and Innovation for Global Health Transformation from the UK National Institute for Health and Care Research, a team led by the University of Liverpool has developed an app that allows a peer (a woman from the same community with no prior experience in healthcare delivery) to deliver a cognitive-therapy-based intervention to women in the second or third trimester of pregnancy who have major depression.
This trial will compare the app with the standard face-to-face version of Thinking Healthy Programme therapy delivered by community health workers in rural Pakistan. We hope that the results of this study will stimulate further innovation and research in this important area that can be a gamechanger in addressing the treatment gap for a common mental disorder globally.
Atif Rahman is a professor of child psychiatry and global mental health at the University of Liverpool, Liverpool, UK.
Stem cells for Parkinson’s disease
Malin Parmar: Most clinical trials for Parkinson’s disease study patients with advanced disease. As a result, patients who are in a much earlier phase of disease, who might be more amenable to treatment, are often left out of trials.
The STEM-PD trial will transplant dopaminergic neurons derived from human embryonic stem cells into the brains of patients 50–75 years of age with moderate Parkinson’s disease. It is important both because it is the first time a human embryonic stem cell therapy is being tested in Parkinson’s disease, and because we are targeting people with moderate disease, which gives them the most chance to benefit from the therapy. The first patients were administered doses in February 2023, and we hope to have preliminary results by the end of 2024.
Malin Parmar is a professor at the New York Stem Cell Foundation and a Robertson Investigator and is a research team manager in Developmental and Regenerative Neurobiology at Lund University, Lund, Sweden.
Machine learning for patient triage
Steven Meex: Identifying which patients in the emergency department are at high or low risk shortly after admission could help decision-making for patient care. Several clinical risk scores and triage systems for the stratification of patients have been developed, but they often underperform in clinical practice. Moreover, most of these risk scores have been only diagnostically validated in an observational cohort, but never for their actual clinical impact.
In a recent retrospective study at Maastricht University Medical Center, a novel clinical risk score, the RISKINDEX, was introduced. This used an AI model to predict the 31-day mortality of patients who sought treatment at an emergency department. The tool was developed and evaluated in four Dutch hospitals, drawing on data from 266,327 patients with 7.1 million laboratory results available. The RISKINDEX outperformed internal medicine specialists, but it remains unknown to what extent these AI models have beneficial value when implemented in clinical practice.
MARS-ED is a prospective multi-center, randomized, open-label, non-inferiority pilot clinical trial of risk-score assistance to clinicians in the emergency department. The objective is to determine the diagnostic accuracy, policy changes and clinical impact of the RISKINDEX as a basis for conducting a large-scale, multi-center, randomized trial. Recruitment for the study is about halfway complete, and we expect to see results in 2024
Steven Meex is a clinical chemist and head of the unit general clinical chemistry and hematology of the Central Diagnostic Laboratory of Maastricht UMC+, Maastricht, the Netherlands.
Intervention model for infant mental health
Dennis Ougrin: Nearly 100,000 children are currently in care across the UK, and many of them face behavioral challenges and an uncertain future. Children who are removed from their home due to abuse and neglect are one of the most vulnerable groups in any society. They have a much higher risk of suicide, unemployment and incarceration, as well as most mental health and many physical health disorders. Better approaches are needed to help these children reach their full potential, such as the New Orleans Intervention Model, which provides intensive assessment and treatment for children 0–5 years of age.
In the first randomized, controlled trial of its kind, the Best Services Trial (BeST?), we are investigating the efficacy and cost-effectiveness of the New Orleans Intervention Model for Infant Mental Health relative to the usual social work services for children 0–5 years of age who are in foster care in Glasgow and London. We will follow the children for 2.5 years and compare their mental health outcomes. Should this model prove advantageous, it might radically change the way such children are supported, not only in the UK but also globally.
Dennis Ougrin is a consultant child and adolescent psychiatrist and a reader in child and adolescent psychiatry and global mental health at King’s College London, London, UK.
More: https://www.nature.com/
2023 11 10
Commission Takes Further Steps to Address Critical Shortages of Medicines in the EU
The European Commission has adopted a set of actions to better prevent and mitigate critical medicine shortages in the EU, this winter, next winter and beyond. Recent critical shortages, including of certain antibiotics last winter, show that continued coordinated action is needed to address supply challenges and to make Europe's medicine supply chains more resilient in the long run.
To better prepare for this winter, a lot of measures have already been taken. For instance, the European Health Emergency Preparedness and Response Authority (HERA) and the European Medicines Agency (EMA) have identified key antibiotics (including specific paediatric formulations) for which they anticipate the risk of critical shortages ahead of the winter. Measures have been put in place to assure the availability of these antibiotics.
However, more needs to be done. This is why we are stepping up our actions with:
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The launch of a European Voluntary Solidarity Mechanism for medicines (October 2023): the mechanism flags a Member State' needs for a given medicine to other Member States, that can respond by redistributing medicines from their available stock.
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A Union list of critical medicines (available by the end of 2023): Once established, this list will be the first step to analyse the supply chain of selected medicines by April 2024. This analysis will then show where additional measures are needed.
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Regulatory flexibilities: Member States can use regulatory exemptions to allow medicines to reach patients in a timely manner, including extending shelf-life or the quick authorisation of alternatives. There will be a dedicated Joint Action in 2024 to promote effective use of these flexibilities.
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EU guidance on procurement of medicines to strengthen security of supply issued by the Commission by early 2024.
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EU joint procurement for next winter for antibiotics and treatments for respiratory viruses.
Member States, the EMA and the Commission have already initiated actions that go in the direction of the proposed pharmaceutical reform to prevent and mitigate critical shortages risks. The Commission will continue working together with Member States to accelerate elements of the pharmaceutical reform to enhance security of supply, where possible.
Structural measures to support long-term security of supply
To diversify supply as well as stimulate and modernise production of critical medicines with all stakeholders, the Commission intends to set up a Critical Medicines Alliance to be operational in early 2024. The Critical Medicine Alliance will add an industrial policy pillar to our European Health Union. This will allow national authorities, industry, civil society representatives, the Commission and EU agencies to coordinate action at EU level against the shortages of medicines and to address supply chain vulnerabilities.
The work of the Alliance will focus on a targeted number of critical medicines with the highest risk of shortages and impact on healthcare systems. It will draw from a varied toolbox of policy measures to mitigate risks of shortages and increase supply, including:
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Coordinating public procurement practices at EU level;
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Exploring how to diversify global supply chains through strategic partnerships;
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Boosting Europe's capacity to produce and innovate in the manufacturing of critical medicines and ingredients in coordinated way;
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Developing a common strategic approach to medicines stockpiling in the EU;
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Helping leverage and align EU and national funding.
This could pave the way for a possible “Critical Medicines Act” in the future. To that end, the Commission will launch a dedicated, preparatory study by the end of 2023, paving the way for an impact assessment.
In the first half of 2024, the Commission will also develop a common strategic approach to medicines stockpiling to prevent and mitigate shortages in cooperation with Member States.
More: https://ec.europa.eu/
2023 10 11
The Five ‘Innovation Areas’ Driving Success in Chronic Diseases
Chronic diseases are characterized by their slow progression, long-term nature, and often incurable status, imposing considerable suffering on individuals and placing a substantial strain on healthcare systems. Globally, chronic diseases such as cardiovascular disease, cancer, and diabetes contribute to the annual mortality of 41 million people, while also constituting a significant proportion of healthcare expenditures. Recent breakthroughs in precision medicine, gene therapy, and the incorporation of artificial intelligence hold promise in reshaping the conventional standards of chronic disease care. Exploring key areas of innovation becomes crucial in discerning which advancements could be most beneficial in enhancing the control and management of chronic diseases.
1. Frequency of dose administration:
People living with chronic diseases usually need to take medication very regularly. Many people with chronic conditions, such as diabetes, have multiple chronic conditions, meaning the intake of medication can be quite significant. This can have an impact on quality of life and, unsurprisingly, medication adherence. Indeed, as many as 40-50% of patients who are prescribed medications for the management of chronic conditions such as diabetes or hypertension miss their medication from time to time.
Reducing the dose frequency can be very beneficial for patients and can also help them in terms of adherence and health outcomes. Mavenclad for relapsing forms of multiple sclerosis (MS) is a great example of a significant advance in dose frequency – two very short treatment courses with pills taken for no more than ten days in a year and no injections or infusions. It improves MS treatment adherence and reduces the need for invasive procedures and intensive monitoring. For some patients, these are significant benefits.
2. Route of administration
Similar to dose frequency, the way a medication is taken can have a significant impact on patients’ quality of life. The ultimate aim is to deliver medication in a way that isn’t invasive or painful for patients, doesn’t involve significant inconvenience (ie, a hospital visit or stay), and minimises healthcare system costs.
Switching from injectables to oral solutions is a great example. Gilenya for relapsing-remitting MS and Olumiant for rheumatoid arthritis are both examples that set new standards for patients in predominantly injectable-dominated treatment landscapes at the time of their respective launches.
Innovative routes of administration can also pave the way to reaching wider patient populations. For example, the recently US Food and Drug Administration (FDA)-approved medication Zavzpret is the first nasal spray-formulated CGRP receptor antagonist for acute treatment of migraine, offering rapid pain relief for patients who cannot take oral medications due to nausea or vomiting.
3. Devices and digital solutions
Devices and digital therapeutics are increasingly becoming sources of differentiation innovation, particularly for more established therapy areas, and can play a big role in disease management.
A good example is the Digihaler range of digital asthma inhalers with built-in sensors that can detect when the inhaler is used and measure inspiratory flow rates. The data from the sensors is sent to its accompanying smartphone application via Bluetooth. Users can then review data over time and have the option to share it with healthcare professionals (HCPs).
Another interesting solution is EndeavorOTC, the first and only FDA-authorised video game treatment for people with ADHD. The treatment takes users through a multilevel interactive game that’s designed to stimulate the senses and aid attention levels. With many countries experiencing a shortage of mental health professionals, solutions like this represent a validated and easily accessible non-drug treatment option for patients.
4. Disease area and patient stratification
By their nature, chronic diseases are biologically complex and driven by multiple mechanisms. Because they can present over time with an array of different symptoms and comorbidities, this can result in patient subgroups that can have varied responses to treatment. This in turn lends itself to innovative approaches to targeting specific patient populations, which can lead to some significant breakthroughs.
For example, the recent FDA approval of Tzield heralded the first ever medication licensed to delay the onset of type 1 diabetes, signalling a major breakthrough for a disease area where historically fewer innovative treatments have been developed compared to type 2 diabetes.
Patient stratification in the treatment of chronic disease has even reached the precision medicine level. Kalydeco is the first treatment developed.
for cystic fibrosis patients harbouring specific mutations in the CFTR gene, as identified by genetic testing. Precision medicine represents an exciting area of potential for chronic disease.
5. Transformational therapy and new clinical standards
Finally, the ‘Holy Grail’ of product differentiations – a truly transformational solution that sets a new gold standard in a given chronic disease area.
Excitingly, there are many such examples. Cell and gene therapy has opened the door to medicines with genuine curative intent, such as Roctavian for haemophilia A and CAR-T therapies such as Kymirah, Carvikti and Yescarta for certain types of cancer.
Most recently, Wegovy appears to have achieved a genuine breakthrough in obesity, where meaningful weight loss has been extremely difficult to achieve by pharmacological means for many decades.
More: https://www.pmlive.com/
2023 09 18
How AI Can Revolutionize Pharma Sales And Marketing
Big Pharma companies in the US tend to spend more on sales and marketing than on R&D, according to a recent analysis by America’s Health Insurance Plans. Looking just at the top 10 pharma companies in the US, spending on sales and marketing in 2020 was $137 billion. These expense ratios often apply to smaller pharma, biotech, and medtech companies as well. It’s a huge investment.
Yet the spend can be poorly targeted. Sales reps, despite costing well over $100,000 per head, can prefer to call on the same accounts they always have, waiting an hour or more to win just a few minutes with a physician. Sales leaders can struggle to prioritize accounts, to change messaging to suit particular account contexts, or to shift their emphasis as market contexts evolve.
The challenge is becoming more acute. As drugs become ever more targeted, the market for each drug becomes smaller and the value of a single patient grows. Finding the right patient and gaining their prescription is an essential battle to win.
The Role for AI
Can AI make a difference? Absolutely. The challenge has been to interpret large quantities of ever-changing data to create clear prioritization and tailored messaging for myriad types of healthcare professionals and their employers, informing sales reps about whom to call on and in what way. It fits the pattern of a problem statement tailor-made for AI in healthcare.
Verix, an Israeli-US firm, is one company attacking this market. Shahar Cohen, Verix’s Chief Technology Officer, explains the need. “To make targeting and promotion decisions, there are getting to be too many variables for humans to consider in a handcrafted way. An oncology drug, for example, should be targeted by physician specialty; geography; typical ages seen; conditions that are typically treated; the mix of insurance used; patient volumes; institutional affiliation; and whether the doctor is seen in a first, second, or third line of treatment. There are dozens or hundreds of potential combinations of factors.”
Historically, life science companies may have used segmentation schemes based on the statistical relationship between clusters of variables. But these approaches are made for situations where there are typically less than a dozen variables in play. The validity of their predictions suffers when the variables multiply, the statistical models aren’t adapted when the market changes, and their logic can be impenetrable to people without substantial training in quantitative analysis. As complexity grows, their usefulness diminishes.
Cohen says, “From a business perspective, it’s impossible to manage too many segments. With our AI, we don’t even try. What we do is prioritize physicians for promotions, channels, and digital engagement. Then we use a supervised learning model to understand the propensity of each physician to change his or her behavior based on action initiated by the pharma company.”
Execution Challenges
Cohen admits that software alone cannot solve this challenge. He notes, “Software is never enough without a proper design of a business process to use it.” Life science companies need to entrust more decisions to these systems, and to align sales reps’ incentives to the priorities programmed into the IT. That can be a significant cultural change.
Another shift with AI is that the implementation of these processes looks different than with other IT platforms. In Verix’s case, Cohen claims, “After a few weeks, we have the data integrated and ready to use. That’s a huge change from situations where IT consultants ran the whole process. It’s quick partly because AI is a platform that continues to adapt and learn. It takes in market data from vendors and merges different sources of information. Then the system keeps re-training. The first configuration is human, of course, but after that it’s automatic.”
One way to ease acceptance is by avoiding AI black boxes. While the deep learning models seen with tools like ChatGPT could be applied to pharma sales and marketing, they aren’t required. In pharma sales and marketing, data ingested by these systems is already structured, reducing the need for deep learning, and the behavioral nature of the recommendations made by the systems enables more of a traditional, algorithmic AI approach. Through the “white box” system, companies can make their models explainable and exert supervisory control more directly.
AI’s Implications for Pharma Sales and Marketing
Through improving targeting and making rep actions more precisely guided, AI can guide the high stakes decisions required in the sales and marketing of personalized medicine. With each patient potentially worth tens of thousands of dollars – or more – this targeting is essential. At the same time, AI may enable trimming expenses on life science sales reps by guiding their actions more directly, and potentially through initiating automated, digital actions instead.
Historically, only rare innovations have enabled simultaneous leaps upward in value and downward in costs. AI may start to make these innovations commonplace.
More: https://www.forbes.com/
2023 08 21
Responsible Manufacturing Can Help Curb Drug Resistance
Antimicrobial resistance (AMR) already kills millions of people worldwide each year, and this urgent global health threat is rising faster than predicted. Among the drivers of drug resistance is the release of antibiotic waste into the environment. By responsibly managing and disposing of their antibiotic manufacturing waste, pharmaceutical companies can help combat AMR and limit their impact on the environment. A new report from the Access to Medicine Foundation, an independent non-profit organisation, zeroes in on responsible manufacturing, setting out three clear areas pharmaceutical companies can focus on to ensure they limit AMR risk when manufacturing their antibiotics.
With the need to tackle AMR growing ever more urgent, it is critical to consider all the various drivers than can fuel drug resistance – including the release of antibiotic waste into the environment.
During the manufacture of antibiotics, waste that is generated at a manufacturing site is typically released into rivers and waterways. If this wastewater contains high levels of active pharmaceutical ingredients (APIs), it poses a serious risk to the spread of AMR.
It is therefore vital than when manufacturing antibiotics, pharmaceutical companies engage in responsible manufacturing practices that limit their impact on AMR and the environment.
Since launching the first AMR Benchmark in 2018, the Foundation has been assessing how some of the world’s leading producers of antibiotics perform on limiting AMR risk from manufacturing. Despite progress, significant gaps remain.
The report analyses selected companies of varying sizes that manufacture and market antibiotics – including research-based pharmaceutical companies, generic medicine manufacturers and business-to- business providers. The report specifically mentions Abbott; Aurobindo; Centrient; Fresenius Kabi; GSK; Pfizer; Novartis and its generics division, Sandoz; Shionogi; Teva; and Viatris.
Pharmaceutical companies like these, which hold market authorisations for antibiotics, are uniquely positioned to drive change across the antibiotic supply chain, and can help transform the industry. Not only can they prioritise responsible manufacturing at the manufacturing sites they own and operate, but they have the ability to influence the standards and practices of the various third-party suppliers they contract in the production of their lifesaving medicines.
“The role of the pharmaceutical industry in tackling AMR has never been more critical. Responsible manufacturing is among the ways in which they can help prevent drug resistance from becoming uncontrollable – and can ensure that the production of their lifesaving antibiotics does not cause unintended harm to human health or the environment. We have seen that progress is possible, but we now need more companies to seize the opportunities we have identified to accelerate industry-wide change" said Jayasree K. Iyer, CEO, Access to Medicine Foundation.
To support and enable progress, this report identifies three specific areas that companies need to focus on to strengthen their responsible manufacturing practices and limit AMR risk more effectively.
1. Companies need to prioritise effective wastewater management methods to establish, quantify and monitor discharge limits for ensuring wastewater safety at their own manufacturing sites.
Specifically, to minimise AMR risk more effectively, companies can develop and implement comprehensive methods to ensure the safety of their wastewater before it is released into the environment.
By implementing comprehensive wastewater treatment processes at their manufacturing sites, Centrient and Shionogi show that reaching compliance in wastewater before releasing it is feasible. Notably, Centrient reports achieving this at its site in Tonsa, India, and in consultations for this report, Shionogi stated that it complies with discharge limits directly in its wastewater for all five of the antibiotics it manufactures.
There are also other ways in which companies can work towards limiting AMR risk in their wastewater. Sandoz, for example, uses a membrane filtration process at its main antibiotic manufacturing site in Kundl, Austria, to remove bacteria from its wastewater. To more accurately determine whether discharge limits are being met, Abbott’s pharmaceuticals division implements a synergistic combination of the mass balance approach and analysis of wastewater samples.
2. By utilising their unique positions in the supply chain, companies can transform the industry.
While pharmaceutical companies can consider replacing non-compliant suppliers, they are also in the position to share resources, expertise and analytical capabilities to support suppliers in achieving compliance with discharge limits, as demonstrated by Abbott, which provides its suppliers with wastewater sample analysis free of charge. This is particularly critical for the sustainability of the antibiotic supply chain, as there is often a heavy reliance on a limited number of producers of a certain API that is needed to manufacture an antibiotic, for example.
For this reason, pharmaceutical companies generally prefer to prioritise long-term partnerships and support suppliers in achieving compliance. Encouragingly, Centrient, GSK, Pfizer and Shionogi report that the majority of their supplier sites have achieved compliance in receiving waters.
3. Ensuring transparency and accountability across the antibiotic supply chain is crucial.
To foster progress, and to encourage other companies to follow suit, companies need to report progress at their own sites and those of their suppliers.
However, to date overall transparency has been lacking. While Centrient, GSK, Shionogi, Teva and Viatris publicly report specific details on their waste management practices, only Shionogi provides clarity on its antibacterial manufacturing supply chain. Critically, no company currently reports actual antibiotic discharge levels at its own sites or supplier sites.
What’s next?
As demonstrated by the most up to date, best-in-class examples assessed in this report, progress is possible. Moreover, beyond curbing AMR, engaging in responsible manufacturing practices will also be critical to ensuring business continuity within the context of the wider global health landscape.
“Stakeholders – including procurers, investors and regulators – are already looking to pharmaceutical companies that demonstrate their commitment to limiting their impact on AMR and the environment. This report offers practical examples that both large and small players can follow to ensure they meet these expectations. By investing in responsible manufacturing, companies can help curb rising drug resistance while positioning themselves favourably in the market.” commented Marijn Verhoef, Director of Operations and Research, Access to Medicine Foundation.
Procurers, for example, who can consider criteria related to the environment and sustainability when making purchasing decisions, are increasingly expecting companies to demonstrate comprehensive and effective practices to keep antibiotic waste within limits.
Notably, three companies in scope of the report – Fresenius Kabi, Sandoz and Viatris – were awarded with tenders as part of the Norwegian Hospital Procurement Agency’s 2019 sustainable pilot procurement programme. The companies won the tenders partly by achieving the highest score on environmental criteria, which specifically considered whether companies had environmentally friendly production in place to reduce the risk of AMR.
By following the recommendations in the report, pharmaceutical companies can ensure the production of their lifesaving antibiotics do not contribute to AMR, or negatively impact the environment, while proactively responding to stakeholders that are increasingly expecting them to demonstrate their commitment to responsible manufacturing.
2023 07 25
AI Poised To Revolutionize Drug Development
There has been much discussion recently about how artificial intelligence and machine learning (AI/ML) will revolutionize pharmaceutical research. Substantial progress has been made in the discovery and identification of new drugs enabled by AL/ML. The US Food and Drug Administration (FDA) is paving regulatory groundwork with a new discussion paper regarding AI/ML in drug development.
Rapid advances in AI-guided automation will revolutionize how scientists discover new medicines in the laboratory according to the McKinsey Global Institute. Scholars are tracking how AI/ML use has been increasing in the pharmaceutical industry, including drug discovery, drug repurposing and improving pharmaceutical productivity.
The next frontier is drug development, including driving innovation in clinical trials. AI will be used to improve clinical trial design, management, and outcomes, allowing for more efficient use of resources while also providing more accurate results.
“There is a lot that will be transformative about AI and ML in drug development,” says Fareed Melhem, SVP and Head of Medidata AI, an early leader applying advanced technology to the drug development industry. “This is a new way to understand data and gives us a better ability to search and unlock knowledge from legacy materials.”
AI Tools For Data Deluge
The scale and speed of AI for processing and analyzing vast quantities of information is far superior to traditional systems which have become overburdened by escalating growth of medical knowledge and clinical trial data. We have been witnessing a drastic increase in data digitization in the medical sector, and the ability to intelligently leverage data insights has been called the pharmaceutical industry’s next blockbuster. One report estimates that the industry could spend more than $4.5 billion on digital transformation by 2030. This investment is partial due to the rapid increase in medical data. It has been documented that medical knowledge has been expanding exponentially. Whereas the doubling time was an estimated 50 years back in 1950, it accelerated to 7 years in 1980, 3.5 years in 2010, and now medical data is expected to double up to 5 times per year or about every 70 days.
The data surge to increasing protocol complexity with industry’s focus on rare diseases as well as greater use of biomarkers and patient stratification being major drivers or increased data requirements. A single Phase 3 trial can generate an average of 3.6 million data points, roughly three times the volume of data collected by late-stage studies a decade ago. Most protocols involve an average of 263 procedures per patient, supporting approximately 20 endpoints, which is an increase of 44% since 2009. In addition, the average number of investigative sites conducting phase 2 and 3 protocols increased 33%.
Experts have said that the rise in data complexity contributes to high failure rates. Complex development processes and difficult disease mechanisms are leading to larger amounts of data and more patients in clinical trials. Success rates are frighteningly low. The likelihood of a new drug advancing to the next stage or regulatory approval is just under 14% for all therapeutic areas. Phase III trials have success rates from a low of nearly 36% for oncology to a high of over 85% for vaccines.
In order for healthcare researchers to keep up with the overwhelming growth in data, new tools such as AI have to be more widely used. AI is a technology-based system involving various advanced software tools and networks that can mimic certain human functions. One of its key attributes is the ability to handle large volumes of data with enhanced automation. AI uses several method domains, such as reasoning, knowledge representation, solution search. ML uses algorithms that can recognize patterns within a set of data that has been further classified. A subfield of the ML is deep learning (DL), which engages artificial neural networks (ANNs). The popular new applications in generative AI, such as ChatGPT, are based on algorithms that can be used to create new content, including audio, code, images, text, simulations, and videos, as described by McKinsey.
AI In The Lab
AI/ML has the potential to revolutionize drug discovery and accelerate the development of new drugs. It can make drug development cheaper and faster while improving the probability of approval. Involvement of AI in the development of a pharmaceutical product has been used to aid rational drug design AI/ML is being used in different parts of drug discovery such as drug design, synthesis, screening, polypharmacology, and repurposing. It has been proposed to reduce the time and capital required to take a drug from the laboratory to clinical trials. AI is expected to deliver value in small-molecule drug discovery by accessing new biology, improving or novel chemistry, increasing success rates, and speeding up discovery processes. AI can also help in preclinical development by testing potential drug targets on animal models and predicting how a drug might interact with them. Deep learning algorithms can analyze the structure of molecules and predict their properties such as their solubility, bio-availability, and toxicity. The prediction of the interaction of a drug with a receptor or protein is essential to understand its efficacy and effectiveness, allows the repurposing of drugs, and prevents polypharmacology. By analyzing large amounts of data, predicting drug efficacy, and optimizing drug design, AI can help researchers identify new drug targets, design new drugs, and optimize the drug discovery process.
AI In Biomarkers
Although AI/ML is getting more attention recently with the new FDA discussion paper and the broader zeitgeist around more novel AI technologies, many of these biopharmaceutical companies have been investing in their data science teams and the evidence to validate their ML models for AL-powered digital biomarkers for the better part of a decade. The industry is poised to have broader adoption with the increased regulatory support.
“Developers of predictive models for digital biomarkers have been using AI/ML extensively for the better part of a decade,” said Andy Coravos, the CEO and co-founder of HumanFirst. “More than 95% of sensor-derived digital measures have used some type of machine learning to develop the technology whether as part of the signal processing or to validate the models.” According to Humanfirst’s Atlas platform, which categorizes more than 2000 DHTs deployed across 1700 clinical trials, more than 100+ sponsors, including top biopharma companies like Janssen (Johnson & Johnson), Roche, and Regeneron have used AI-powered digital biomarkers and endpoints across a range of Phase 1 through 4 clinical trials.
Digital endpoints drive not only scientific progress but also R&D efficiencies. At the J.P. Morgan Health Care Conference this past year, Roche CFO Alan Hippe gave a presentation about how the organization using novel digital endpoints for prasinezumab Ph II (PASADENA) where the trial duration with the digital measure was two times faster and the sample size was 70% leaner, resulting in 2x return ratio.
AI In The Clinic
AI is being proposed to improve clinical trials and drug development in multiple ways. According to a life sciences digital innovation survey, 76% of respondents are currently investing in AI for clinical development. AI-enabled data collection and management can reduce the time and effort required for clinical trials, accelerate the drug development process, and help companies get new treatments to market more quickly.
“AI/ML will initially be a great help in the automation of many tasks,” says Melhem from Medidata AI. “It will be supervised, but the technology will help reduce much of the human time that is now spent on creating analytics or charts, as well as documentation of the large amount of data collected in clinical trials.”
AI technologies can be used to create structured, standardized, and digital data elements from a range of inputs and sources. These tools can interpret vast data elements, feed downstream operating systems, and help populate required reports and analyses.
Protocol design. AI-enabled study design could help optimize and accelerate the writing of appropriate targeted clinical trial protocols. It would promise to decrease the number of amendments, increase the likelihood of success, and improve overall efficiencies, in addition to reducing patient burden. AI algorithms can analyze historical clinical trial data to identify potential areas for protocol optimization, such as selecting appropriate endpoints, sample sizes, and study durations. By leveraging AI's ability to analyze complex data, researchers can design more efficient and informative trials.
Data collection. AI could help develop innovative ways of collecting trial data and reducing the need for patients to visit hospital sites. Body sensors and wearable devices such as bracelets, heart monitors, patches, and sensor-enabled clothing, can monitor vital signs and other information from patients’ homes. Advanced algorithms would help reveal real-time insights into study execution and patient adherence.
Patient screening. AI powered algorithms could facilitate identifying which patients would be appropriate for clinical trials based on their specific personal medical characteristics and aligned to trials’ enrollment criteria. In this way, patients with subtle combinations of symptoms might be identified and diagnosed early and provided options for clinical trials. AI/ML can be used to mine vast amounts of data, such as data from clinical trial databases, trial announcements, social media, medical literature, registries, and structured and unstructured data in EHRs, which can be used to match individuals to trials. AI/ML has been explored and used as part of a clinical investigation in the prediction of an individual participant’s clinical outcome based on baseline characteristics, and predictive models can be used to enrich clinical trials and may be possible to reduce variability and increase study power. Such models can also be used for participant stratification which could lead to predict the probability of a serious adverse events.
Dosing. AI/ML can be used to characterize and predict pharmacokinetics (PK) profiles after drug administration. It can also be used to study the relationship between drug exposure and response.
Real-time monitoring and safety. AI-powered systems can enable continuous monitoring of patients during clinical trials, providing real-time insights into their health status and potential adverse reactions. This can help ensure participant safety and allow for timely intervention when needed.
Adherence and retention. AI/ML can be used to monitor and improve adherence during a clinical trial through remote tools, such as smartphone alerts and reminders. It also has the potential to improve retention by increasing participants’ access to relevant trial information by enabling tools, such as AI chatbots, voice assistance, and intelligent search.
Documentation. One of the significant work loads of clinical development is the need to summarize large amounts of information, and then analyze for regulatory submissions. For example a new drug application (NDA) can have over 100,000 pages. AI automation can greatly facilitate and accelerate documentation, analysis and submission.
Data management. As pharmaceutical companies produce hundreds of thousands of pages of reports and documentation for regulators, AI can help automate the production of much of that information, and must be share across the sponsor company and external partners such as clinical research organizations, clinical trial sites, academic partners and investigators, diverse and complex real world data (RWD) extracted from electronic medical records (EHRs), medical claims, and disease registries. It would be used for a range of data cleaning and curation purposes, including duplicate participant detection and imputation of missing data values.
Digital twins and external control arms. AI/ML can also be used in the context of creating digital twins,which are essential in silico duplicates of relevant patient characteristics. This is an emerging method that can be utilized to build replicas of an individual that can dynamically reflect molecular and physiological changes and potentially predict pharmacological effects including safety events. Such a concept would be beneficial in external control arms (ECAs) where aggregated digital twins could provide a comprehensive, longitudinal, and computationally generated clinical record to understand what would have happened in a traditional placebo control arm of a clinical trial.
Regulatory AI Leadership
The FDA released a discussion paper covering AI/ML in drug development in June 2023, Using Artificial Intelligence and Machine Learning in the Development of Drug and Biological Products. The FDA’s stated goal is an initial communication with stakeholders, including academic groups, researchers, and technology developers, that is intended to promote mutual learning and discussion. The agency provides some much needed definitions including that machine learning (ML) is considered a subset of AI that allows “ML models to be developed by ML training algorithms through analysis of data, without models being explicitly programmed.”
The discussion paper outlines a wide array of how AI/ML is being implemented across drug discovery for target identification and compound screening and also in clinical research for recruitment, selection of trial participants, dose/socing regime optimization, adherence, retention, site selections, trial data collection and clinical endpoint assessments. Two other sections look at AI/ML applications for post market safety surveillance and advanced pharmaceutical manufacturing. One of the key sections of the FDA discussion paper is references for how AI/ML are being applied to real-world data (RWD) and data from digital health technologies (DHTs) in support of drug development.
The FDA states that, “AI/ML is being utilized to analyze vast amounts of data from both interventional studies (also referred to as clinical trials) and non-interventional studies (also referred to as observational studies) to make inferences regarding the safety and effectiveness of a drug. Additionally, AI/ML has the potential to inform the design and efficiency of non-traditional trials such as decentralized clinical trials, and trials incorporating the use of RWD extracted from EHRs, medical claims, or other data sources.”
Clinical Research Change Long Overdue
Traditional development for clinical trials is an unsustainably expensive process. As a rule, the cost of phase two clinical trials can be anywhere between $7 million and $20 million, while the average cost of phase three can surpass as much as $50 to $100 million. According to academics, the overall price tag to get one drug all the way to approval is well over a billion dollars.
Longer timelines contribute to the cost of developing new drugs. It often takes 10 to 12 years to bring a potential medication through research and development (R&D) to market. The clinical-trial phase averages five to seven years often due to the traditional flow of data through clinical operations systems, which can be a complicated maze of manual effort, rework, and inefficiency.
The first randomized control trial of streptomycin in pulmonary tuberculosis was carried out in 1946. Life sciences executives sometimes lament that the same processes that used over 50 years ago are still the standard. Now AI has the potential to significantly improve clinical trials in several ways. It promises to assist in various stages of the drug discovery and development process. It can help identify potential drug targets, simulate drug interactions, and optimize lead compounds. Additionally, AI can analyze scientific literature to support researchers in assessing the latest findings, potentially accelerating the development of new treatments. In drug development, AI can process and analyze vast amounts of clinical data, including patient records, genetic information and imaging data. By utilizing machine learning techniques, AI algorithms can identify patterns, detect correlations, and make predictions that could aid in identifying potential treatment outcomes, adverse events, or drug interactions.
More: https://www.forbes.com
2023 07 10
EU looks to get ahead of potential winter antibiotic shortages
The European Commission, the Heads of Medicines Agencies (HMA) and the European Medicines Agency are today issuing recommendations for actions to avoid shortages of key antibiotics used to treat respiratory infections for European patients in the next winter season.
If the demand in the coming winter season is similar to an average level of consumption in previous years, the data collected suggest that supply to the EU of oral formulations of key first and second-line antibiotics for respiratory infections will match demand in the coming winter season. EMA and the European Health Emergency Preparedness and Response Authority (HERA) will continue to work with marketing authorisation holders to strengthen measures to increase the supply of some intravenous antibiotics.
To be better prepared for the winter season, the EMA’s Executive Steering Group on Shortages and Safety of Medicinal Products (MSSG) agreed on the following recommendations for pro-active actions:
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Increase the production of key antibiotics: To avoid shortages in the upcoming autumn and winter season, EMA and HERA are recommending to continue to engage with marketing authorisation holders to step up measures to increase production. Early action ahead of the autumn and winter season should give manufacturers enough time to ensure they have sufficient manufacturing capacity to meet the demands.
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Monitoring of supply and demand: EMA and the Commission, together with Member States will continue to monitor the demand and supplies in cooperation with companies. Given that the measures taken are designed to ensure sufficient supply, all stakeholders are reminded to order medicines as normal, with no need to stockpile medicines. Stockpiling medicines can put further strain on supplies and cause or worsen shortages.
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Public awareness and prudent use: Antibiotics should be used prudently to maintain their efficacy and avoid antimicrobial resistance. Medical professionals have a key role to play, and antibiotics should only be prescribed to treat bacterial infections. They are not suitable for treating viral infections such as cold and flu, where they are not effective. Citizen awareness-raising initiatives are also advised.
Stella Kyriakides, Commissioner for Health and Food Safety, said “The availability of medicines is a crucial component of a strong European Health Union. It is essential that we take action now to prepare ahead of next winter and ensure that potential shortages of antibiotic medicines are avoided. Today we are taking an important step on measures to address our immediate needs for key antibiotics, complementing the on-going process to develop an EU list of critical medicines. Operational follow-up by HERA and EMA will now follow as a matter of priority.”
In line with the European Council conclusions of June 2023, EMA and the Commission through HERA will continue to closely monitor demand and supply and interact with marketing authorisation holders throughout the rest of the year to detect early any unexpected shortfalls of supplies and take any necessary measures.
“Based on the data received we are working to ensure that we are better prepared for the upcoming winter season,” said Professor Karl Broich, chair of the Heads of Medicines Agencies (HMA). “These early projections will allow the European medicines regulatory network to work with stakeholders to take measures in advance and prevent potential shortages for patients in the upcoming winter season.”
2023 06 22
Changes in the list of the most profitable pharmaceutical companies
For the first time in biopharma history, a company has topped the $100 billion mark in annual revenue. Pfizer passed that milestone, propelled by its massive haul from its COVID-19 products. Its BioNTech-partnered vaccine, Comirnaty, raked in $37.8 billion, and oral antiviral Paxlovid made $18.9 billion. With its windfall, Pfizer displaced Johnson & Johnson as the industry’s No. 1 revenue generator. J&J had occupied that position for a decade and had been closing in on the $100 billion mark.
Pfizer had a gaudy 23% revenue increase over 2021, the second largest jump among the top 20 revenue generators. The top gain was registered by Novo Nordisk, which saw a 26% surge, owed largely to demand for its diabetes and obesity products Ozempic and Wegovy. The only other companies in this heady territory were Merck, with a 22% increase, and AstraZeneca, with an 18% boost.
Merck rode the coattails of cancer superstar Keytruda, which rang up $20.9 billion, and COVID oral antiviral Lagevrio, which pulled in $5.7 billion. AZ owes its growth largely to $2.2 billion in sales of short-lived COVID antibody Evusheld plus major sales gains from blockbusters Calquence, Farxiga and Ultomiris.
Right behind Merck, which ranked No. 4 in revenue, was No. 5 AbbVie, which also was propelled by a single megablockbuster. Generating $21.2 billion in sales, Humira accounted for 35.6% of AbbVie’s revenue, compared to Keytruda’s 35.2% share of Merck’s sales.
The lone companies that saw a significant revenue decline were GSK (11%) and BioNTech (9%). GSK's decrease can be chalked up to its separation from consumer health unit Haleon, which reported sales of 10.9 billion pounds sterling ($13.4 billion). Take Haleon out of the equation, and GSK actually had a healthy revenue gain.
Small revenue declines were registered by Novartis (2%), Bristol Myers Squibb (0.5%) and Gilead Sciences (0.1%).
Falling out of the top 20 from last year is Viatris, which saw a 9% revenue decline in 2022, continuing a sales slide that began in 2020. Entering the top 20 is Merck KGaA on the strength of a 13% sales increase.
1.Pfizer, 2022 revenue: $100.33 billion
Sagging COVID-19 vaccine demand wasn’t enough to bring down Pfizer in 2022. The pharma behemoth once again tops the list of drugmakers by revenue, reeling in a monumental $100.3 billion last year, which represented an all-time high for the company.
Still, those salad days won’t last forever. 2023 is set to put a squeeze on Pfizer’s COVID-dependent revenues. All told, Pfizer’s BioNTech-partnered mRNA COVID shot, Comirnaty, brought home sales of $37.8 billion in 2022, just barely topping the $36.8 billion the vaccine made in 2021.
2. Johnson & Johnson, 2022 revenue: $94.94 billion
As a global healthcare conglomerate operating in pharma, medtech and consumer healthcare, Johnson & Johnson has routinely ranked at the top of this annual report. But for 2022, the company had to step aside.
J&J’s $94.9 billion in 2022 sales placed it behind only Pfizer, also among the world’s largest pharmaceutical companies. Of course, J&J’s figure includes revenues from its medical devices and consumer healthcare units, so it’s not a direct comparison to some of the other top dogs in the biopharma world.
Still, J&J does boast a massive pharma outfit, which is its largest unit. The pharma business generated $52.56 billion last year, representing nearly 2% growth from 2021. J&J's cancer drug Darzalex, immunology blockbuster Stelara, prostate cancer therapy Erleada and many other meds drove the performance, the company said in its earnings announcement (PDF).
In 2022, J&J's global sales for Imbruvica came in at $3.78 billion, a 13% decline from 2021. The drug was approved in 2013 and should still be in the midst of its growth phase, but it's being somewhat muscled out by BeiGene's newer contender.
While J&J’s pharma group has been delivering steady growth lately, the company faces at least one notable challenge in 2023 and beyond. The company's top drug, Stelara, could face biosimilars later this year. The drug’s key patents are expiring in September, and several biosim players are either in late-stage development or are already seeking FDA approval.
3. Roche, 2022 revenue: 63.28 billion Swiss francs ($66.26 billion)
Roche’s 2022 was a story about several pipeline setbacks, a battle against biosimilars and declining COVID-19 sales.
A few blockbuster drugs continued their growth trajectory in 2022. These include multiple sclerosis drug Ocrevus’ 17% increase, hemophilia A therapy Hemlibra’s 27% jump and PD-L1 inhibitor Tecentriq’s 14% rise. But the three drugs are facing competitors that could blunt their momentum.
Roche has a few new rollouts that are challenging existing mainstays. First approved in 2020, self-administered spinal muscular atrophy treatment Evrysdi crossed the blockbuster threshold in 2022, with sales up 87% to 1.1 billion Swiss francs. Macular degeneration bispecific Vabysmo, a rival to Regeneron and Bayer’s Eylea, collected 591 million Swiss francs since an FDA green light in January 2022. Last year, Roche also introduced the first CD20xCD3 blood cancer bispecific Lunsumio and is awaiting an FDA decision for a second agent, glofitamab.
4. Merck & Co., 2022 revenue: $59.28 billion
Long known for its vaccine prowess, Merck & Co. came up short in its effort to develop a shot for COVID-19. But the company did emerge with an oral antiviral treatment, Lagevrio.
While its success was short-lived—it recently flunked a trial and got a thumbs-down from Europe’s drug regulator—Lagevrio did have a lucrative run in 2022, generating $5.68 billion after pulling in $952 million in 2021.
Bell cow Keytruda also played no small part in Merck’s 2022 revenue increase. In fact, the drug's 22% sales jump last year matched the company’s revenue rise. Keytruda’s $20.9 billion figure last year accounted for 35.2% of Merck’s revenue. That’s a daunting imbalance to consider as the company faces the loss of patent protection for Keytruda in 2027-28.
Meanwhile, another of Merck’s longtime blockbusters is continuing its upward trajectory. Gardasil sales reached $6.9 billion last year, for a 22% increase on 2021. The company credits higher demand especially in China for the HPV vaccine.
Three other Merck blockbusters are on the upswing. Sales rose 5% for varicella shot Varivax (to $2.2 billion), 10% for anti-muscle relaxant Bridion (to $1.7 billion) and 13% for ovarian cancer drug Lynparza (to $1.1 billion).
5. AbbVie, 2022 revenue: $58.05 billion
While AbbVie in 2023 won't look much different from the AbbVie of prior years, a major change is underway at the Illinois drug giant.
Humira, the company’s top drug and the bestselling non-COVID product in biopharma history, has lost its market exclusivity in the U.S. AbbVie expects to retain much of Humira’s sales in the coming months as biosims slowly gain a foothold, but the erosion is only just starting.
That means AbbVie must shift its focus to other offerings. Specifically, the company is hoping immunology duo Skyrizi and Rinvoq can meet the moment and inherit Humira’s throne. The company expects the pair to eventually surpass Humira’s peak sales. Already, the drugs pulled in a combined $7.69 billion in 2022. Both drugs won their original FDA approvals in 2019.
As for Humira, the star immunology drug generated $21.24 billion last year, a 3% increase from 2021. That came despite the fact that the drug has been facing a host of biosimilar competitors in Europe. Price concessions represent the main threat to Humira in 2023 as the company competes aggressively for market share, AbbVie has warned. Volume losses could pick up in 2024 and beyond.
Aside from Humira’s biosim woes, another top AbbVie drug—hematology and oncology therapy Imbruvica—has shown signs of weakness lately. AbbVie reported global Imbruvica sales of $4.57 billion last year, a 16% decline from the same period in 2021.
On a conference call in February, AbbVie CEO Richard Gonzalez cited “recent challenging market and share dynamics” surrounding the drug. More specifically, a rival medicine from BeiGene, Brukinsa, has been turning in solid data, notching regulatory approvals and edging in on Imbruvica’s territory.
6. Novartis, 2022 revenue: $50.54 billion
Novartis launched a major overhaul in 2022. Under a new corporate structure, CEO Vas Narasimhan combined the company’s oncology and other therapeutic areas into one, and the business is now divided by U.S. and international markets. Several C-level executives along with about 8,000 employees worldwide were sent to the exit as Novartis adopted a leaner organization and expects to save about $1.5 billion in annual costs by 2024.
The Swiss pharma is taking a “U.S.-first mindset,” aiming to become a top five drugmaker in the U.S. by 2027 from its current No. 10 position, according to an investor event last September.
For 2022, heart drug Entresto grew by 31% to $4.6 billion. The combo med now looks on track to supplant inflammatory disease therapy Cosentyx—sales of which only increased by 1% to $4.8 billion—as Novartis’ best-selling product.
Multiple sclerosis drug Kesimpta, Novartis’ challenger to Roche’s Ocrevus, joined the blockbuster club as sales doubled to $1.1 billion. CDK4/6 inhibitor Kisqali also reached blockbuster land, with sales up 31% to $1.2 billion, although the drug still lags far behind rivals from Pfizer and Eli Lilly.
On the flip side, gene therapy Zolgensma reached a bottleneck, with declining sales in the latter half of 2022. Novartis attributed the slowdown to a hiatus in adding new markets, suggesting sales will hover around $1.5 billion until a potential intrathecal formulation could help the drug reach older patients around 2025.
7. Bristol Myers Squibb, 2022 revenue: $46.16 billion
Unlike most companies in the top 20, Bristol Myers Squibb saw a sales drop, however slight, at 0.5%. The decline came after the company's revenue increased by 9% in 2021.
Chalk the trend up to the loss of exclusivity for Revlimid. Sales for the multiple myeloma powerhouse peaked at $12.8 billion in 2021 but dropped 22% to $10 billion last year. BMS expects an even more significant decline in 2023 to $6.5 billion.
The company, however, is predicting an overall revenue increase of 2%. To make that happen, BMS will need healthy increases from its two other megablockbusters—Eliquis and Opdivo—and strong launches from three highly promising products that were approved last year.
Sales of anticoagulant Eliquis came in at $11.8 billion last year, which was an increase of 10%. Meanwhile, cancer immunotherapy Opdivo generated $8.2 billion last year, which also represented a 10% increase.
Eliquis (2026) and Opdivo (2028) are on the patent protection clock, as well, so it is vital that the company’s new first-in-class products flourish. Those are Opdualag for melanoma, Camzyos for obstructive hypertrophic cardiomyopathy and Sotyktu for plaque psoriasis. The company projects that the trio will generate between $10 billion and $13 billion by 2025.
BMS has a few other newer medicines that are growing rapidly. Anemia drug Reblozyl had a 30% rise to $717 million. Sales of leukemia therapy Onureg rose 70% to $124 million. And multiple sclerosis drug Zeposia saw an 87% increase to $250 million.
8. Sanofi, 2022 revenue: 43.00 billion euros ($45.22 billion)
Sanofi CEO Paul Hudson spent years reshaping the company, and it’s safe to say the French biopharma giant looks quite different than it did when he took over. But not all the company’s efforts have panned out under Hudson's watch.
In 2022, Sanofi’s global sales came in just shy of 43 billion euros, good for 7% growth, thanks largely to the contributions from Dupixent and the company’s vaccine franchise.
Dupixent, Sanofi’s star drug, generated 8.3 billion euros last year, nearly a 44% increase from the prior year. The FDA originally approved the med in 2017 for atopic dermatitis. And, recently, Dupixent's been picking up new uses—including moderate to severe asthma and eosinophilic esophagitis—to help drive growth.
Sanofi expects Dupixent sales to reach 10 billion euros in 2023. And the drug is nearing a key COPD readout.
Based on this momentum, Sanofi executives in March 2022 raised their peak sales target for the drug to 13 billion euros. As for 2023, Sanofi expects Dupixent to reach 10 billion euros.
The company does have some key launches underway. Beyfortus, an RSV prevention antibody for infants, won approval in Europe last year and is nearing a key FDA decision in the third quarter of 2023.
9. AstraZeneca, 2022 revenue: $44.35 billion
With the Alexion acquisition sinking in for a full year, AstraZeneca’s total annual revenue busted $40 billion for the first time. It reached $44.4 billion in 2022. The British pharma therefore met CEO Pascal Soriot’s ambitious sales goal, originally set in 2014 when he fended off a takeover bid from Pfizer.
For 2022, AZ's main growth came from SGLT2 inhibitor Farxiga. Boosted by its heart failure and chronic kidney disease approvals, Farxiga jumped 46% year over year to $4.4 billion.
In the oncology department, EGFR lung cancer med Tagrisso, PD-L1 inhibitor Imfinzi and BTK inhibitor Calquence all enjoyed double-digit sales growth. Ill-fated CTLA-4 inhibitor Imjudo finally scored its first FDA nod last October. However, an expansion bid for Merck-shared Lynparza in prostate cancer is now hanging in the balance, as the FDA targeted the therapy in its classwide scrutiny of PARP inhibitors .
A big focus for AZ is antibody-drug conjugates, led by Daiichi Sankyo-partnered Enhertu. For 2022, AZ recorded Enhertu collaboration revenue of $519 million and direct sales of $79 million.
After an impressive presentation that drew a standing ovation at ASCO 2022, Enhertu won a landmark FDA approval as the first agent targeted to treat advanced breast cancer expressing low levels of HER2. SVB Securities predicted the HER2-low area alone could give Enhertu $4.6 billion sales by 2030.
Despite all the commercial and R&D achievements, one historical bright spot in AZ’s business dimmed in 2022. China, instead of posting double-digit growth, pulled AZ back thanks to price reductions for both innovative drugs and generics. Last year, AZ’s China sales declined by 4% to $5.8 billion.
10. GSK, 2022 revenue: 29.32 billion pounds ($36.15 billion)
GSK went through several important changes in 2022. The British drugmaker abandoned the GlaxoSmithKline moniker in favor of the short GSK abbreviation that it’s been known as colloquially. The company officially left consumer health with the spinoff of Haleon. It’s also leaving for a new global headquarters.
Because of the Haleon separation, GSK’s reported revenue and place on our top pharma list dropped. But the company’s remaining business did grow by 13% at constant currencies.
A big part of GSK’s 2022 growth came from Shingrix. As people catch up with their vaccinations outside of COVID, Shingrix sales jumped 60% to nearly 3 billion pounds sterling.
Newer HIV meds Dovato and long-acting Cabenuva expanded their reach, helping boost GSK’s HIV sales by 12% to 5.75 billion pounds. But both Cabenuva and its PrEP sister Apretude have a ways to go to attract more use from daily orals.
Sales of Vir Biotechnology-partnered COVID antibody Xevudy reached 2.3 billion pounds. But like other anticoronavirus antibodies, Xevudy lost its authorization in the U.S., and GSK recently stopped further COVID collaboration with Vir.
Oncology remained a small part of GSK’s business, chipping in 602 million pounds at a 17% growth. GSK suffered several setbacks in oncology last year. It pulled or restricted certain ovarian cancer indications for PARP inhibitor Zejula in the U.S. and withdrew an accelerated approval for the BCMA-targeted antibody-drug conjugate Blenrep after a confirmatory trial flop.
2023 06 01
What pharma innovations must we embrace to stay competitive?
The pharmaceutical manufacturing and life sciences industry is in a state of significant disruption, and subsequent transformation. The fallout from Covid, combined with supply chain and global geopolitical challenges, plus the reality of climate change, has caused higher costs, difficult operating conditions, and market flux for the entire industry. However, these threats also run alongside an explosion of technological advancement.
The pharma industry is in a position to realise radical transformation by building on today’s budding technologies – particularly in terms of research and development (R&D), which is often the most challenging area for life sciences companies. While considerable breakthroughs have been achieved through digitalisation, opportunities to drive efficiencies within innovation are being missed.
Driving efficiencies with blockchain
As with all sectors, data is the true lifeblood of life sciences, but despite leaps in its use in R&D, there is still a lack of effective strategies that ensure the safe and effective use of big data to drive cost-effective innovation. Consider blockchain, a technology already used to significant effect in the financial services sector, similar in its high security and data regulation levels.
Clinical trial data is an essential component of drug development, and the integrity and security of this data are critical to ensuring patient safety and bringing new drugs to market. However, to realise the power of data within R&D, companies must be able to securely access and analyse sensitive data at scale.
As recent research published in the International Journal of Molecular Sciences highlights, “one of the major problems in the use of big data in medicine is that medical data has been collected across different states, hospitals, and administrative departments using different protocols.
Therefore, new infrastructure resources are required to better cross-examine the medical data through proper collaboration between different data providers.”
Blockchain distributed ledger technology can help to address some of the challenges associated with managing vast sets of clinical trial data, including data privacy, security, and transparency - especially when considering the collaborative nature of today’s R&D. By storing clinical trial data on a blockchain, pharmaceutical companies can ensure that patient data is protected and anonymised while providing greater transparency and accountability to the numerous stakeholders involved in the process.
Furthermore, blockchain technology can help streamline data management. Automating processes such as data verification and validation reduces the time and cost of managing clinical trial data, freeing up resources to focus on other aspects of drug development. Trust, transparency, and immutability - the three fundamentals of blockchain - align perfectly with the requirements of the pharma industry. By improving data security, transparency, privacy, and efficiency, blockchain can help to improve patient safety, increase trust in the drug development process, and accelerate the pace of innovation in the industry.
Making personalised precision medicine possible
One of the pervasive issues in the industry is the escalating costs of R&D. Not only that but patients and governments increasingly want more for less, especially in this new era of personalisation. As Elias A. Zerhouni, MD, former director of America’s National Health Institutes and Centres, accurately predicted, we are now in the era of P4 medicine – predictive, personalised, pre-emptive, and participatory. Now individuals expect services to be tailor-made and targeted to their specific needs.
Personalised precision medicine aims to provide individualised treatments based on a patient's genetic makeup, lifestyle, and other factors and relies heavily on the effective use of big data and AI. This is where blockchain technology could come into its own, enabling big data and AI to come together to develop hyper-personalised medicine at scale.
While personalisation is often associated with higher costs, AI can reduce the cost of drug development for hyper-personalised medicine by enabling researchers to predict drug efficacy and safety more accurately. By analysing vast amounts of data, including genetic data, medical histories, and drug response data, AI can identify biomarkers and other indicators that can predict how an individual patient will respond to a given drug. This can reduce the need for expensive clinical trials and help researchers identify promising drug candidates more quickly. AI-enabled hyper-personalisation approaches can also help researchers design clinical trials that are more targeted and efficient, reducing the cost and time required to bring a drug to market.
As the above research summarises:
Advanced machine learning approaches such as artificial intelligence and deep learning represent the future toolbox for the data-driven analytics of genomic big data. The emerging progress in these areas will be indispensable for future innovation in health care and personalised medicine.”
Putting 3D printing into practice
Developing personalised medicine through AI opens many doors, but production is another challenge. This is where 3D printing technology can support the development of small-batch medication, whether for prototyping or personalisation. For example, 3D printing can allow pharmaceutical companies to easily adjust the production process to accommodate small batch sizes, allowing the fast development of prototypes and custom medications for individual patients, supporting the aim of ‘batch of one’, through personalised precision medicine.
By enabling more targeted drug development, more efficient clinical trial design, and more accurate prediction of drug efficacy and safety, AI is critical to R&D and will be fundamental to the realisation of personalised medicine. Add to this the secure foundation of blockchain and the potential of 3D printing to support effective production, and the roadmap for future medicine is paved with today’s most innovative technologies. Through focused digitalisation within R&D, the industry can realise innovative channels for growth that could redefine life sciences for the better of all.
2023 05 04
European Commission publishes reform of pharmaceutical legislation
The European Commission’s (EC) long-anticipated pharma reform plans in the European Union have finally been unveiled, indicating a focus on improving access to medicines across the bloc while cutting down on market exclusivity.
The Commission is proposing to revise the EU's pharmaceutical legislation - the largest reform in over 20 years - to make it more agile, flexible, and adapted to the needs of citizens and businesses across the EU.
The revision will make medicines more available, accessible and affordable. It will support innovation and boost the competitiveness and attractiveness of the EU pharmaceutical industry, while promoting higher environmental standards. In addition to this reform, the Commission proposes a Council Recommendation to step up the fight against antimicrobial resistance (AMR).
The challenges this reform addresses are fundamental. Medicines authorised in the EU are still not reaching patients quickly enough and are not equally accessible in all Member States. There are significant gaps in addressing unmet medical needs, rare diseases and antimicrobial resistance (AMR). High prices for innovative treatments and shortages of medicines remain an important concern for patients and healthcare systems.
In addition, to ensure that the EU remains an attractive place for investment and a world leader in the development of medicines, it needs to adapt its rules to the digital transformation and new technologies, whilst cutting red tape and simplifying procedures. Finally, the new rules need to address the environmental impact of medicine production in line with the objectives of the European Green Deal.
To achieve these objectives, the reform addresses the entire lifecycle of medicines.
Key elements of the proposal:
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Better access to innovative and affordable medicines for patients and national health systems: new incentives will encourage companies to make their medicines available to patients in all EU countries and develop products that address unmet medical needs.
Furthermore, earlier availability of generic and biosimilar medicines will be facilitated, and market authorisation procedures simplified. Measures for greater transparency of public funding of medicines development will be introduced and the generation of comparative clinical data will be incentivised.
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Promoting innovation and competitiveness through an efficient and simplified regulatory framework: the reform will create an innovation-friendly regulatory environment for the development of new medicines and the repurposing of existing ones.
The European Medicines Agency (EMA) will provide better early regulatory and scientific support for developers of promising medicines to facilitate the fast approval and help SMEs and non-profit developers. The scientific evaluation and authorisation of medicines will be sped up (e.g., EMA authorisation procedures will take 180 days, helping reduce the current average of around 400 days) and the regulatory burden will be reduced through simplified procedures (e.g., by abolishing in most cases marketing authorisation renewal and introducing simpler procedures for generic medicines) and digitisation (e.g., electronic submissions of applications and electronic product information). The highest quality, safety, and efficacy standards for the authorisation of medicines will be maintained.
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Effective incentives for innovation: regulatory protection of up to a maximum of 12 years for innovative medicines, combined with the existing intellectual property rights, will ensure Europe remains an attractive hub for investment and innovation. To create a single market for medicines, the reform will move the current system away from its ‘one-size-fits-all' regulatory protection towards a more effective incentives framework for innovation that also promotes public health interests.
To achieve this, it proposes a minimum period of regulatory protection of 8 years that can be extended in the following cases: if medicines are launched in all Member States, if they address unmet medical needs, if comparative clinical trials are conducted, or if a new therapeutic indication is developed.
The combination of the existing intellectual property rights and the new regulatory protection periods will also safeguard the EU's competitive edge in pharmaceutical development, one of the most protective world-wide. The reform will drive efforts so that research and development will focus on the patients' greatest needs and there is more timely and equitable patient access to medicines across the EU.
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Addressing shortages of medicines and ensuring security of supply: the reform introduces new requirements for monitoring of shortages of medicines by national authorities and EMA and a stronger coordination role for EMA. Obligations on companies will be strengthened, including earlier reporting of shortages and withdrawals of medicines and development and maintenance of shortage prevention plans.
An EU-wide list of critical medicines will be established, and supply chain vulnerabilities of these medicines will be assessed, with specific recommendations on measures to be taken by companies and other supply chain stakeholders. In addition, the Commission can adopt legally binding measures to strengthen security of supply of specific critical medicines.
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Stronger protection of the environment: better enforcement of current environmental requirements will limit the potential negative consequences of medicines on the environment and public health.
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Tackling antimicrobial resistance (AMR): AMR is considered one of the top three health threats in the EU. The reform offers incentives through transferable vouchers to companies that invest in novel antimicrobials that can treat resistant pathogens, addressing the current market failure. Measures and targets for prudent use of antimicrobials, including adapted packaging and prescription requirement, will also be introduced to keep the antimicrobials effective.
More: https://ec.europa.eu/
2023 04 07
Antibiotic use dropped post-COVID. Which countries rely on them the most?
The downward trend in antibiotic use in Europe accelerated dramatically during the COVID-19 pandemic when it reached its lowest levels. However, a Eurobarometer survey found that a very large proportion of Europeans take antibiotics without justification. Their knowledge of antibiotics is also alarming.
According to Eurobarometer, there has been a gradual downward trend in the use of antibiotics in the EU. It decreased from 40 per cent in 2009 to 32 per cent in 2018, and then reached 23 per cent in 2021.
The significant fall in 2021 corresponds with the COVID-19 pandemic. "While the survey does not explore the underlying reasons behind this drop, one could surmise that the COVID-19 pandemic may have been a major influencing factor," Eurobarometer suggested.
When asked about the impact of COVID-19, over a quarter (28 per cent) of survey respondents said that the pandemic had decreased their need for antibiotics because they were ill less often due to strengthened personal protective measures such as masks, physical distancing, and better hand hygiene.
Germany and Sweden use the least antibiotics
Among the respondents from the EU, 23 per cent said "yes" when asked "Have you taken any antibiotics orally such as tablets, powder or syrup in the last 12 months?"
Antibiotic use was found to vary from 15 per cent in Sweden and Germany to 42 per cent in Malta. In France (28 per cent), it was higher than the EU average.
More than half did not undergo testing before starting the antibiotics
Participants who consumed antibiotics in the last 12 months were also asked whether they had any tests performed, such as blood or urine tests or a throat swab, to determine the cause of their illness before or at the same time they started taking the antibiotics.
More than half of Europeans (53 per cent) said they did not undergo any testing.
"Taking a test is important to discern whether an infection is truly caused by bacteria or a virus and should be standard practice before prescribing an antibiotic," the Eurobarometer report stated.
Respondents who said they had testing performed varied from 29 per cent in Poland to 67 per cent in Czechia. Participants were also less likely to have undergone diagnostic testing in Romania (31 per cent) and the Netherlands and France (both 39 per cent).
An organic compound found in trees could be the answer to fighting antibiotic-resistant infections
Knowledge of antibiotics is alarming
In the EU, only half (50 per cent) of the respondents knew that antibiotics do not kill viruses. Almost two out of five (39 per cent) incorrectly thought that antibiotics kill viruses, and 11 per cent said they did not know.
A majority of Europeans (62 per cent) correctly stated that antibiotics are ineffective against colds, while 30 per cent incorrectly believed that they are effective against colds.
Reasons for taking antibiotics
Why do we need to take antibiotics? According to the Eurobarometer report, antibiotics are only effective against bacterial infections such as urinary tract infections or strep throat.
They are not effective against viral infections such as COVID-19, colds, flu, and most types of sore throat, bronchitis, and sinus or ear infections.
The survey concluded that a substantial proportion of Europeans are still citing reasons for taking antibiotics that are not fully justified.
About one-third (30 per cent) of participants said that they still generally took antibiotics for colds (11 per cent) or flu (10 per cent), and another 9 per cent reported having taken antibiotics for COVID-19. These are diseases generally caused by viruses, not bacteria.
Antimicrobial resistance (AMR) – the ability of microorganisms to resist antimicrobial treatments, especially antibiotics – has also a direct impact on human and animal health. AMR is responsible for an estimated 33,000 deaths per year in the EU according to the European Centre for Disease Prevention and Control (ECDC).
2023 03 01
Europe's mental health in data: Which country uses the most antidepressants?
Global consumption of antidepressant drugs (AD) has increased dramatically in the last two decades, with Europeans the largest consumers. Use of antidepressants increased by nearly two and a half times from 2000 to 2020 in 18 European countries, according to Organization for Economic Cooperation and Development (OECD) data. OECD data also shows a dramatic increase in anxiety and depression during the COVID‑19 pandemic. Do the happiest countries use fewer AD drugs? How do researchers explain the sharp rise in the consumption of antidepressants?
The average antidepressant consumption across 18 European countries was 30.5 DDD per 1,000 people per day in 2000 rising to 75.3 DDD in 2020, a 147 per cent increase.
The Czech Republic recorded the highest increase with 577 per cent while it only rose by 38 per cent in France making it the lowest change in these countries between 2000 and 2020, albeit from a relatively high level.
It rose by 304 per cent in Portugal, 256 per cent in the United Kingdom, 208 per cent in Spain and 200 per cent in Germany in the same period.
Which countries have the highest antidepressant consumption?
Looking at changes in the last decade, we have data for 24 European countries.
In 2020, the consumption of AD pharmaceuticals per 1,000 people per day varied from 20 DDD in Latvia to 153 DDD in Iceland. It is followed by Portugal (131 DDD), the UK (108 DDD in 2017), Sweden (105 DDD) and Spain (87 DDD).
In 2020, the average use across these 24 countries was 68 DDD. The largest three countries by population namely Turkey (49 DDD), France (55 DDD) and Germany (62 DDD) all recorded below average use.
Any correlation between happiness and the use of antidepressants?
The short answer is no. The data on European countries does not suggest that the happier people are the less they consume antidepressants.
Iceland, which was the second happiest country in the world in 2020 according the World Happiness Report, has the highest antidepressant consumption in Europe.
Sweden, which ranked sixth in the Happiness Report, has the fourth highest use of antidepressants with 105 DDD.
Latvia which has the lowest consumption with 20 daily doses ranked 34th in the World Happiness Report. Hungary which follows Latvia with 30 DDD was on the 43th place in the happiness list.
Antidepressant consumption decreased only in Denmark in last 10 years
The consumption of AD drugs increased by 36.5 per cent between 2010 and 2020 in 24 European countries with average daily use up from 49.8 DDD to 68 DDD. Denmark is the only country to see a decrease in the use of antidepressants in the last decade with a 4 percent decline.
Estonia recorded the highest increase with 133 per cent while consumption only increased by 2 per cent in France.
It doubled in the UK, and increased by 50 per cent in Turkey. The change was under 25 per cent in 10 countries.
What about spending on antidepressant drugs?
The cost of antidepressant drug spending is a burden on citizens and their countries.
In 2020, Germany spent $812 million (€783 million) on antidepressants. Spain ($649 million or €626 million) and Italy ($456 million or €440 million) are the other leading countries for spending on antidepressants.
The ratio of spending on antidepressants to total pharmaceutical sales suggests that it is a significant cost in some countries.
In 2020, antidepressant drugs accounted for 4 per cent of pharmaceutical sales in Portugal, compared to 2.7 per cent in Spain, 2.2 per cent in Austria, 1.9 per cent in Turkey and 1.4 per cent in Germany.
The prevalence of chronic depression in Europe
There is no official comparable data on the share of people reported having chronic depression or consulting a psychologist, psychotherapist or psychiatrist.
However, survey results released by Eurostat provide some insights. In 2019 Eurostat found that 7.2 per cent of EU citizens reported having chronic depression which was only a tiny increase compared with 2014 (+0.3 percentage points).
In 2019, among EU countries Portugal (12.2 per cent) had the highest share of the population reporting chronic depression, followed by Sweden (11.7 per cent), Germany and Croatia (both 11.6 per cent).
The share of people reporting chronic depression was lowest in Romania (1.0 per cent), Bulgaria (2.7 per cent) and Malta (3.5 per cent).
It is interesting that the top two countries Iceland (15.6 per cent) and Portugal (12.2 per cent) in reporting chronic depression also had the highest antidepressant consumption with 153 DDD and 131 DDD in 2020 respectively.
The impact of COVID on mental health
Recent surveys released by the OECD found that mental health has deteriorated significantly since the start of the COVID‑19 pandemic.
From March 2020 onwards, the prevalence of anxiety and depression increased in 15 selected OECD countries, including several European ones.
The prevalence of depression in early 2020 was also double or greater than double that observed in previous years in Mexico, Australia, Belgium, Canada, France, the Czech Republic, Mexico, Sweden, the UK and the US.
However, since the survey methods differ between studies, it is not possible to offer any robust cross-country comparisons.
Did antidepressant consumption increase during the COVID?
While the prevalence of anxiety and depression rose significantly during the COVID-19 pandemic, did the consumption of antidepressant drugs also increase?
There is an increase of 10 per cent or more in consumption between 2019 and 2021 in the 14 OECD countries for which data is available. For example, use increased by 22 per cent in Latvia in these two years but only 1 per cent in Hungary.
However, this is against a background of a steady trend in the increase of antidepressants consumption over the last 20 years. Therefore, more research is needed to understand any possible impact from the pandemic on these recent increases.
Why the antidepressant consumption increase?
There are a number of potential explanations for this rise in the last two decades.
Researchers who studied the influences on antidepressant prescribing trends in the UK between 1995 and 2011, suggested that the increase can be attributed to the improved recognition of depression, availability of new AD drugs, changes in patient/GP attitudes, availability of therapies, evolving clinical guidelines, and a broadening of the range of indications treated with ADs.
2023 02 06
What happened to all the antibiotics?
In many countries across the world, there are shortages of common antibiotics. The problems have been reported across many countries in Europe, in the US, Canada, and Australia. Though the shortages have been more widely reported in more economically developed countries, the reality is that supply is likely limited globally. This poses significant issues for healthcare systems in those countries, as those in the Northern Hemisphere are dealing with infectious diseases that commonly occur in winter. At a time when COVID-19 is still putting a strain on health services, the more severe cases of infectious diseases that could arrive with antibiotic rationing would be an unwelcome addition.
The supply issues appear to be broadly impacting a number of different antibiotics, including amoxicillin, penicillin, and ceftolozane, among other products. As a result of the shortages, pharmacists have resorted to substituting alternative antibiotics, and dispensing alternative formulations. The supply constraints have also led some countries to approve companies sourcing antibiotics from outside of the country to ease demand, such as Health Canada’s decision to approve Juno Pharmaceuticals’ move to import 100,000 bottles of amoxicillin powder.
What is behind the shortages?
The current shortages have been spurred by a number of issues, some short-term and many long-term, that have plagued the manufacture and development of antibiotics. One of the reasons is the most evident: the winter months often lead to greater need for antibiotics, and that can lead to shortages. Moreover, more than any other medicine, antibiotics are more likely to face insufficient supply, with analysis by the US Pharmacopeia suggesting they are 42% more likely to be in shortage compared to all other drugs.
For its part, the US Food and Drug Administration (FDA) posted its drug shortage list, which contains various formulations of amoxicillin that are limited in supply. Three of the suppliers (Aurobindo, Hikma Pharmaceutical, and Teva Pharmaceuticals) posted their own reasons behind the shortages, which they stated had been due to an increase in demand for the products.
Seeking more detail on how these shortages occur, pharmaphorum reached out to the European Medicines Agency (EMA). A spokesperson for the agency stated, “Shortages can have multiple causes, including manufacturing problems causing delays or interruption in the production, shortages of raw materials, increased demand of medicines, distribution problems, labour disruptions, and natural disasters.”
More specifically on the current situation, they added, “Recently, shortages have been exacerbated by geopolitical events or trends, such as the war in Ukraine, the energy crisis, high inflation rates, as well as the recent surge of COVID-19 cases in China.”
Indeed, China plays a crucial role in the supply of antibiotics. According to Statista, the country accounts for approximately 42.2% of global antibiotic exports by value. This became an issue during the pandemic, as lockdowns limited some of the operations of facilities in the territory, and the recent rise in COVID-19 cases in the country means that a shortage of workers could be disrupting operations.
Finding a temporary fix
Different countries have reacted to the shortfall in antibiotic products in different ways. As mentioned, Health Canada has authorised the importation of certain products to alleviate shortages. The UK has expanded the list of medicines that cannot be exported or hoarded by wholesale dealers, in an attempt to ensure supply remains available.
In terms of what the European Union (EU) is doing to counteract the issue, the spokesperson stated, “EMA’s Medicines Shortages Single Point of Contact Working Party (SPOC WP) is taking a proactive approach to monitoring and reporting events that could affect the supply of medicines in the EU in accordance with the Regulation on EMA's Reinforced Role (Regulation (EU) 2022/123). The group is continuously gathering information from Member States and industry associations to identify signals of availability issues as early as possible.”
They added, “In the light of recent potentially critical shortages of medicines, e.g. antibiotics, EMA and the Executive Steering Group on Shortages and Safety of Medicinal Products (MSSG) are encouraging the national competent authorities in the Member States to consider the use of some of the available flexibilities, as foreseen by the EU regulatory framework for pharmaceuticals. These include allowing the exceptional supply of certain medicines that may not be authorised in a particular Member State or granting full or partial exemptions to certain labelling and packaging requirements to address severe problems in respect of the availability of some medicines.”
Looking to the long-term
However, despite the issue being exacerbated by geopolitical factors, the challenge is a broad one that requires a long-term strategy to navigate. As a ready solution that could improve reaction time to potential shortages, the FDA is advising companies to warn the agency when they notice demand spikes for products, alongside the current requirements to notify the agency when supply disruptions occur. The production of antibiotics takes months, and therefore any boost to production has a delayed ability to alleviate any disruption to supply. As a result, any earlier warnings can help regulatory agencies and countries prepare ahead of problems.
Larger questions remain around the way in which to support manufacturers to invest in production capabilities for antibiotics. With the production of generic medicine being concentrated in China and India, the ability to flexibly influence the supply of generic medicine, which most antibiotics are, is relatively limited. This is crucially tied to remuneration for the products themselves – despite being vitally important to global health, developing and manufacturing antibiotics is not a lucrative business.
As such, new strategies have to be devised to encourage continued investment into the area. The UK government recently attempted something along these lines when it launched a ‘Netflix-style’ payment model for antibiotics. This model will see the NHS in England pay a fixed annual fee for access to certain antibiotic medicine, calculated on the value to the service, rather than how many units are used to treat patients. Such innovative solutions may be the future of ensuring both that new antibiotics are developed, but also in creating a secure supply of these medicines.
2022 12 29
Trends and estimates for the pharmaceutical industry in 2023
The pharmaceutical sector has grown quickly in recent years, and by 2023 it is anticipated to reach USD 1.5 trillion. With the use of cutting-edge digital platforms, big data analytics, cloud computing, and artificial intelligence (AI), the sector is undergoing a transition. Digital technologies are developing in the pharmaceutical sector to streamline important, labor-intensive procedures. They are widely applied in the pharmaceutical value chain in several areas, including drug discovery and development, drug production, smart process automation, maximising predictive maintenance, and supply chain management.
Big data and AI are here to stay
Pharma businesses are reorganising their business models, rationalising biopharma manufacturing, improving financial decisions, reducing human error, boosting performance, and speeding up time to market thanks to AI and big data technologies. Each stage of the pharmaceutical value chain, including drug development, clinical trials, logistics, commercialization, marketing, and pharmacovigilance, benefits from the technologies’ synergistic interaction.
In 2022, it was noted that there had been an increase in alliances and collaborations between major pharmaceutical companies and suppliers of AI technology. For instance, Sanofi and Exscientia collaborated to create a pipeline of precision-engineered pharmaceuticals for oncology and immunology therapies early this year. With the help of Exscientia’s end-to-end AI-driven platform and real patient samples, the firms have teamed up once more to create about 15 novel specific molecular options against oncology and immunology illnesses.
The same year, a contract was reached between BenevolentAI and AstraZeneca to broaden their AI-powered drug development collaboration to create novel treatments for systemic lupus erythematosus (SLE) and heart failure (HF). Additionally, Pfizer strengthened its multiyear collaboration with CytoReason to create immune system models based on cells using AI-driven drug development. Pfizer is researching the immune system and creating novel immuno-oncology medications by using these biological models.
Greater use of cloud computing
Cloud computing is one of the pharma industry’s most revolutionary technologies. Compared to old approaches, the use of cloud computing is assisting pharmaceutical companies in innovating and bringing new treatments to market more quickly.
A cloud-based architecture can be used to safely store large amounts of sensitive data. It also encourages data security, compliance with regulatory bodies, and the integrity of information. Once regulatory restrictions and safety issues are addressed, the cloud computing business in the pharmaceutical industry is anticipated to expand quickly. The technology additionally guarantees expert-level security and secrecy all through the clinical trial process.
The newest fashion is blockchain
Blockchain technology has steadily established itself as a significant new technology in the pharmaceutical industry. Each record, event, or transaction is kept in sequential, verifiable blocks on an open, decentralised, secure public ledger. A secure and convenient method of legitimate information sharing between enterprises is provided by blockchain.
Blockchain has a huge impact on the production of pharmaceuticals, the tracking and verification of medical supplies, the prevention of record fraud, and research and development. Its use is expected to grow in the future years. With data, the system enhances end-to-end supply chain visibility. Blockchain fills the gaps between stakeholders by giving everyone a precise, real-time perspective of the supply chain, which is essential for ensuring the resilience of manufacturing and the supply chain.
The General Data Protection Regulations (GDPR) and the Drug Supply Chain Security Act (DSCSA) of the FDA are anticipated to go into full effect in 2023. To comply with these rules, the pharmaceutical industry will need blockchain technology to enhance traceability and visibility throughout the value chain.
Additionally, businesses are working to ensure data security in the pharmaceutical value chain, where patient confidentiality is one of the main issues during clinical studies. For instance, Nebula Genomics is seeking to improve genome sequencing by using the technique. The company is building a genomic database to eliminate middlemen and help people sell their protected genetic data in a secure manner.
Another company operating in this sector at the moment is Encrypgen. The Florida-based startup has developed a platform based on blockchain that streamlines the exchanging, storing, selling, and buying of genetic data.
R&D in personalised medicine is increasing
Tailor-made or precision medicine offers immense promise for curing a wide range of illnesses, with the worldwide custom medicine industry predicted to rise to USD 717 billion by 2025. Particularly in terms of illness treatment and prevention, customised medicine has a lot of potential to advance healthcare in the future.
Research and development processes for customised medicine have advanced significantly as a result of recent advancements in big data, artificial intelligence, and genomic testing. Due to the availability of data, improvements in processing power, and artificial intelligence (AI), researchers may now create customised treatments using a constantly expanding collection of medical data.
The leading companies in the sector are creating revolutionary, tailored treatment methods. For instance, in 2022, AMPEL BioSolutions will unveil a cutting-edge genomic platform technology that employs RNA analytics and machine learning to define the patient’s genes and aid clinicians in clinical decision-making. The doctor will then be able to decide on the best course of action, especially for each patient, by doing this.
Digitalization has become essential in all areas for pharmaceutical businesses to maintain leadership, deliver accurate results, and improve operations. Companies that stay at the forefront of technical advancements are those that continue to pave the way and open up new chances in the healthcare sector.
2022 11 17
Big pharma not doing enough to improve drug access – analysis
Since the pandemic emerged, more top drugmakers have made progress in improving access to medicines in the developing world, but those gains are largely limited to middle-income countries leaving the poorest behind, an analysis has found.
The report, published by the non-profit Access to Medicine Foundation every two years, found that companies are employing strategies including voluntary licensing and building manufacturing capacity to improve access to medicines in low- and middle-income countries, although these advances have limited depth and breadth.
While there has been progress, there remain some gaping holes that need to be prioritised in the coming years, Access to Medicine Foundation CEO Jayasree Iyer told Reuters.
The findings mirror a long-established pattern - that the pharmaceutical industry will prioritise countries where there is a market, she said.
Companies must ensure their products are registered in low-income countries too, signalling to their governments they need to invest in fortifying their healthcare systems and find ways to get the drugs into patients, Iyer said.
"If we consistently see that the industry leaves low income countries behind, then we're never going to solve the problem of access at scale," she said.
In terms of overall rankings on the Access to Medicine Index, British drugmaker GSK (GSK.L) retained the top spot, closely followed by U.S. pharma major Johnson & Johnson (J&J) (JNJ.N).
Anglo-Swedish drugmaker AstraZeneca (AZN.L) leapt to third place from seventh, helped by a series of voluntary licences issued for its COVID-19 vaccine.
Of particular concern is the lack of progress made by global drugmakers in investing in developing medicines to address emerging infectious diseases, which disproportionately affect people in developing nations and are on the rise thanks to climate change and migration, report authors highlighted.
Only five companies, including J&J, Bayer (BAYGn.DE), Merck (MRK.N) and Takeda (4502.T), are targeting such infectious diseases other than COVID, but even these projects target a small number of priority pathogens. This leaves most pathogens capable of triggering the next pandemic or epidemic unaddressed, they wrote.
"It is true that COVID has taught us that companies can be agile ... but we're banking on fewer and fewer companies to solve the world's crises when we do have a pandemic," Iyer said.
"There's hardly any investment going into it. So it is disappointing, more than it is surprising."
2022 10 20
Making pharmacy more sustainable
The growth of pollution arising from the production and consumption of synthetic chemicals now outpaces all other environmental disruptors (e.g., rising carbon dioxide emissions). Humans consume more pharmaceuticals than ever; in 2020, the volume of medicines used globally reached 4.5 trillion doses, and consumption continues to rise. Although pharmaceutical use brings huge benefits to human and animal health, it has also led to increased pharmaceutical pollution of ecosystems throughout the world.
Once administered, human pharmaceuticals enter aquatic environments through wastewater; according to the UNESCO, currently 80% of all wastewater flows into ecosystems without any form of treatment, and virtually all of the 20% that is treated still contains excreted pharmaceuticals and pathogens. As a result, pharmaceuticals are found in many different environments, on all continents, where they invoke potentially far-reaching ecological impacts for the very reason they are effective as drugs: They are molecules designed to trigger biological changes, even at extremely low concentrations.
Despite increasing evidence for potentially widespread effects, pharmaceutical pollution has not received the attention it deserves. Although there are signs that this might be changing—for example, in Europe, where the “Green Deal” commitment to tackling climate- and environment-related challenges has turned attention to pharmaceutical pollution—there is still much to be understood. For example, investigation of the ecological impact of temporary drug spikes in surface waters has been limited, meaning that the environmental effects of increased pharmaceutical consumption during the COVID-19 pandemic is unknown.
To date, most attention on the ecological effects of pharmaceuticals has focused on endocrine-disrupting compounds (which mimic, block, or interfere with hormonal signaling) and the overuse of and attendant development of resistance to antibiotics. Although this is of huge importance for the environment and human health, the ecological impact of most other drugs remains unknown, especially within the setting of complex natural ecosystems. To date, 88% of drugs that target human proteins are lacking comprehensive environmental toxicity data.
Furthermore, viruses play an important role in regulating ecosystem processes, but the potential impact of antivirals is yet to be studied in depth, despite widespread contamination of freshwater with these substances. This is pressing because antiviral consumption has greatly increased owing to the COVID-19 pandemic.
Ecological impacts of active pharmaceutical ingredients must be studied in different environments throughout the world. Socio-ecological systems in tropical and arid regions are not equivalent to those in the temperate zone. However, to date, most research about the presence and impact of pharmaceuticals in the environment has been conducted in high-income, temperate-zone countries.
Consequently, very little is known about the ecological effects of drug pollution in most of the world’s ecosystems, especially those of the Global South, where water is often scarce and used repeatedly by people before being discharged containing high drug concentrations. For example, in 2017, ~1.7 billion treatments were delivered to >1 billion individuals in mass drug administration programs aimed at combating neglected tropical diseases. These included ivermectin and azithromycin, which have deleterious environmental effects, but the impact of this mass drug administration remains unknown.
In addition, these countries also frequently experience pollution from the manufacturing of drugs that are used in high-income countries, but the ecological impacts of this are also largely unstudied. These issues must be addressed to meet the ambitious Sustainable Development Goals (SDGs) set by the United Nations, especially objective 6: clear water and sanitation.
The pharmaceutical industry and its customers must assess and adjust many aspects of the pharmaceutical life cycle. “Greener” drugs with lower environmental impact—for example, that are less biologically reactive or more easily eliminated from the environment—need to be designed and formulated, and drug manufacturing facilities need better wastewater management.
The judicious and responsible use of drugs in both human and veterinary medicine is necessary, and nonpharmacological interventions should be prioritized when possible. Educating health care practitioners about the impact of drug pollution is also key. Redefining the concept of “rational use of drugs” by including a One-Health approach should be considered.
Drugs should be removed from wastewater before they enter the environment. The technical solutions to clean pharmaceuticals from wastewater exist (e.g., advanced oxidation with ozone), but to date, only a few countries (e.g., Switzerland) have implemented large-scale tertiary treatment of wastewater. Owing to the relatively high cost and lack of regulatory pressure, implementation of treatments that remove drugs from wastewater is still uncommon.
However, although conflicts of interest may be unavoidable, it is possible to limit the negative consequences of pharmaceuticals while still allowing society to benefit. It is time to make green pharmacotherapy a reality.
More: https://www.science.org/
2022 08 18
Why aren’t digital pills taking off?
While ingestible sensors offer a chance to improve adherence, their use in vulnerable populations remains rife with challenges.
In 2017, the US Food and Drug Administration (FDA) broke ground by approving the first drug with an embedded biosensor to track its use. Proteus Health’s Abilify MyCite had an ingestible sensor that could track if a patient consumed the drug.
Proteus’s profile grew considerably; it was soon valued at $1.5 billion. The enthusiasm for digital approaches involving sensors, apps, and wearables that could transmit information across systems, commonly grouped under the term Internet of Things, also spread.
Yet, despite the landmark FDA approval, digital pills have not exploded in pharma. Privacy and logistical concerns, especially while studying such applications for vulnerable populations, have lingered.
Not even three years later, Proteus filed for bankruptcy.
There’s no dearth of interest, however, in applying digital solutions to healthcare. “Controlling the parameters of our health with an app has become comfortable, and many people welcome this type of progress,” says Marina Morla Gonzalez, an assistant lecturer in the public law department at Universidad de León in Spain. Glucose monitoring through implantable sensors, and portable electrocardiograms have also become increasingly common in real life.
Still, the field is in its infancy when it comes to unequivocally establishing a financial and clinical benefit with digital pills. But researchers hope that this may soon change.
Privacy concerns present but variable
When you’re dealing with vulnerable populations, it is important to make sure patients are aware of what the sensors in digital pills intend to do; that there is no penalty if they forget a medication, and that they are intended to be helpful and not a hindrance, says Susanne Haga, PhD, associate research professor at the Sanford School of Public Policy, Duke University.
“You don’t want to use this to stigmatize patients or to ‘catch them lying.’ You want to use it to show what is working well, or tell ‘if you’re struggling in this situation, I want to identify the trigger,’” says Dr. Jose Castillo-Mancilla, associate professor, School of Medicine, University of Colorado, Anschutz.
Abilify MyCite may not have been the best case study for how future applications of these ingestible biosensors may be viewed or taken up by patients and providers, says Haga. Abilify MyCite was FDA-approved to treat schizophrenia, and manic and mixed episodes associated with bipolar I disorder, and as an add-on treatment for depression. “One of the strongest mistrusts that existed towards [Abilify MyCite] was how are you going to treat a patient with a medicine that has a sensor to track and control them, when one of their main pathologies is paranoia,” says Gonzalez.
When it comes to working with vulnerable populations, it is important to have an open dialogue that allows participants to learn more about the technology and have the research team available to them for any questions, Castillo-Mancilla says.
Dr Peter Chai, an ingestible biosensors expert, has been involved in several studies to improve and measure drug adherence, including trials that have enrolled men who have sex with men with substance abuse disorders, and individuals with stigmatized or marginalized identities. His early research involved fieldwork on how people would integrate such interventions into their daily life. “It takes some groundwork to develop those messages. Once that is done, you can be honest with people who want to use the device or participate in the study,” says Chai, who is also an assistant professor of emergency medicine at Brigham and Women’s Hospital.
But traditional consent forms and data privacy policies are written in a very complex vocabulary that is very hard to understand even for a lawyer, says Gonzalez. A patient probably does not know what they’re signing, but still do it in order to access whatever treatment they need, she adds.
When it comes to privacy-related concerns, the possible risk of third parties using that data in a way that affects the patient, for example while looking for a job, is a factor that contributes to loss of trust in this type of technology, says Gonzalez. Haga, whose background deals in genetics, says that such privacy concerns are still present in that field, but she did not view the information being transmitted through digital sensors any differently than other health data that we may or may not knowingly make available.
“A lot of those [scenarios] are opinions and kind of philosophical debates. When you go and talk to people who actually use the system, you find that that’s not the case,” says Chai.
In Chai’s experience, participants found such a digital approach improves their connection to the medical system. “It potentially helps individuals lend some kind of concrete evidence to discussions with their healthcare provider. So, it’s not essentially a ‘he said, she said’ about adherence, but this is more like what happened….’I’m trying to be better or I’m doing well or I’m not doing well.’” In the trial led by Castillo-Mancilla, when a patient takes a pill, that creates a signal that is stored in a phone. This is then acknowledged with a ‘Thank you.’ Something as simple as that positive feedback has been well-received, he says.
The fundamental thing is that the patient agrees to be treated with this type of therapy, says Gonzalez, adding that they would opt for such technologies if they think it empowers them to have better control of their health.
“Just because the population is vulnerable or difficult, doesn’t mean that you shouldn’t do anything for them. It’s the right thing to do, and it’s hard but I think hard things tend to be correct,” says Chai.
Payers remain to be convinced
Ultimately, the commercialization of these products is what’s going to bring them into the hands of the people who need them the most, says Chai. However, as seen in the last few years, that is easier said than done.
Proteus is not the only company that has found the market challenging. Pear Therapeutics, a digital therapeutics company with three FDA-authorized products, recently announced an operations restructuring exercise. In an SEC filing made public on 25 July, Pear announced the company would lay off 9% of its full-time staff, among other things, and shift focus to prioritizing certain commercial efforts.
“The issue here is we haven’t created a compelling case from an economic, payer, and user space, all at the same time, in such a way that everybody can win,” says Buffkin. If the field were to show a pharma company that a digital pill can reduce costs because it can improve adherence, which then consequently prevents a negative outcome, then that’s a compelling enough reason for all the stakeholders in this field, says Castillo-Mancilla. Payers have to see the health and economic benefit to buy in.
2022 07 12
Pharma can no longer rely on traditional sales and marketing to launch new drugs
The pharma industry has seen dramatic shifts in scientific innovation over the last decade with a trend toward orphan markets, personalized medicine, higher priced products and new modalities including cell and gene therapies, oligonucleotides and others. COVID served to further accelerate scientific innovation and remarkably rapid development and rollout of ground-breaking products. However, the commercial models employed by companies that launched new products during 2020 and 2021 were largely based on traditional sales and marketing approaches, despite the massive shifts in the pharmaceutical industry and the healthcare environment at large.
Most pharma drug launches over the past three years have “underperformed expectations” and show the need for a major change in thinking about how to sell and market new meds.
That’s according to a new report, "Empowering the Next-Generation Launch Model," out by life science consultants Trinity Life Sciences, which shows 62% of drug launches between September 2019 and December 2021 “underperformed expectations.”
These launches have not kept up with the “dramatic shifts” in scientific innovation over the last decade, the report found.
“No longer can life sciences companies rely on the traditional sales and marketing approaches to launching new products,” Leslie Orne, president and chief commercial officer of Trinity Life Sciences, said in a statement. “Commercial teams need to be in lockstep with the innovation that is happening on the development side—it is time to re-think and revolutionize the commercial model.”
COVID has also had a clear impact on launches. In terms of timing, the report's focus coincides largely with the start of the pandemic disruption in early 2020. The Trinity team, however, said the pandemic merely “exposed and exacerbated challenges” on the road to launches and that these challenges had been emerging prior to the crisis.
The rate of underperformance can be attributed to many factors, but Trinity found it mainly reflects “a failure to employ innovative strategies to effectively engage patients, providers and payers throughout both clinical development and commercialization.”
The report’s authors say the “ingrained conventional wisdom” indicates the overall success of a drug launch is determined in the six months following approval, something that has in turn shaped stakeholders’ expectations.
Instead, companies should plan more time for initial uptake and develop a go-to-market strategy that recognizes an extended (and more sustainable) early period of sales growth, the report says.
Pharma companies should not be dissuaded from pursuing opportunities that may “require greater patience” in the initial one to two years post-launch to “reap substantial rewards in the many years that follow.” And investors should be prepared to fund a company “well past the initial launch phase” before profitability can be achieved, the report says.
Lastly, companies should prepare a “robust [return on investment] assessment” to prioritize customer targets and right-size a field team, Trinity says.
“This feeds into a forecast that will serve as a reliable foundation for planning commercial activities and that will allow leadership teams to set realistic expectations with investors ahead of launch.”
2022 06 03
The future of clinical trials: artificial patients, synthetic data and real-time analysis
In this article, we summarise three concepts that are already/ will soon become familiar for everyone interested in the future of clinical trials. These are 1. the concept of the artificial patient, 2. using synthetic data and 3. real-time analyses. What connects the three is that all are potential tools to make clinical trials faster, cheaper and safer.
The artificial patient
As of today, there is no final, widely accepted definition of what an artificial/virtual/synthetic patient is. An artificial patient is a set of data representing the desired human characteristics the best possible way that is based on large amounts of real patient data, without actually including any backtracable real-patient data.
Artificial patients can be the answer to more than one problems of modern medicine. One of them is patient privacy. With ever more machine learning and deep learning models being used, A.I. needs huge amounts of data to learn from. But providing a lot of real patient data is against their privacy rights, and we have seen ample examples of how bad an idea it is to allow random companies to access heaps of sensitive health data. On the other hand, taking a real-life dataset of existing humans, and generating a synthetic dataset that resembles the original in all important aspects without actually including anything personal can be a solution.
What is it good for? Artificial patients can be used for a number of things, from medical education to clinical trials, this time we are only focusing on the latter. One day, virtual patients might become the go-to tools for
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estimating efficiency and potential side effects of promising drug molecules or optimising the use of existing ones,
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to model the success rate of future medical devices or treatment methods,
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or, as the latest, they can substitute the placebo control group for clinical trials
While using artificial patients for drug development or medical device development is a promising field, there is a long way to go until the models can reach the required complexity while being truly representative of the human population.
On the other hand, artificial patients as the placebo control group have arrived. AppliedVR recently conducted a trial for VR treatment for chronic back pain patients. And instead of recruiting patients to sign up for the trials to not receive the treatment (being the control group), they decided to turn to an existing database of chronic pain patients, provided by healthcare data company Komodo Health.
Using real-world data as a patient group in a trial, often known as a synthetic control arm, can make research trials more efficient — companies don’t have to enrol as many people in clinical trials and can guarantee that those who apply will indeed receive the treatment.
Synthetic control groups can also improve equity in clinical research. “That allows us to go look at all those different subpopulations and underrepresented patient populations to see if they have different outcomes,” Web Sun, president and co-founder of Komodo Health says.
Synthetic data
If you felt like your head started spinning from dealing with the concept of the artificial patient, behold, synthetic data is probably an even wilder ride.
What is it? The definition is simpler this time: synthetic data is the use of A.I. to create datasets that mimic the real world.
Why is it? Because we 1. don’t have enough real-world data or 2. don’t want to use real-world (sensitive) data.
Feeding any algorithm that needs massive amounts of data to learn and either develop new prediction capabilities or recognise patterns. Synthetic data is widely used in a number of industries and segments, not just in medicine, but also in self-driving vehicles, security, robotics, fraud protection, insurance models, military and so on.
Artificial intelligence has earned its place in multiple fields of medicine, from recognising patterns, supporting diagnoses and setting up treatment pathways to optimising healthcare logistics. Smart algorithms can sift through large volumes of data no man can, deriving clear-cut trends from such analyses.
Privacy concerns limit the amount of available data in medicine. Working with sensitive patient data is a tricky issue. It seems we cannot keep our privacy intact AND also benefit from A.I.’s advantages in our care. We saw in many cases how sensitive information can get leaked even unintentionally – and we are not even talking about hacking or privacy, just a poorly protected database. New methods like federated learning might make it possible to do this without breaching patients’ privacy, but its scope is limited.
That’s when synthetic data comes in. It can fill in the missing data, making it possible to produce entirely fabricated patient datasets that are just as useful for training A.I. as the real thing, while keeping patient data protected.
Using synthetic data could help overcome this challenge as the training could focus on such variables, making use of real-world environments. Using the above-mentioned example, how to diagnose melanoma on dark skin toned patients – as often previous algorithms have failed to be able to do so.
Real-time / decentralised clinical trials
What is it? The use of electronic health data/records/devices to carry out clinical trials in near real-time with patients not needed to be present on site.
Why is it? Real-time trials offer faster results and the possibility of participants to directly connect to other patients, share their experiences and get access to results.
What is it good for? More committed participants and faster results.
We have seen a few good examples in recent months.
One is that of Royal-Philips rolling out a new at-home ECG system for decentralised clinical trial use. The company is pitching this new technology as a way for clinical trial participants to record ECG data without travelling to a clinical site or requiring an in-home clinician. Data of trial participants can be transmitted near real-time to the cloud servers for analysis.
A number of digital health companies are designing tools to enable decentralised trials. In September, digital wound-care company Swift Medical launched a new digital-imaging platform designed to support decentralised clinical trials. The technology was designed to aid in large-scale image collection and management in order for researchers to monitor the impact of medical interventions at various sites or at home.
Deploying such advanced technologies in clinical trials will require pharmaceutical and biotech companies to commit not only financially but also to the idea that technologies can significantly contribute to clinical trials, making drugs cheaper, making the process faster and much more importantly, making the lives of patients participating in them more comfortable.
2022 05 12
Precision medicine for complex chronic diseases: how near are we?
Targeting medicines against the genetic and molecular drivers of lung, breast and other tumours has helped transform cancer care for many patients, yet precision medicine for complex chronic diseases has lagged behind.
Precision medicines combine information about an individual’s unique biology, matching the right treatment to the right person at the right time of their disease to improve patient outcomes.
Since the completion of the Human Genome Project in 2003, significant progress in understanding the genetic and molecular drivers of diseases has led to the design of precision medicines that specifically target underlying disease mechanisms. Alongside these treatment advances, diagnostics have been developed that identify biomarkers in tissues and body fluids that can be used to predict a patient’s likely response to a medicine.
To date, the delivery of precision medicines and their associated companion and complementary diagnostics has been largely in the treatment of cancer, with researchers targeting the underlying causal biology including individual driver mutations. So why is it taking longer to apply a similar approach for chronic diseases?
The challenges of complexity
The simple answer is that treating chronic diseases, such as heart failure and chronic obstructive pulmonary disease (COPD), is more complex than targeting a single mechanism that may be a driver of a given tumour type.
Common chronic diseases can be driven by multiple genetic and molecular defects, many still unrecognised or not fully understood, combined with environmental factors to cause a range of disease-producing mechanisms.
Furthermore, by their nature, chronic diseases progress over time and often present differently between patients depending on other aspects of their health. Researchers are therefore not only looking for the proverbial needle in a haystack, but for multiple needles in a continually changing haystack.
Early scientific research into the complexity and clinical or mechanistic heterogeneity of common chronic diseases, however, has started to break down this seemingly insurmountable barrier. In heart failure, for example, we are not looking at the heart in isolation but at patients as a whole, many with multiple co-morbidities such as obesity and diabetes which may influence the progression of heart failure; adding to the disease complexity and affecting the likely response to heart failure therapies. In COPD, an estimated 500 different genes are expressed abnormally in a sample of diseased lung tissue, with considerable heterogeneity in expression of these genes between patients. Many of these could play a mechanistic role and represent a viable drug target.
To understand these hugely complicated datasets arising from multi-omic analyses, the latest artificial intelligence and machine learning technologies are used to discover associations between data and diseases including the development of knowledge graphs describing the molecular interconnections and interdependences within a disease. Without these advances, it is almost impossible to imagine being able to make sense of all the exciting data we are accumulating.
Currently the management of many complex chronic diseases is the same for most patients, based on clinical diagnosis and symptomatic treatment. This limits opportunities to slow or halt disease progression leading to potentially sub‑optimal outcomes for some patients. However, with evolving research, this paradigm is changing.
2022 04 06
Fighting the public health threat of falsified medicines
It is extremely difficult to tell how many people lose their lives due to counterfeit drugs every year globally. The share of counterfeit drugs is around 10% globally. It is as high as 30% in some countries, while it stays below 1% in others. Some may guess that the one-digit numbers belong to the richest countries, while the big figures are typical in Sub-Saharan Africa. But fake drugs are getting a thing in the Western world too.
According to the World Health Organisation (WHO), fake malaria drugs alone could be responsible for 270,000 deaths in Africa every single year. The dangers of fake or substandard drugs are obvious: sick people lose time while waiting to recover after taking the fake pills. Instead of reaching out for medical help, they are assured they are on track to get better.
In some cases, fake drugs ‘only’ do nothing. In other cases, they are actually harmful, making the patient’s prospects even worse. And while people trust the tablets they took, malaria is damaging their bodies. Oftentimes to a degree that their lives cannot be saved by the time they get actual medical treatment.
No money? No medication!
And the lack of genuine malaria remedies is only the tip of the fake drugs iceberg. The pandemic highlighted the magnitude of this phenomenon. Fake COVID test kits, fake vaccines, falsified chloroquine, and falsified protection products have been taking their toll in large parts of both Africa and Asia. During a public health crisis such as the current pandemic, the fight against pharmaceutical trafficking becomes even more acute and urgent – WHO stresses.
Why are people taking fake drugs? There are several reasons. The first, of course, is money. Older malaria drugs – Chloroquine, the standard, effective drug for decades – were as cheap as 0.1 US dollars per course. But they stopped working. The new effective drugs— artemisinin combination therapy (ACT)1—today cost US$2.40 per course wholesale and can be marked up to five times that amount in pharmacies in Africa. Easy to see how extremely unavailable it is in countries where many people, living in remote, undeveloped regions often don’t even earn that amount of money in a month.
The other reason is the lack of access to proper medical care. Geographical distance, lack of funds to travel to the point of healthcare, to pay for healthcare and medications are the most important reasons.
Fake drugs are getting a problem in the Western World as well
But if you think fake drugs are only the problems of the third world, you are wrong. With online pharmacies and drug delivery services becoming increasingly popular, substandard or falsified drugs have found their way to Western customers as well.
According to this report, in the U.S. alone, 19 million consumers are buying medications outside of the safe drug supply. Either from online pharmacies outside the legal jurisdiction of their government or other unlicensed sources.
In 2018, out of twelve thousand internet drug sellers, 94.8% were non-compliant and listed as “not recommended”. The majority of these sites (89%) did not require a valid prescription. That is one of the many signs that a pharmacy is not legitimate.
Three pillars of the solution: people, government, pharma
The solution has to come from three directions, all supporting each other. The first and most important is raising awareness. People have to know that fake drugs exist, and how easily they can slip into the supply chain even in the most developed countries. Every tool that allows users to check if a drug is safe is a big step forward.
The second pillar is to ensure the integrity of the drug supply chain, from the production right to the customer. This includes government initiatives. Calling on doctors to draw their attention to the importance of the source they buy from, a good reporting system and other regulatory measures.
The third pillar is to include various safety measures in the supply chain of drugs. The US has come probably the closest to this with its Drug Supply Chain Security Act. It requires data collection at each transfer of the chain of custody: when one company transfers the product to another or one person gives it to another person, and so on. If a product is tracked at each transfer, not only is it possible to more easily prevent counterfeit medicines from entering the supply but the process is also more data-rich overall.
New methods emerging: blockchain, watermarks and mobile phones
However good the regulation, if people keep buying drugs from shady online outlets or on street corners. That is why simple solutions, easily implementable by the users, are extremely important.
While it is nice that pharma companies can track the route of a box, we could take one more step further. Tracking each and every pill. If anyone could (and would!) check the actual pill before taking it, it would dramatically decrease the chances of fake drug complications.
How can you check if your medication is genuine? There are several ideas. From blockchain-based identification of each box to this brand-new idea of printing tiny, unique watermarks to individual pills.
Adding traceability directly on a pill could require adding numerous manufacturing and data management steps. But it is possible – so it seems.
A research team at Purdue University’s Weldon School of Biomedical Engineering came up with an idea to make this possible. The solution is very similar to paper watermarks used on bills. It is especially ingenious as it only requires one tool practically every individual has on this planet: a mobile phone.
“We are affixing a watermark on an individual medicine that is readable by a smartphone camera to extract a hidden digital key,” Young Kim, associate head for research explains. This cyber-physical watermark is printed on specialised fluorescent silk with FDA-approved food dye through an inkjet printer. A common technique bakers use for placing edible photos on cakes.
This is a solution without using any synthetic or artificial materials, and fluorescent silk makes a counterfeiter very difficult to duplicate the watermark – the team says.
2022 03 28
What is the technological future of medication management?
When a patient heads to a healthcare institution and is prescribed medicines, one might expect them to stick to the regimen to improve their health. However, up to 50% of patients fail to adhere to their medications as prescribed.
And around the world, millions are taking medicines. In the U.K., over 26% of adults take prescription medications. Across the Atlantic ocean, in the U.S., some 66% of the adult population take prescription drugs. Down in the South Hemisphere, around 35% of Australians take such medicine on a daily basis.
With those millions of medicine-taking people comes the inevitable risk of medication errors. In England alone, researchers found that over 237 million medication errors are made every year.
Ultimately, these add up to the losses incurred both financially and, more importantly, in terms of patient lives. One way to tackle these issues is through medication management. The UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) defines medicine management as “the clinical, cost-effective and safe use of medicines to ensure patients get the maximum benefit from the medicines they need, while at the same time minimising potential harm”.
However, the methods employed have been stagnant for long and still leave room for improvements, as the numbers show. As healthcare steadily pivots towards digital health, what future do the relevant, novel technologies paint for medication management? Stick with us as this is what we will explore in this article.
Medication management, compliance and the empowered patient
When it comes to medication management, there are two key sides: the administrative (clinician/pharmacist) and the patient side. This means that issues can sprout from either end of the spectrum and at every stage of the process from prescribing to dispensing. Considering recent statistics from the U.K. alone, medication errors cost the NHS around £98 million and over 1700 lives every year; and researchers consider these losses as “definitely avoidable”. On a global scale, it’s easy to project the magnitude to be severalfold.
This represents a significant burden that is preventable but that novel technologies in the digital health era can help address. However, the same era is defined by the empowered patient. Equipped with digital health tools, having access to their own health data and engaged in online patient communities, the latter are more proactive in managing their care. As such, being tasked to comply with their treatment regimen in the traditional, passive way is an obsolete, if not offensive, approach for the empowered patient.
The patient of the digital health age would rather be more proactive in this process. Engaging them in medication management can thankfully be made possible through digital health approaches and we’ll consider those in the next section.
Medication management in the digital health age
With the accessibility of connected devices and remote care, patients and their physicians can share a new dynamic to manage medication. Physicians can prescribe digital health tools to assist patients in managing their medications while also enabling them to monitor adherence. For their part, patients can turn to their doctors remotely in case of concerns. Below are 5 digital health tools that could improve medication management and enable this new dynamic.
1. Smart pill dispensers
Digitally enabled pill dispensers like the Hero Automatic Medication Dispenser and MedaCube deliver audible and visual cues to remind patients to take their medications at the right time and dose. Caregivers and physicians can even track adherence through the companion app.
2. Medication reminder apps
For a software-based option, patients can adopt apps like CareZone and Medisafe. These help manage medication regimens and can sync the data with a caregiver or doctor. With this approach, they can be notified when refills are needed or when the patient doesn’t respond to several missed dosage alerts.
3. Digital therapeutics
Other types of software also hold promise in the future of medication management, in particular digital therapeutics (DTx). These evidence-based, clinically evaluated software undergo regulatory oversight to ensure their safe and effective use as well as their clinical impact.
One such DTx product is Insula, a prescription-only software that assists type 2 diabetics to manage their condition. It recommends patients personalised insulin doses as well as acts as a coach in managing their diabetes.
4. Digital pills
The longer-term goal would be to integrate a tracking option in pills themselves, and this is where digital pills come into play. While adoption is still in the early stages, some companies are working on drugs with ingestible electronics for adherence monitoring purposes. Of note, not all drugs will bear the same technology; as digital pills are more appropriate in niche areas where compliance can and should be improved with this tech. For example, patients with severe mental health conditions or the elderly having challenges adhering to their medications can benefit more from digital pills.
etectRx’s FDA-approved digital pill gives patients more control over when monitoring starts. It involves a removable lanyard rather than a patch, which patients can remove after taking their medicine. Infármate’s SIGUEMED comes with a trackable blister pack, alerting caregivers when patients take a pill. A companion app informs of the right time to take the pill and alerts contact persons if a mistake has been made.
5. Telemedical platforms
All of these above options can be supplemented by telemedicine consultations. Through these, patients and physicians can routinely hold remote consultations to ensure proper adherence of patients’ to medications. The latter can in turn use such platforms to request advice or raise concerns with their physicians. Given their rising adoption as a result of the pandemic, patients and physicians can leverage the availability of such platforms for medication management.
While these tools hold promise in medication management and enable the digital health patient to become more proactive in their care, they are not definitive solutions. They might not be accessible in all communities due to lack of resources, but their assistance can nevertheless help put a dent in the avoidable burden associated with medication management.
2022 02 22
Cutting the carbon footprint of pharma’s supply chain
Pharma, like every other industry, has an impact on the environment – and in the face of a climate crisis, it has a responsibility to find ways to mitigate this harm. In a GlobalData survey conducted last year, 43% of respondents considered the environment the most pressing ESG (environmental, social and governance) issue for the pharmaceutical sector to address. As members of an industry that emits more greenhouse gases than the automotive sector, drug companies must make a concerted effort to reduce the carbon footprint associated with getting their medicines to patients – but how?
Starting sustainably
Every stage of the pharmaceutical supply chain has a carbon footprint – right down to the very beginning, where the raw materials for active pharmaceutical ingredients (APIs) are sourced.
Jing-Ke Weng is co-founder of Double Rainbow Biosciences, a sustainable biotech dedicated to developing new therapeutics with minimal impact on the environment. He says the manufacture of APIs for small-molecule drugs relies largely on chemicals derived from petroleum, a fossil fuel.
There are many energy-intensive steps in the chemical synthesis of raw materials and solvents, Weng explains. And the amount of carbon it takes to make these molecules is usually not calculated, he says.
Double Rainbow’s long-term vision is to replace fossil fuel-reliant manufacturing with methods based on synthetic biology and inspired by the chemical processes seen in nature.
Mitigating manufacturing’s impact
Manufacturing the finished pharmaceutical product is another carbon-intensive stage of a pharma company’s supply chain. One of the most effective ways of reducing carbon emissions associated with drug production is to adopt continuous manufacturing, an efficient alternative to batch manufacturing, that combines multiple separate production stages into one single, continuous production line.
In 2014, US biotech Amgen opened a $200m biomanufacturing plant incorporating continuous purification methods in Singapore. Compared with a traditional manufacturing facility, Amgen’s plant produces 69% less carbon emissions – a promising figure for a company that plans to be carbon neutral in its operations by 2027.
French healthcare company Sanofi followed suit in 2019, opening a continuous manufacturing plant in Massachusetts, US, that it said will generate 80% less carbon emissions than the company’s first-generation facility.
Winds of change: renewable energy in pharma manufacturing
If pharma and biotech companies are to meaningfully curb their carbon emissions over the next few years, moving away from fossil fuels and shifting to greener, renewable energy resources is a necessary step.
In 2021, energy and automation giant Schneider Electric – ranked the world’s most sustainable corporation that same year – launched the Energize programme to increase the pharmaceutical industry’s access to renewable energy. Through Energize, drugmakers will be given the opportunity to access and contract for renewable energy throughout their value chain. Ten global pharma companies signed up to promote the initiative and encourage their suppliers to decarbonise with renewables: AstraZeneca, Biogen, GSK, Johnson & Johnson (J&J), MSD, Novartis, Pfizer, Novo Nordisk, Sanofi, and Takeda.
When it comes to greener energy, at least, the pharma industry at large appears committed to making changes. In addition to collaborating on the Energize programme, many large drug companies have set ambitious carbon reduction targets for themselves.
Cold chain shipping: a carbon culprit
While drug manufacturing carries a large carbon footprint, the steps taken to distribute medicines out of the factory and into patients’ hands also have a significant impact on the environment.
One way by which distribution in pharma contributes to climate change is the use of cold chain shipping, which allows temperature-sensitive products, like insulin or some vaccines, to be transported at controlled temperatures that do not compromise their efficacy or safety. Usually, these temperatures must be maintained all the way from point of manufacture to the patient.
Refrigerated vehicles require additional energy to power the cooling systems, known as transport refrigeration units (TRU), that keep products at a controlled temperature.
An obvious way to reduce the carbon emissions caused by cold chain shipping is by investing in greener fuels and energy sources for transport vehicles. Hydrotreated vegetable oil, a renewable, bio-based fuel that can be used in diesel engines, can reduce greenhouse gas emissions by up to 90% compared with regular diesel. Other alternative fuels include compressed natural gas, liquefied natural gas, liquefied petroleum gas (LPG), and their renewable counterparts—biomethane and bio-LPG. Research published in 2019 found that gas-fuelled vehicles saved over 1,400 tonnes of Co2 emissions compared to diesel across a two-year period.
Lawson adds that companies should look to invest in localised manufacturing – producing the medicines nearer to where they’ll be used – as a means of reducing the carbon footprint associated with pharmaceutical distribution.
Packaging and pollution
It’s not just fuel consumption that makes cold chain shipping environmentally unsound. Peter Gisel-Ekdahl is CEO of Envirotainer, a provider of secure cold chain solutions for the air transport of pharmaceuticals. He says the environmental impact of cold transport can be reduced by adopting alternatives to ‘passive packaging’ that consist of less-than-eco-friendly single-use plastics, dry ice, and cardboard, to keep medicines at a stable temperature.
“An ‘active’ solution is different; it’s more like a reusable, battery-powered unit,” Gisel-Ekdahl says. “These are used in a circular economy where sharing, leasing, reusing, repairing, refurbishing and recycling is central to their design.”
“Over the lifetime of their use, these thermally controlled containers are environmentally superior, he explains. “In fact, greenhouse gas emissions associated with active solutions are more than 90% lower than passive, even considering an extra leg of return transport.”
Whether passive or active systems are employed, reducing the amount of packaging materials used can make a big difference. By finding more efficient ways to store products in transport vehicles, pharma giant J&J cut the amount of packaging used to transport its medicines by 60%. Taking up less shipping space means fewer vehicles are needed to transport the products – which translates into less carbon emissions at the distribution stage of the supply chain.
A greener future for pharma?
The solutions for an eco-friendlier supply chain are gaining traction in the pharma industry – but is there a desire to change?
Weng is hopeful that the industry will move in that direction in the next decade. “But the strongest force will come from the market itself – just the inability to do business as usual will be the main driving force for companies to think, at a fundamental level, how they could replace non-sustainable steps with more sustainable biology-based approaches.”
Government action, for example enforcing stricter environmental standards or incentivising greener operations, would also push pharma companies in the right direction, Weng adds.
2022 01 11
Pharmaceuticals in 2022
The pharma industry has been pivotal during the pandemic and this focus looks set to continue into 2022. The coronavirus pandemic has not only boosted many pharmaceutical companies’ revenues, it has also bolstered their reputations.
The value of pharma innovation is undoubted, after COVID-19 vaccines were developed at record speed. Pharma manufacturing and supply chains have so far delivered 7.5bn vaccine doses, and also kept supplies of most other medicines on track. The industry is entering 2022 in good shape, less vilified, more central to government policy, and with less budget pressure than in recent years. Even so, there will be challenges ahead.
Market growth will not be one of them. Overall, we expect pharmaceutical sales in the 60 biggest markets worldwide to increase by 4.6% in US-dollar terms to about US$1.5trn. That is about half the growth rate seen in 2021, but still faster than that seen in most of the previous decade. Little wonder that many pharma companies, having upgraded their 2021 earnings forecasts, are issuing bullish (if tentative) forecasts for 2022. COVID will continue to be a major driver for several companies.
Vaccine supply problems should also ease in 2022. Unicef, which is monitoring COVID vaccine supply deals as part of its COVAX programme, reckons that global production capacity will jump from 8.5bn doses this year to over 40bn doses next. Some of that extra capacity will come from India, where the export ban for vaccines has finally been lifted despite the lingering caseload. India already has deals in place to produce 2.8bn doses. More groundbreaking, though, will be expansion of vaccine production in Africa.
From late 2022 the Pasteur institute of Dakar in Senegal will start producing 25m doses of coronavirus vaccines a month, with international backing. Vaccine capacity will also rise in Egypt, South Africa and Morocco as existing plants expand.
Other medicines will see some supply-chain problems – albeit not as extreme as in other markets. The disruption suffered in 2020 - when many countries were scrambling for medtech supplies - has died down, but the pharmaceuticals industry is still suffering from the rapid rise in shipping and delivery costs, particularly in Asian shipping routes. Coronavirus cases and power cuts are also causing occasional production stoppages in China, affecting supplies of active pharmaceutical ingredients as well as inputs such as magnesium (used to make aluminium foil packaging). The effect is likely to last into 2022, and will push the EU and US, among others, to move ahead with the reshoring initiatives they started last year, when supply-chain disruption underlined their reliance on China.
New regulations will also hold risks. The EU, for example, plans a full overhaul of its regulatory framework for pharmaceuticals, encompassing everything from incentivising innovation to securing supplies and ensuring equal access. A 12-week consultation process began in September 2021, with the aim of delivering the final revisions of the regulation by the end of 2022. The pharmaceutical industry is likely to be most concerned about the European Commission’s efforts to boost market competition, with the aim of promoting generics and bringing down prices. A promise to overhaul the R&D incentives on offer, such as extended marketing exclusivity, will also ring alarm bells, although the results are unlikely to involve drastic cutbacks. As always, the Commission will need to tread carefully between innovation and affordability.
Other countries are also pursuing new regulations. In 2022 Japan will implement the final stage of its rules requiring barcodes on pharma packaging – the kind of small-sounding reform that can be surprisingly difficult to implement. Taiwan is also introducing rules to improve traceability, though these will not come into effect until January 2023. In China, meanwhile, drugmakers are still feeling their way forward as the National Health Commission tries to centralise and streamline drug procurement processes, including instructions designed to ensure “more rational drug use”.
Tax changes will prompt strategic reviews. Although the outlook for innovation is generally good, companies will need to be planning ahead for tax changes. Most immediately, in the US more elements of the 2017 tax reforms will come into effect, changing the extent to which companies can offset their R&D expenses. Some of the changes will promote innovation, by extending new credits, but others will be detrimental, particularly when it comes to orphan drugs.
None of this will prevent more innovation. Not only is global R&D spending in good shape, but the pandemic has accelerated several pathways to innovation. One is the access to and use of digital data to drive research. Pharma companies may not want to repeat the cooperative data sharing that helped them to develop COVID vaccines, but the data analytics tools are still there and being used. The sudden move from site-based clinical trials to virtual trials during lockdowns, though painful at the time, has opened up new ways of getting such trials done quickly. The same has happened for diagnostics, with the rapid roll-out of home testing kits. Liquid biopsies, an innovation that predated the pandemic, are now helping clinicians to catch up with missed non-COVID investigations. And mRNA and lipid nanoparticles, the innovation behind some of the vaccines, may have new uses for other treatments. The pandemic was an ill wind for the world, but it has blown the pharma industry some good.
2021 12 28
Digital transformation will improve clinical trial process, say experts
In a new survey, 35 percent of healthcare professionals believe that digital transformation would improve pre-clinical and clinical trials.
A total 35 percent of healthcare professionals worldwide believe that digital transformation — involving the use of technologies such as artificial intelligence (AI), big data, cloud computing, application programming interfaces (API) and digital platforms — would improve both pre-clinical and clinical trials, according to a survey by GlobalData. GlobalData also noted that drug developers that had not considered virtual trials will have no option but to rapidly invest in this technology if they want to stay competitive.
The survey, published in GlobalData’s latest report, ‘Digital Transformation and Emerging Technology in the Healthcare Industry – 2021 Edition’, reveals that digitalisation of pre-clinical and clinical trials was positively viewed by 39 percent of healthcare and pharmaceutical professionals in North America, also 39 percent in Europe and 28 percent in the Asia-Pacific region. Furthermore, these results were in line with those found in both the 2019 and 2020 surveys.
According to GlobalData, clinical trials are said to be one of the most critical and expensive stage in drug development process. Planning and trial design, patient recruitment, site selection and coordination, logistic and operation, and the time needed, all constitute a significant cost.
“While the digitalisation of clinical trials had been well underway prior to COVID-19, the pandemic has accelerated this trend,” commented Elton Kwok, Associate Research Manager in Pharma at GlobalData. “These new technologies are helping reduce time, labour, costs, and other associated risk factors. Healthcare professionals are seeing the benefits of digital transformation in everything from patient selection and enrolment, to target identification, drug approval, data collection and analysis, supply chain management, marketing and sales.
“Emerging technologies such as AI, big data, APIs, and digital platforms are expected to improve patient selection, enhance data collection, integration and analysis, while at the same time reduce time, labour, and associated risks,” Kwok continued. “Over 130 respondents to a survey by GlobalData, believe that eventually total cost would be lowered. Their businesses would stay competitive in the market and business continuity could be maintained.”
2021 11 16
Revealed: the pharma companies leading the way in artificial intelligence
Novartis and Johnson & Johnson are among the companies best positioned to take advantage of future artificial intelligence disruption in the pharmaceuticals industry, Global Data analysis shows.
The assessment comes from GlobalData’s Thematic Research ecosystem, which ranks companies on a scale of one to five based on their likelihood to tackle challenges like artificial intelligence and emerge as long-term winners of the pharmaceuticals sector.
According to our analysis, Novartis, Johnson & Johnson, AstraZeneca, Bristol Myers Squibb, GlaxoSmithKline and Bayer are the companies best positioned to benefit from investments in artificial intelligence, all of them recording scores of five out of five in GlobalData’s Drug Development Thematic Scorecard.
Novartis, for example, has advertised for 327 new artificial intelligence jobs from October 2020 to September 2021, has completed one deal related to artificial intelligence with other companies and mentioned artificial intelligence in company filings 10 times.
Johnson & Johnson indicated good levels of AI investment, with the company looking for 969 new artificial intelligence jobs since October 2020 and mentioning artificial intelligence in filings 30 times.
The table below shows how GlobalData analysts scored the biggest companies in the pharmaceuticals industry on their artificial intelligence performance, as well as the number of new artificial intelligence jobs, deals, patents and mentions in company reports since October 2020.
Higher numbers usually indicate that a company has spent more time and resources on improving its artificial intelligence performance, or that artificial intelligence is at least at the top of executives’ minds. However, it may not always mean that it is doing better than the competition.
A high number of mentions of artificial intelligence in quarterly company filings could indicate either that the company is reaping the rewards of previous investments, or that it needs to invest more to catch up with the rest of the industry. Similarly, a high number of deals could indicate that a company is dominating the market, or that it is using mergers and acquisitions to fill in gaps in its offering.
Nevertheless, these trends are useful in showing us the extent to which top executives in the pharmaceuticals sector – and at specific organisations – think about artificial intelligence, and the extent to which they stake their future on it.
2021 10 21
Major disruptors for the generics and biosimilars market
In a recent whitepaper the International Generic and Biosimilar Medicines Association (IGBA) described their 2030 Vision for the Generic and Biosimilar Medicines Industry, outlining six factors promoting growth and six major disrupting factors for the industry over the next decade.
All about the price tag
Pricing pressures placed on marketed generics and biosimilars remain relatively high. Key contributors include reimbursement restrictions and regulations both in Europe and the rest of the world; and accelerated biosimilar launch price erosion with the stronger and faster adoption of biosimilars – one paper describes how, in instances with five or more competitors, a 70 percent decrease can be reached relatively quickly. According to IGBA, if not managed properly, these factors could create an unfavourable economic environment for such products and significantly negatively impact on the availability and access of critical products
Jumping through regulatory hoops
The whitepaper suggests that there are still regulatory barriers facing complex generics entering the markets, even in developed countries, including the amount of evidence required for the approval of products in areas like respiratory and long-acting injectables, and the requirement for market-specific reference samples across key geographies.
For biosimilars, clinical trial cost requirements remain substantial, resulting in a high degree of risk of developers and manufacturers, and certain emerging markets have regulatory processes with failings that slow product development, such as complex approval processes or a lack of clarity on clinical study design. So, streamlining the regulatory guidance is critical to enabling accelerated product development, states IGBA.
Shaky supply
The vulnerability of supply chains, particularly sources of active pharmaceutical ingredients (APIs) and key starting materials (KSMs), was revealed by the COVID-19 pandemic and the whitepaper cautions that various governments’ attempts to combat them – through on-shoring of manufacturing or building local manufacturing competency – could result in protectionism, damaging overall product access and economic viability by raising costs for both companies and patients. Additionally, sustainability pressures will impact supply chains, said IGBA, as companies are called upon to reduce greenhouse gas emissions and waste. The paper states that balancing global and local supply, as well as quality and environmental requirements will be critical going forward.
Novel modalities present new problems
While enabling growth, the emergence of novel technologies in the pipeline is increasing the complexity of products and simultaneously the risk-profile of investments. The whitepaper stresses that the emergence of new modalities such as cell and gene therapies will require a new set of capabilities and thus substantial investment.
Emerging markets: a hurdle to development
Emerging markets present severe competitive and structural market challenges for generics and biosimilars companies, according to IGBA. The competitiveness of local markets makes it a challenge for players to build their presence, while factors such as the drive to localise manufacturing, increasing pricing controls and unharmonised regulatory requirements only add to the undertaking. In addition, currency effects can make growth difficult, rendering the markets less attractive and limiting their development. Traditional growth strategies, such as partnerships, mergers and acquisitions have also shown mixed performance, according to the whitepaper, limiting player engagement in these markets. The IGBA states in the report that international players will have to carefully select their target markets and adopt a focused portfolio approach in order to draw business from local players, familiar with the market dynamics, and avoid costly expansion endeavours.
Evolving commercial strategies and channels
The whitepaper also reveals that there is significant disruption in the channel landscape, as consolidation gradually increases in Europe and non-traditional players enter the market looking to disrupt it. The authors state that such disturbances have already had a significant impact in markets like China. They added that both developed and emerging markets are seeing increasingly aggressive moves, such as the scale up of end-to-end solutions and the entry of digital-first players and large conglomerates along the value chain. According to IGBA, to survive under these market conditions, generics companies will need to adapt their portfolio strategies to make them attractive to new customers and partners, while also innovating their commercial models to reduce risk.
2021 08 05
The Top Vitamin and Supplement Trends of 2021
The COVID-19 pandemic has led to dramatic changes in our day-to-day lives.
With more people focused on improving their health and fitness, vitamin and supplement use has risen dramatically. In fact, the nutritional supplement industry observed its highest growth in over 2 decades, with a 12.1% increase in 2020.
This trajectory is expected to continue into 2021, with many people looking to support their health through supplements. That said, you may wonder which supplement trends are topping the list.
Here are the top vitamin and supplement trends in 2021.
1. Stress SOS
2020 was filled with stressful, life-changing events that led to an increase in mental health issues and stress across all age groups (1Trusted Source, 2Trusted Source, 3Trusted Source, 4Trusted Source).
As a result, many people are turning to supplements to support their mental health. In fact, a report by Coherent Market Insights anticipates a growth rate of 8.5% in the brain and mental health supplement market over the next 6 years.
Expect to see ingredients like magnesium, B-complex vitamins, L-theanine, melatonin, valerian, chamomile, and cannabidiol (CBD) become more popular, as they’re linked to better sleep and lower levels of stress and anxiety (5Trusted Source, 6Trusted Source, 7Trusted Source, 8Trusted Source, 9Trusted Source).
Furthermore, plant-based adaptogens have soared in popularity, as they may support the body’s stress response. In particular, ashwagandha and rhodiola are popular Ayurvedic herbs that may lower anxiety, cortisol levels, and mild depressive symptoms (10Trusted Source, 11Trusted Source, 12Trusted Source).
Although these ingredients may benefit your mental health, you should always consult a healthcare provider before taking new supplements — especially if you’re already on medications to treat depression, anxiety, or other mental illnesses.
2. Beauty support
Expect beauty and skin care supplements to grow significantly this year. Whether it be a scoop of collagen in a smoothie or a pinch of turmeric on a salad, treating skin issues from the inside out will be a growing priority.
In fact, according to Google Ads, search inquiries for collagen increased 33% from March 2020 to December 2020, suggesting there’s an increased interest in beauty as a subsection of the supplement industry.
Top beauty-boosting ingredients include collagen peptides, vitamin C, omega-3s, hyaluronic acid, ceramides, and antioxidant-rich green tea and vegetable blends.
In particular, a diet rich in collagen peptides, vitamin C, and omega-3s is linked to better skin health and fewer signs of aging, such as wrinkles and fine lines (13Trusted Source, 14Trusted Source).
Despite their popularity, don’t expect these supplements to compensate for poor skin care and lifestyle practices. Wearing sunscreen daily, getting quality sleep, exercising regularly, and eating a nutritious diet are key to healthy skin (15Trusted Source, 16Trusted Source, 17Trusted Source).
3. Immune health
The COVID-19 pandemic taught us all to prioritize our health. As such, there’s been a 50% reported increase in consumers seeking out immune-supporting supplements.
Expect to see supplements that contain ingredients like zinc, selenium, B complex vitamins, and vitamins C and D, as well as alternative health remedies like elderberry, echinacea, astragalus, turmeric, and ginger.
Furthermore, some industry experts believe that medicinal mushroom extracts will become a part of this trend. In particular, chaga, cordyceps, lion’s mane, and reishi mushrooms have been used in traditional medicine to strengthen the immune system (18Trusted Source, 19Trusted Source).
Notably, the medicinal mushroom industry is projected to be worth $261.8 million by 2026.
It’s important to note that despite their potential role in supporting immune health, no nutritional supplements have been shown to prevent or cure illnesses, such as COVID-19 (20Trusted Source, 21Trusted Source, 22Trusted Source, 23Trusted Source).
4. Vitamin D
Vitamin D continues to lead the charge as the top nutrient for overall health. It plays a key role in immunity, mental well-being, bone and skin health, and protecting against chronic disease (24Trusted Source, 25Trusted Source, 26Trusted Source).
In particular, this vitamin made headlines in 2020, as various studies associated low vitamin D levels with an increased risk of COVID-19 complications. However, further research is needed (27Trusted Source, 28Trusted Source, 29Trusted Source).
While your skin naturally produces vitamin D when exposed to the sun, concerns about ultraviolet (UV) rays and regional limitations to sunlight have led many consumers to supplement with vitamin D.
Notably, the vitamin D market is expected to increase 7.2% by 2025.
5. More enjoyable supplement experiences
Consumers are looking for convenient, more enjoyable experiences with supplements, which will involve shifting away from traditional pills. In particular, flavored gummy vitamins for people of all ages will continue to grow in popularity.
What’s more, consumers can expect companies to provide various options to consume other supplement products. For example, collagen peptides are beginning to be offered as powders, flavored drinks, capsules, and gummies.
As supplements are now considered to be part of a healthy lifestyle, companies will be expected to create products that fit seamlessly into your daily routine.
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6. Trustworthy companies
Health-savvy consumers are becoming increasingly knowledgeable about their own health needs. At the same time, they’re growing tired of misleading or false health claims, which has led to a rise in distrust of the supplement and pharmaceutical industries.
With 59% of people reporting that they’ll gladly pay more for a brand they trust, companies will be expected to be transparent and honest about their products, disclosing not only their ingredients but also their efficacy, safety, and absorption.
Companies may also be held accountable for ensuring their health claims are backed by science, as the growing distrust of the industry has stemmed from companies making claims that have little to no scientific backing.
This ongoing expectation of trust and transparency will likely lead consumers to turn to health professionals and online forums to weigh in on product quality and call the shots on acceptable practices by the supplement industry.
The bottom line
In 2021, you should expect to see several changes in the vitamin and supplement industry.
After the life-changing pandemic, we’ll see more supplement companies focusing on nutrition for both the mind and body, as well as being more transparent about the ingredients they’re using.
While these trends are exciting, it’s important to speak to your healthcare provider before trying new supplements.
As a dietitian, I look forward to seeing positive changes in the supplement industry toward a more well-rounded, evidence-based approach.
2021 08 05
Regulators call for clinical research to include pregnant and breastfeeding women
The FDA and other global regulators are co-operating to address inadequacies in clinical research that leave pregnant and breastfeeding women lacking data on which to base their medical decisions.
Global regulators are calling for a paradigm shift in clinical research and urging pharma to include pregnant and breastfeeding women in their studies in order to “address inadequacies in research”.
In a statement, various stakeholders at the US Food and Drug Administration (FDA) asserted that too often pregnant and breastfeeding women, along with their health care providers, are left to make important clinical decisions on the basis of little to no data on the safety or effectiveness of medications used during and shortly after pregnancy. This, they said, is true even for serious medical conditions like asthma, epilepsy, depression and COVID-19.
Though the FDA stakeholders emphasised that the agency and its global regulatory counterparts had taken steps to address the approach to studying medical products in these populations in recent years, they said that the spotlight cast by COVID-19 had prompted further action.
According to the spokespeople, pregnant women were shown to suffer increased risks for severe illness and serious adverse obstetrical outcomes with COVID-19; however, despite not being specifically excluded, no pregnant women were enrolled in clinical trials of the COVID-19 treatment remdesivir. Additionally, despite regulatory encouragement, COVID-19 vaccine developers did not enrol pregnant women in prelicensure COVID-19 vaccine trials (including for the Pfizer-BioNTech, Moderna, or Janssen COVID-19 vaccines), they said.
Moreover, to address the issue with regards to COVID-19, the International Coalition of Medicines Regulatory Authorities hosted a pregnancy and lactation workshop in February 2021, led by the FDA, EMA, MHRA and others. The meeting supported international collaboration and explored how experiences with providing COVID-19 therapies and vaccines to pregnant and breastfeeding women can be leveraged to support a global strategy for collecting systemic data for this patient population.
The FDA stakeholders concluded that “a major paradigm shift is necessary from the systemic exclusion to the inclusion of pregnant and breastfeeding women in clinical studies” adding that it is essential to ensure patients who are pregnant or breastfeeding can make informed decisions about their health.
“Developing a roadmap for change, through international co-operation and harmonisation, will take much care, consideration and communication between all stakeholders. Despite persistent challenges, there is strong interest from the international regulatory community to continue the momentum in this important area of women’s health.”
2021 07 07
Key clinical trials to watch for the rest of 2021
The first half of 2021 was historic for biotech. The Food and Drug Administration cleared the first new Alzheimer's drug in nearly two decades, a controversial decision with far-reaching consequences. Multiple effective coronavirus vaccines and antibody drugs helped bring the pandemic under control in the U.S., while the first-ever treatment for a common genetic driver of cancer won approval.
But there's plenty more in store over the next six months or so, with important clinical trial results still to come. A big moment for gene editing, for instance, is just around the corner. A closely watched antiviral drug could change how COVID-19 is treated. And a gene therapy could bring new hope to patients with a debilitating neurological disease. Here are eight important clinical trials to watch.
Antiviral drug for the treatment of COVID-19
Merck, one of the world's top infectious disease drug developers, has played only a supporting role during the coronavirus pandemic. Two of its experimental vaccines failed in January and, in April, Merck scrapped a drug for hospitalized COVID-19 patients after regulators demanded more testing. The company's biggest contribution to the pandemic response to date is helping to make doses of Johnson & Johnson's vaccine.
A drug named molnupiravir, however, could change that if it succeeds in an ongoing Phase 3 trial. Merck is co-developing the drug, an antiviral pill, with Ridgeback, which licensed the medicine last year from Emory University. In early testing, molnupiravir helped patients who had recently developed COVID-19 symptoms clear the virus more quickly, leading to a $1.2 billion supply deal with the U.S. government.
If testing proves molnupiravir can protect patients from COVID-19's worst outcomes, the pill could become the first oral medicine for the disease and a convenient alternative to the intravenously infused antibody drugs from Regeneron, Eli Lilly and Vir Biotechnology.
Pfizer and Atea Pharmaceuticals and partner Roche also have oral treatments in clinical testing. An alliance between Novartis and Molecular Partners is developing an infused antiviral as well. But they all trail Merck, which expects Phase 3 results later this year, possibly by September.
Gene editing tool for the treatment of amyloidosis
CRISPR gene editing has taken significant steps forward over the past year. Jennifer Doudna and Emmanuelle Charpentier, two of the scientists who invented its use as a gene editing tool, won the Nobel Prize in chemistry. A treatment from CRISPR Therapeutics and Vertex has shown potential to be a functional cure for the blood diseases sickle cell and beta thalassemia. Multiple clinical trials are now underway in other diseases as well, as are efforts to better refine and improve on the technology.
The next CRISPR milestone could be imminent. Intellia Therapeutics, which Doudna co-founded, will soon disclose the first results from an early-stage trial of a gene editing treatment for the rare disease transthyretin amyloidosis, a potentially deadly condition that can cause damage to the heart and nerves.
Unlike CRISPR and Vertex's treatment, in which gene editing is performed outside the body, Intellia's is infused into the bloodstream. That's a riskier approach, as it involves altering DNA directly in the body. But it's a far less complex procedure, and one that could significantly broaden the reach for CRISPR technology if proven safe and effective.
A positive result could also establish Intellia's treatment as a potential rival to Alnylam, Ionis and Pfizer, which each have marketed treatments for transthyretin amyloidosis. Alnylam and Ionis have already proven that stopping the buildup of the mutated transthyretin protein can help treat the disease. Intellia aims to use gene editing to achieve the same goal.
Monoclonal antibodies for the treatment of lung cancer
Drugmakers have tried for years to find medicines that can boost the effects of immunotherapy, which has changed the way many cancers are treated but still doesn't help everyone. The search for the perfect pairing has yielded mixed results, with chemotherapy remaining the most effective partner. But new approaches keep emerging, and one of them that's gained considerable traction is a class of antibody drugs that block a protein called TIGIT.
Early studies from Roche and Merck indicated adding anti-TIGIT antibodies could improve on a type of immunotherapy known as checkpoint inhibitors. Gilead, Bristol Myers Squibb, BeiGene, GlaxoSmithKline and others are in the mix as well, making TIGIT one of the most competitive races in oncology. Several late-stage trials are underway.
The next key moment for TIGIT blockers could come when Arcus Biosciences discloses Phase 2 results for a study testing its drug domvanalimab in two- or three-drug combinations, each of which involve a checkpoint inhibitor, in lung cancer.
The result could also have implications for Gilead, which last year secured options to license several Arcus drugs, among them domvanalimab, in a broad alliance. But it also may provide insight into a scientific debate involving TIGIT blockers, as Arcus' drug is designed differently than several others in advanced testing.
Sanofi / GlaxoSmithKline vaccine against coronavirus
In the early days of the coronavirus pandemic, Sanofi and GlaxoSmithKline, two vaccine rivals, formed an unusual alliance to work together and develop a shot to prevent COVID-19. Their partnership seemed like as good a bet as any. Both have decades of experience making vaccines and were relying on a well-established method to make their shots. The drugmakers received $2 billion to fund their work, one of the U.S. government's largest investments in a vaccine developer.
Their efforts have yet to pan out. Sanofi and GSK scrapped their first vaccine candidate when it was found last December to be too weak to advance into late-stage testing. The setback contrasts with successes from Pfizer, Moderna, Johnson & Johnson and AstraZeneca, which each developed shots that are now cleared for use in various countries. Novavax, which uses a more traditional approach like Sanofi and GSK, recently announced positive study results, too.
Yet Sanofi and GSK could still play an important role. An upgraded vaccine proved potent enough in a Phase 2 test for the companies to move it into a 35,000-participant Phase 3 trial, which began in late May. The study will test two formulations of the shot, tailored to either the original form of the coronavirus or a particularly evasive variant known as Beta, and could produce results by the fourth quarter.
If successful, Sanofi and GSK's vaccine could help in areas where vaccines aren't widely available and as a booster for people who have gotten other shots.
Gene therapy for the treatment of Huntington's disease
Two of the most significant clinical setbacks of the year have come in Huntington's disease, a degenerative neurological condition with no effective treatments for its underlying cause. In two successive weeks in March, three closely watched Huntington's programs were all shelved — one, from partners Roche and Ionis, the other two from biotech Wave Life Sciences.
Roche's failure was particularly notable. The drug, known as tominersen, had been the first potentially disease-modifying Huntington's treatment to make it to late-stage testing. Earlier studies showed it could lower levels of a mutant protein closely intertwined with Huntington's. Yet the drug performed worse than a placebo, and it's still unclear why.
The failures raised questions about how to target the underlying biology of Huntington's, and raised the stakes for a gene therapy from UniQure known as AMT-130 to succeed.
Like other Huntington's treatments, such as Roche's, UniQure's therapy is meant to lower levels of a toxic protein, just in a different way. The idea is to stop mutated genes from producing the proteins in the first place.
Later this year, UniQure is set to release initial data from that program, specifically brain scan results and other biological tests from the first patients to receive treatment. Those results will be preliminary and won't prove whether AMT-130 can change the course of Huntington's. But they should show whether the treatment appears to be working as intended, which would be welcome after a difficult year.
Treatment of lung cancer with molecules targeted to the KRAS gene
Late last month, Amgen won FDA approval for the first drug that can target KRAS, the most frequently mutated gene in cancer. For decades, researchers had tried and failed to design medicines that can block proteins encoded by the gene, making clearance of Amgen's drug a scientific and medical milestone.
But the research breakthroughs that enabled Amgen have opened the door to others too, as a field of companies have assembled to quickly follow with their own versions. Mirati Therapeutics, a San Diego-based biotech, is one of the furthest along and later this year will present data that could support an application to the FDA for approval.
The results will be the first since Mirati revealed findings from an early study of the drug in advanced non-small cell lung cancer last fall. Should Mirati's drug match or outperform Amgen's, the biotech could see its market valuation rise even further than the roughly $8 billion it is currently worth.
In addition, Mirati is also expected to disclose proof-of-concept data from another group within its study who were given its drug together with Merck's Keytruda.
The latter data are particularly important as, even though Mirati's drug has appeared effective, a majority of patients didn't respond to treatment. Combinations are therefore viewed as critical for the long-term success of first-generation KRAS-blocking drugs.
An antimicrobial drug for patients with ulcerative colitis
After years of progress and several notable setbacks, medicines that change how our bodies interact with the bacteria, fungi and viruses that colonize them could arrive. Last August, Seres Therapeutics became the first company to detail successful results from the pivotal trial of a so-called microbiome drug, a treatment for a type of recurring bacterial infection. Others, such as Finch Therapeutics and Rebiotix, have reported positive findings for similar treatments. The field's first approval filing could come next year.
But microbiome therapeutics could be useful in treating more diseases, from inflammatory conditions to cancer, and that makes an upcoming study from Seres an important proof point for the field's progress. The biotech should soon report data from a Phase 2b study testing a microbiome drug called SER-287 in patients with mild-to-moderate ulcerative colitis, a form of inflammatory bowel disease affecting about 1 million Americans.
Though several drugs are available for ulcerative colitis, they don't always work and can cause side effects associated with tamping down the overactive inflammatory response that drives the disease.
Seres's drug, known as SER-287 and consisting of a group of gut bacteria packed into a pill, isn't immunosuppressive. That resulted in a safety profile in Phase 1 testing that was similar to placebo, according to data published in the journal Gastroenterology earlier this year. If found effective in mid-stage testing, the drug could have potential as a safer alternative or potential combination partner for existing drugs.
2021 05 12
Digitalising drug discovery
As data and digital technology become vital to every aspect of life sciences, the industry is increasingly looking beyond biologists, chemists, and doctors to drive its drug development – and finding that technology has a chief role to play in the future of medicine.
According to an article by Stephens, Zachary D., et al. on Big data: astronomical or genomical? by 2025 more than 500 million human genomes will be sequenced, creating more data than YouTube and Twitter combined.
Mining this data to advance drug discovery and new scientific breakthroughs relies on overcoming the overwhelming conundrum of extracting meaningful insights from massive data that is distributed, non-standardised, complex, and inaccessible to most.
Dr Maria Chatzou Dunford, a bioinformatician by background, recognised the fundamental role technology could play in accelerating drug discovery through overcoming these challenges, and as a result founded AI-bioinformatics technology company Lifebit in 2017.
“We found ourselves spending 90% of our time dealing with computational data hassles rather than focusing on the biology and the results,” Dunford says. “Gradually we realised this problem was becoming a norm for the entire industry, and that’s when we founded Lifebit.”
Dunford also believes the industry has just entered a new ‘Genomics 2.0’ era. Legacy technologies are built for an old genomics model – a world with very few, very small centralised genomic datasets that were not very diverse.
“Today, companies have exponentially more datasets, and genomic data by itself is no longer enough. They need clinical, phenotypic, and observational data to supplement genomic data to uncover next-level insights,” says Dunford.
“There’s added complexity in that all this data resides across multiple different sites – including research institutions, clinical settings, pharma companies and biotech companies.”
The ability to bring all this data together, she says, will completely change drug discovery and give companies an important competitive edge.
“If you look at the history of pharmaceuticals, initially it was all about chemistry, and it took pharma about 100 years to get that right, and it took another 50 years to start getting biology right.
“The next ‘big thing’ for the new generation of pharma to get right is its approach to data. The industry needs to shift towards operationalising personalised medicine, creating drugs that are more valuable and precise, and unlocking value-based pricing. But they don’t have 50 years – to stay competitive they need to innovate over the next five years, and investing in Genomics 2.0 technologies could be a game-changer in bringing new drugs to market in just a few years.”
Accelerating drug discovery
To digitise the drug discovery process, pharmaceutical companies need to better access and manage data, but the industry is far from where it needs to be.
“Companies should aim to get their drug discovery to a point where approximately 80% is digital and only 20% physical, with the latter part just being confirmation,” says Dunford. “Right now it’s the opposite – 80% is physical and observational, and sometimes anecdotal, even. That makes extrapolation difficult, and increases the chance of the trial failing.”
Access to population data speeds the process as the majority of experiments needed to develop some drugs have already been completed in the real world.
“Rather than starting with a random drug in the hope that it will treat a particular disease, you can flip drug discovery on its head and look at which patients are more prone to the disease, understand the genetics and protein-functions behind it, and then work backwards to find a chemical to treat it.”
Consequently, drug discovery timelines could be reduced to as little as one year. COVID-19 vaccines have demonstrated pharma R&D’s ability to move with speed, and Dunford sees no reason why similar timelines can’t be achieved for personalised medicines.
“If you have enough data from hospitals across the world, you essentially have pre-existing clinical trial data that you can analyse endlessly, as well as being able to call in those patients for more samples. It brings the clinical trial into the real world.”
Examples of this already exist. Genomics England, for instance, is currently analysing the genetic code of 35,000 patients with COVID-19 to help scientists understand whether a person’s genetics may influence their susceptibility to the virus.
“We wouldn’t need to lockdown an entire city or country if we knew more about the genomics of COVID-19,” says Dunford. “Instead, we’d only need a specific group of people to stay indoors.”
The industry could also start to take a disease-wide approach to drug development by selecting a disease, gathering all the related population and clinical data, bringing together the right tools and experts to analyse and assess potential treatments, and then manufacture the right pill.
2021 01 04
Top 10 Medical Innovations For 2021
An up-and-coming gene therapy for blood disorders. A new class of medications for cystic fibrosis. Increased access to telemedicine. These are some of the innovations that will enhance healing and change healthcare in the coming year, according to a distinguished panel of clinicians and researchers from Cleveland Clinic.
1. Gene Therapy for Hemoglobinopathies
Hemoglobinopathies are genetic disorders affecting the structure or production of the hemoglobin molecule – the red protein responsible for transporting oxygen in the blood.
The latest research in hemoglobinopathies has brought an experimental gene therapy, giving those who have the condition the potential ability to make functional hemoglobin molecules – reducing the presence of sickled blood cells or ineffective red blood cells in thalassemia to prevent associated complications.
2. Novel Drug for Primary-Progressive Multiple Sclerosis
In individuals with multiple sclerosis (MS), the immune system attacks the fatty protective myelin sheath that covers the nerve fibers – causing communication problems between the brain and the rest of the body that can result in permanent damage or deterioration and eventual death.
A new, FDA-approved therapeutic monoclonal antibody with a novel target is the first and only MS treatment for the primary-progressive population.
3. Smartphone-Connected Pacemaker Devices
Implantable devices like pacemakers and defibrillators deliver electrical impulses to the heart muscle chambers to contract and pump blood to the body. They are used to prevent or correct arrhythmias – heartbeats that are uneven, too slow or too fast.
Remote monitoring of these devices is an essential part of care. Traditionally, remote monitoring of this device takes place through a bed-side console that transmits the pacemaker or defibrillator data to the physician.
Though millions of patients have pacemakers and defibrillators, many lack a basic understanding of the device or how it functions and adherence to remote monitoring has been suboptimal.
Bluetooth-enabled pacemaker devices can remedy these issues of disconnection between patients and their cardiac treatment. Used in conjunction with a mobile app, these connected devices allow patients greater insight into the health data from the pacemakers and transmit the health information to their physicians.
4. New Medication for Cystic Fibrosis
Today, more than 30,000 people in the United States are living with cystic fibrosis (CF) – a hereditary condition characterized by thick, sticky mucus that clogs airways and traps germs, leading to infections, inflammation and other complications.
CF is caused by a defective cystic fibrosis transmembrane conductance regulator (CFTR) protein.
A class of drugs called CFTR modulators correct the protein’s action, but medications developed prior to last year had only been effective in a subset of people with certain mutations. A new combination drug, FDA approved in October 2019, provides relief for patients with the most common CF gene mutation (F508 del) – estimated to represent 90 percent of individuals living with the disease.
5. Universal Hepatitis C Treatment
Classified as a “silent epidemic” by the CDC, hepatitis C has emerged as a major public health issue in the U.S.
With no vaccine for the virus, patients have been limited to medication, but many treatments were accompanied by adverse side effects or only effective for certain genotypes of the disease.
A new, approved fixed-dose combination medication has vastly improved hepatitis C treatment. More than 90 percent effective for hepatitis C genotypes one through six, the therapy represents an effective option for a wider scope of patients.
6. Bubble CPAP for Increased Lung Function in Premature Babies
Underweight and frail, babies born prematurely often require specialized care – including ventilation for those with infant respiratory distress syndrome (IRDS). For IRDS, infants are commonly administered surfactant during mechanical ventilation, a practice that can cause lasting lung injury in preterm infants and contribute to the development of chronic lung disease.
Unlike mechanical ventilation, b-CPAP is a non-invasive ventilation strategy – delivering continuous positive airway pressure to newborns to maintain lung volumes during exhalation. The oscillating, rather than constant pressure, plays a role in its safety and efficacy, minimizing physical trauma and stimulating lung growth when administered over a prolonged period.
7. Increased Access to Telemedicine through Novel Practice and Policy Changes
COVID-19 saw increased adoption of telemedical practices as clinicians needed to conduct patient visits online. An increasingly virtual care model and increased consumer adoption came by way of fundamental shifts in policy at both the government and provider level.
Since March, state and federal regulators have moved quickly to reduce barriers to telehealth, understanding that these new tools can speed access to care while protecting healthcare workers and community members. These measures opened the floodgates for telehealth, allowing for new programs and the expansion of existing networks.
8. Vacuum-Induced Uterine Tamponade Device for Postpartum Hemorrhage
Characterized as excessive bleeding after having a baby, postpartum hemorrhage is a devastating complication of childbirth, affecting from one to five percent of women who give birth. Mothers experiencing postpartum hemorrhage may require blood transfusions, drugs which may cause dangerous side effects, long uncomfortable procedures, and even emergency hysterectomy with loss of fertility. Non-surgical interventions directed at the site of bleeding has been limited to balloon devices that expand the uterus while compressing the site of bleeding.
But the newest advancement is that of vacuum-induced uterine tamponade – a method that uses negative pressure created inside the uterus to collapse the bleeding cavity causing the muscle to close off the vessels. The vacuum-induced device represents another minimally invasive tool for clinicians as they treat the complication and provides a low-tech solution that is potentially translatable to developing countries with low resource availability.
9. PARP Inhibitors for Prostate Cancer
About one man in nine will be diagnosed with prostate cancer in his lifetime. While there has been progress in the last decade, the disease remains the second-leading cause of cancer death among men in the U.S. PARP inhibitors – pharmacological inhibitors for cancer treatment – block proteins called PARP that help repair damaged tumor DNA in people with BRCA1 and BRCA2 gene mutations.
Though known for their success in women’s cancers, two PARP inhibitors have been demonstrated to delay the progression of prostate cancer in men with refractory cancer and DNA repair pathway mutations. Both were approved for prostate cancer in May 2020.
10. Immunologics for Migraine Prophylaxis
Migraines affect more than 38 million people in the U.S. – an estimated 12 percent of the adult population. For some time, multi-purpose drugs like blood pressure medications, antidepressants, anti-seizure drugs and Botox injections have been used to prevent attacks. However, not developed specifically for migraines, these methods have been met with mixed results.
In 2018, new medications were developed to help head off migraine pain. The class of drugs works by blocking the activity of a molecule called calcitonin gene-related peptide (CGRP), which spikes during a migraine.
Actively prescribed in 2020, this new FDA-approved class of medication is the first to be specifically designed for the preventive treatment of migraine, marking a new era of migraine therapeutics.
2020 12 09
EC announces new Pharmaceutical Strategy for Europe
The strategy outlines actions to strengthen EU supply chains, encourage innovation and ensure medicines are affordable and sustainable.
European union flag (Blue with circle of yellow stars in center) with a white pill bottle spilling tablets over it - idea of EU medicine supply
The European Commission (EC) has today adopted a Pharmaceutical Strategy for Europe to ensure that patients have access to innovative and affordable medicines and to support the competitiveness, innovative capacity and sustainability of the EU’s pharmaceutical industry.
According to the agency, the strategy will allow Europe to cover its pharmaceutical needs, even in times of crisis, through robust supply chains and will be a key component of building a stronger European Health Union. One that is future-proof and crisis-resilient.
The strategy has four main objectives:
Ensuring all patients have access to affordable medicines and addressing unmet medical needs, such as antimicrobial resistance, cancer and rare diseases;
Supporting competition, innovation and sustainability within the EU’s pharmaceutical industry and, by connection, the development of high quality, safe, effective and more environmentally friendly medicines;
Addressing security of supply and enhancing crisis preparedness and responsemechanisms; and
Promoting a high level of quality, efficacy and safety standards.
The EC said that while the strategy is more than a crisis-response instrument, it draws lessons from the initial response to the COVID-19 pandemic to make Europe’s pharmaceutical sector better prepared and more resilient.
The plan also prevents concrete actions to ensure accessibility, availability and affordability of medicines, while also supporting diversified and secure supply chains and promoting environmentally sustainable pharmaceuticals.
The EC said the strategy will also support patient centred innovations and accommodate digital and technological change.
Stella Kyriakides, Commissioner for Health and Food Safety, commented: “Today we launch the work to ensure that safe and effective medicines are accessible and affordable at all times and to all patients across the EU. With our Pharmaceutical Strategy for Europe, we are delivering on our commitment to create a future-proof and patient-centred pharmaceutical environment in which the EU industry can innovate, flourish and continue to be a global leader. It is our long-term vision for open strategic autonomy, and our response to the challenges of today and the vulnerabilities exposed by COVID-19. Today we put in place another pillar of the European Health Union.”
The EC added that implementing this strategy over the next three years will include both legislative and non-legislative actions covering the whole ecosystem of pharmaceuticals and some aspects of medical devices.
Some of the key actions include:
A revision of the basic pharmaceutical legislation to make the framework future-proof and innovation friendly;
A revision of the regulations on medicines for children and rare diseases;
Using dialogue to identify vulnerabilities in the global supply chain of critical medicines and shape policy on strengthening the continuity and security of EU supply;
Cooperation between national authorities on pricing, payment and procurement policies, to improve the affordability and cost-effectiveness of medicines;
Creating a robust digital infrastructure, including a proposal for a European Health Data Space; and
Actions to promote innovative approaches to R&D and public procurement for antimicrobials (and their alternatives) as well as measures to restrict and optimise their use.
2020 10 27
Missed Opportunities – the Importance of Brand in Pharma Marketing
Even within the backdrop of science, with a dominance of logical, left-brain thinkers, still only 5% of the decisions we make are conscious – 95% are unconscious. This is because humans are not rational decision makers. Emotions, metaphors and memories are the tools our brains use to help us make sense and make decisions.
It’s that concept that the most powerful pharma marketing campaigns tap into. However, this idea is at odds with the well-worn process of pharma marketing, particularly when promoting a new drug.
First missed opportunity
Pharma launches all tend to follow a similar pattern. It takes years of scientific research, clinical trials and analysis of trial data before a drug is ready to be branded, typically this is several years before launch. Branding involves devising a name and packaging. At this stage, the motivation is usually compliance approvals.
Creative agencies aren’t necessarily brought into this process, in favour of skillsets like graphic designers to work on logos. Yet, I’d argue this is a critical phase – the right, experienced creative minds could help create a brand from the start which will engage emotionally with health care professionals and therefore be more likely to have an impact and remain memorable.
Bearing in mind the prevalence of biosimilars, drugs must now compete with others, and part of that competition is the way they’re communicated. Once the drug – including branding and packaging is approved, making changes becomes harder.
Second missed opportunity
The second opportunity comes after phase three trials, where a sales and marketing launch team are recruited for pre-launch and launch. They will be new to the drug and it’s their job to get it successfully launched.
There’s always an emphasis on scientists and medical staff, who are an important part of the team, but what’s often missing is the creative brains who understand the healthcare world but also understand creative communications, and how to tap into that all-important emotional part of decision making.
Experts in healthcare, not necessarily just pharma
Of course, any creative thinkers who are brought in have to be experienced in healthcare – they need the knowledge and expertise to understand the drug, the process, compliance rules etc. However, they don’t need to be just pharma or med ed specialists. Broader healthcare agencies with genuine expertise in branding and campaign work have a lot to offer. They could be device marketers who understand how to communicate in the space but have holistic communications expertise and draw on a mix of talents, for example.
Parallels with over the counter medicines
Over the counter medicine communication has needed to be differentiated for a while (think about Nurofen or Calpol being chosen for their brand attributes, rather than their contents). We’re now seeing parallels for communication to doctors – the more compelling a drug’s brand, the more likely it is to be chosen. Whereas trial data will always be central to the messaging, branding and campaign ideas are needed to better engage doctors. No longer is a detailed PowerPoint presentation enough – there needs to be more to help with the decision-making process.
Don’t be too literal
Strong imagery can help and often metaphors are used in communications that work within compliance. However, they shouldn’t be too literal – something being strong doesn’t have to involve weights and something progressing doesn’t have to involve a mountain climber. If a metaphor is eroded down it becomes too literal, and loses its power and differentiation. The truth is, rational is not engaging. A good, experienced agency can allow you to be more creative and impactful within restraints.
Launch and marketing teams are well stocked for science but less well stocked for art. Yet, it’s the fusion of arts and science that creates the impact these brands really need – actively bringing in the right marketing skills married with healthcare knowledge.
A good creative healthcare specialist will offer value at each stage. They bring experience but also diverse, richer thinking – and therefore will help a new pharma brand stand out.
2020 10 12
The Pandemic’s Impact On Pharmacy
The Covid-19 pandemic continues to have a major impact on the pharmaceutical space. The global health crisis has emphasized the role of pharmacists as part of the front-line care team and exacerbated long-standing issues like drug shortages, drug recalls and drug diversion. As pharmacists continue to step up, address these challenges and adjust to the “new normal,” it is important to recognize the tools and technology that can help.
The Evolving Role Of Pharmacists
The pandemic has altered the role of the pharmacist and further emphasized their position as an integral part of a patient’s care team. Gone are the days of pharmacists being perceived as just distributors of medication. As front-line medical workers and physicians remain busy and focused on combating Covid-19, pharmacists continue to take an active part in providing counsel, education and support to patients who have concerns about the virus.
In fact, the U.S. Department of Health and Human Services (HHS) recently permitted pharmacists to administer childhood vaccines to prevent the outbreak of preventable diseases. Pharmacies have also shifted operations to best support patients through means like at-home delivery, ensuring that every patient receives the medication and care they need. The pandemic has revealed the need for a holistic, all-encompassing approach to patient care — one that is not possible without the input and expertise of pharmacists. While their role has only expanded in scope amid the pandemic, pharmacists are still combatting surprisingly quotidian challenges like drug shortages, diversion and recalls at an increased rate.
The Impact On Common Challenges Faced By Pharma
The pharmaceutical industry faces a number of challenges that have been further compounded by the pandemic. For example, the global health crisis has worsened drug shortages, a long-standing obstacle for pharmacists. According to recent data from my company’s Annual Hospital Pharmacy Operations Report, about 60% of pharmacy staff reported dealing with up to 20 medications on shortage at a time, and nearly 30% with more than 20 shortages at a time. Additionally, the American Medical Association shared that “[r]oughly half of all drugs in shortage are injectables, including analgesics, sedatives and paralytics used for intubating critically ill patients.”
Drug recalls are another major issue that has only intensified. According to my company’s annual report above, 79% of surveyed medical professionals reported that each recall can require up to 10 staff hours to sort through, and 35% reported that they are experiencing more than 10 recalls annually. As the industry continues to focus on testing and learning about new treatment options for Covid-19, it can be difficult for organizations to keep up with drug recalls, potentially inadvertently putting dangerous medications in front of patients. Take metformin, for example.
The global health crisis is also shedding light on another major concern — mental health strain on frontline care workers. Medical professionals are under immense amounts of pressure between caring for patients with Covid-19, keeping track of vital medications and maintaining their own health. According to the National Institute on Drug Abuse, “Many clinicians and addiction medicine specialists suggest that stress is the number one cause of relapse to drug abuse.” This means that drug diversion, or theft of controlled substances by a medical professional — an extremely under-reported part of the opioid epidemic — is much more likely to occur during times of high-demand and stress, like the pandemic.
Adjusting To Healthcare’s New Normal
As medical professionals navigate a changed healthcare landscape and address issues further compounded by the pandemic, it’s important for them to understand and embrace the technology available to ease the transition.
For starters, keeping track of medication inventory throughout the supply chain is more important than ever, particularly to address drug recalls and shortages. RFID technology, combined with cloud-based solutions that monitor the chain of custody from manufacturer to patient, hold a lot of promise — particularly for pharmacists.
RFID is a reliable way to track medication from manufacturer through to patient use. It can help pharmacists keep track of drug shortages and recalls by providing reliable, item-level visibility, allowing pharmacists to keep track of drugs that are in danger of shortage or consider alternatives where appropriate. However, according to a press release from DoseID, a consortium of which my company is a part, “It is only with participation across the entire pharmaceutical supply chain — from drug manufacturers, to inlay providers, to automation vendors and hospitals — that RFID unit-level medication tracking can achieve the DoseID goals of interoperability, reliable tag performance and complete and accurate data.” We’re seeing this promise manifest through the formulation of organizations like DoseID, which was created to improve insight into the pharmacy supply chain, ultimately protecting patients and making the lives of pharmacists easier in the process.
AI solutions are also proving to be beneficial when it comes to researching and testing possible drug treatments for Covid-19. In an effort to prevent shortages, researchers have figured out how to use AI technology to create alternative preparations and routes for drugs that are now being used in clinical trials to treat Covid-19. Hopefully, this will make treatments more widely available.
Furthermore, AI solutions that can monitor the prescribing habits of medical professionals can help organizations keep a constant pulse on where medication — especially high-demand controlled substances like propofol and fentanyl — is throughout the hospital and flag any concerning or suspicious behavior, preventing issues like drug diversion. Additionally, item-level inventory management systems ensure proper sanitization occurs if a medication cart or tray enters a room with a Covid-positive patient. Some technology solutions can actually incorporate a sanitation check as part of the standard medication dispatch workflow, ensuring that it remains top of mind for busy medical staff.
There’s no denying that the pandemic has changed the pharmaceutical industry forever. It has expedited and highlighted the need for pharmacists as a part of the patient care team and continues to exacerbate existing issues. While there is still plenty of uncertainty, there is one thing we know for sure: configurable technology is critical to the future of pharmacy.
More: https://www.forbes.com
2020 10 12
The Evolution of Pharma Marketing
Integrating advanced technologies with the current approach to pharma marketing will work best in the evolving new world of patient advocacy.
FREMONT, CA: The availability of life-saving pharmaceutical products by their very nature plays a prominent role in the wellbeing of a society. In fact, the marketing cost in the pharmaceutical sector has increased by nearly 70 per cent in the past 20 years and now totals nearly 30 billion dollars. Only those businesses that embrace the power of digital innovation and a customer-first business model will succeed in building more effective interactions, deeper loyalty, and lasting brand preference. Hence it can be said that the marketing world of pharma will adapt going forward to focus on the user, not the drugs. Here is more to it.
With technological advancement, many existing methods and practices of pharma marketing have been replaced or modified in combination with technologies. Electronic detailing is one of the methods of drug promotion introduced a few years back as technologically developed. In the pharma industry, it has been introduced as a new communication channel for the promotion of drugs among physicians. For e-detailing digital technologies like the internet, video conferencing, and interactive voice response are adapted to communicate with physicians.
The fast-changing market demands more value from pharmaceutical manufacturers than ever before. In this case, value stands for more cost efficiency, proven patient outcomes, and better contribution to the health care experience throughout the value chain. The key will be emerging as a key player in the integrated health management model that combines pharmaceutical marketing into mobile communications, and other online platforms for marketing.
As the result-based approach to health care grows in popularity, now is the time for pharmaceutical marketing agencies to drive value from a new personalized, omnichannel approach to marketing. This means allowing different strategies for marketing directly to customers and in detailing to physicians and payers. Advanced pharma marketers are shifting into precision marketing, which is all about focusing on digital platforms with custom messaging for a segment of consumers who want the information most.
For pharmaceutical firms to continue thriving, they need to engage with patients, who are more focused on holistic health outcomes. This means expanding their products and its capabilities with a focus on continuous patient outcomes, using new methods to drive market-specific drugs, and using the appropriate channels to provide the right information.
2020 09 22
Pharma and the modern patient: creating a real connection
Over the past few years, our digital world has moulded a new kind of patient – engaged and empowered individuals who want to be actively involved in their health journey.
With access to different treatment paths and complementary approaches based on location, physician, insurance, funds and more, therapies are becoming more personalised than ever before. The market is evolving – the patient voice has been amplified, forcing pharma companies to develop new ways to listen and interact, creating new opportunities to understand and prioritise patient needs and preferences.
What Do Patients Really Want?
Today, the expert knowledge that once exclusively belonged to physicians is now easily accessible to patients, thanks to the internet. As a result, patients have become active consumers instead of passive participants in their medical care. Their expectations have risen in conjunction with their increased participation and they want to be personally engaged. Patients want to be treated as people, and in our digital age this is not always easy. Like any other business, healthcare providers and pharmaceutical companies can appear as faceless entities. Pharma can benefit greatly from bridging this gap and creating an approachable brand that patients feel they can engage with.
What Can Pharma Do?
To engage patients, pharma must create trust and opportunities for genuine, two-way interactions. Traditionally, pharma has only interfaced with healthcare professionals and patient advocacy groups about patients’ needs, but for various reasons have spent less effort communicating with individual patients directly about their experiences, needs and wants. The time has come for this to change!
Here are some ways this change can be achieved, keeping current regulations in mind:
* Always be there: Deliver value to patients all the time, even before they use your drug
* Take a holistic approach: Go beyond the medicine and try to understand the ecosystem they are navigating and their challenges. This may especially help when trying to optimise adherence
* Be patient-centric: Focus on the patients’ needs, personalise your value and timing, and focus on how to improve patient quality of life and quality of care
* Provide emotional support: Build a community around a medical condition/medication, encouraging patients to share their experiences with others going through the same
* Research and improve care: Listen, understand and evaluate real- world patient experiences to help shape future decision-making
* Use artificial intelligence (AI): The world is data driven. If you engage patients, you can use advanced tools to discover patterns that let you anticipate patient needs and create an even more personalised experience, providing information and support exactly when it is needed
A Strengthened Connection
Just as Amazon revolutionised the way we consume products, so too digital health is changing the way patients consume medical care. Patients are becoming digital consumers with the ability to manage their own treatments, and the results are significant. Over the next few years, patients will continue to play a crucial role in pharma decision-making – impacting millions of lives and billions of dollars. Making this shift is undoubtedly challenging as it requires shifting mindset and business models. However, the future is undoubtedly patient engagement. Companies who can successfully achieve the above points will have a far greater understanding of patient needs and market demands. Furthermore, they will help patients better appreciate the critical role of pharma in the healthcare continuum. Ultimately, this shift is a win-win for pharma and patients and will improve healthcare for everyone.
2020 07 28
Technologies Shaping The Future Of Pharma
Digital health is poised to bring its disruptive force across the healthcare landscape, and the pharmaceutical industry isn’t immune to the upcoming changes. Turning the point-of-care towards patients and empowering them with their health data, this cultural transformation brings about a radical shift in the traditional functioning of the drug industry.
1. Integrating A.I. in drug development slashes time and cost
From finding suitable candidates to animal trials, from unexpected side effects in clinical trials to multiple trial-and-error sequences, drug development is understandably a lengthy and costly process. In fact, estimates put the numbers at about 12 years and $2.9 billion for an experimental drug to advance from a lab to the market. However, developments in the field of artificial intelligence can now help diminish the cost and time traditionally associated with drug development.
To exemplify this possibility, Insilico Medicine, an A.I. pharma startup, identified a potential new drug in only 46 days. Its algorithm achieved this by analysing vast amounts of data which would otherwise take humans years to go through. The drug didn’t result in a commercial one as this achievement was to prove the potential of A.I. in drug development. However, practical examples do exist within the industry.
Partnering with IBM and the University of Toronto in 2015, A. I. startup Atomwise used its algorithm to identify two drugs with significant potential to reduce Ebola infectivity. It accomplished this effort in less than a day. In 2020, the company teamed with researchers to find broad-spectrum treatments for COVID-19 and future coronavirus strains.
Also on the COVID-19 pandemic, the BarabasiLab paired its network medicine toolset with A.I. to find potential treatments. The team managed to obtain a list of such drug candidates in less than 10 days. The latter are undergoing tests on human cell lines in experimental labs. These are only a few examples of drug companies employing A.I.’s benefits in drug development. In fact, there are more than 230 such startups and this number will only keep climbing.
2. With 3D-printing, pharma companies can make personalised medicine a reality
Despite being a relatively new technology adopted in healthcare, 3D-printed drugs show great promise. The first FDA approval of a 3D-printed pill was issued only in 2015, but the technology already paves the way for personalised medicine. This was, in fact, the conclusion of a 2019 research. The researchers involved successfully 3D-printed 6 different drugs into a single, multilayered polypill. With this technique, pills of specific dose tailored to individual patients are made possible.
Medical 3D-printing company FabRx is aggressively working towards this goal. This April, they released M3DIMAKER – the first pharmaceutical 3D printer to manufacture personalised medicines. It allows for the printing of medicines according to the user’s manufacturing needs, which in some cases means one-month’s medication (28 pills) in around 8 minutes!
Traditionally, pills were manufactured in standard doses and had to be manually adjusted, for example, by crushing or splitting them in order to fit the dose for children. This method potentially leads to dosage errors or even improper drug use. With M3DIMAKER, this issue is a thing of the past. The printer allows pharmacists to make pills with the precise dose for individual patients. “I truly believe that we are one step closer to personalised medicine thanks to the M3DIMAKER,” said Dr. Alvaro Goyanes, Director of Development at FabRx.
3. Boosting adherence of chronic care patients with digital pills
Yearly, lack of adherence to one’s medication amounts to 125,000 deaths and around $300 billion in healthcare costs; all of these are in fact preventable. However, adhering to treatment plans with multiple medications is especially challenging for patients suffering from chronic conditions such as schizophrenia or cardiovascular diseases. To remedy this pressing issue, digital pills can boost adherence while preventing deaths.
The FDA first approved of such a digital pill in 2017 produced by Otsuka Pharmaceutical and Proteus for chronic mental disorders. This pill contains an ingestible sensor which is a wearable patch sensor and an app track. This allows physicians to monitor a patient’s compliance to the treatment.
However, Proteus filed for bankruptcy in June 2020. Despite one company’s struggles, the technology it employed isn’t at fault. In fact, an independent study in 2019 showed the improved adherence to treatment by tuberculosis-infected patients using oral pills equipped with Proteus’ system. Their adherence rate was even comparable to in-person medication adherence programs. As such, the technology has the potential to boost adherence and can be further developed by other companies.
2020 07 13
Pharma sales to increasingly rely on virtual platforms due to COVID-19
Virtual tools are being increasingly utilised by pharma companies to replace in-person meetings following the outbreak of the COVID-19 pandemic.
According to researchers at GlobalData, virtual platforms will experience an extra boost after already having a transformational impact on the healthcare landscape, due to the impacts of the COVID-19 coronavirus.
Dr Valentina Gburcik, Senior Director of Cardiovascular and Metabolic Diseases, Gender Health and Digital at GlobalData, commented:
“The shift from in-person to digital is seen in advertising, medical conferences and sales rep meetings with physicians. Virtual health tools are already there enabling companies to have broader engagement with patients and physicians throughout various phases of the patient journey. Sales forces from pharma companies are now using this technology even more to interact virtually with physicians – particularly during the COVID-19 outbreak.”
The COVID-19 pandemic has seen cancellations of clinical events and conferences, as well as a surge of virtual meetings while all face-to-face contacts halt, highlight the researchers.
Gburcik added: “In-person meetings have their own advantages such as people being able to express themselves by using body language and facial expressions that can better convey a message and create a deeper bond with a customer.
The lack of a physical component in human interaction may lead to a weaker influence of reps over respective physicians and therefore reduced drug sales. Nevertheless, the ever-increasing internet speed, with 5G on our doorstep and evolving video conferencing software such as WebEx, Zoom and Skype for Business, will somewhat alleviate these problems.”
However, the researchers emphasise that it remains to be seen whether the current conditions will push humanity further into virtual space with no return to the circumstances seen before the COVID-19 pandemic.
Gburcik concluded: “The pharma sales forces and physicians may get used to the new reality and thus the use of the virtual meetings and tools may get boosted far beyond the pandemic.”
2020 01 02
Key trends studied for diabetes drug development in 2020
Functional links between metabolic, cardiovascular and renal diseases, as well as a focus on digital and a personalised approach will shape diabetes drug development in 2020, says new analysis.
Valentina Gburcik, PhD, Senior Director of Cardiovascular and Metabolic Disorders and researchers from GlobalData have looked at the key trends that will shape the metabolic disorders pharmaceutical space in 2020, focusing on diabetes.
Gburcik says that the diabetes market has long been a lucrative space for drug developers. According to a GlobalData study, 165 million people were diagnosed with type 2 diabetes globally in 2018, a number that is going to increase to 212 million by 2028.
“It comes as no surprise that many major pharmaceutical companies have been investing in this area,” Gburcik said, “leading to an enormous number of marketed branded therapies, as well as a rich pipeline, which is predominantly filled with ‘me-too’ therapies entering a very saturated market.”
According to the researchers, a growing body of scientific evidence is showing interconnectedness between metabolic disorders and cardiovascular and renal diseases and an increasing number of scientists and drug developers are now focusing on understanding the functional links between these diseases to jointly address cardio-renal-metabolic risks, which should eventually lead to better and more holistic treatments.
Gburcik further continued that digital transformation in healthcare, including the use of artificial intelligence (AI) and big data, will be a game-changer to diabetes prevention and management.
“These technologies have the potential to revolutionise the treatment of diabetes by employing continuous remote monitoring of patients’ symptoms, physiological data and environmental factors through the use of wearable tech, sensors and smartphone technologies,” Gburcik said.
Patient centricity and personalised medicine are another rising theme in healthcare, Gburcik said. “Oncology is currently the most advanced field in terms of personalised molecular diagnosis and treatments tailored based on genetics. However, diabetes research is experiencing rapid progress too,” Gburcik explained.
“Although the studies so far have not shed enough light on the genetic contribution to the phenotype of heterogeneous metabolic diseases such as diabetes, big data that is currently being accumulated through the use of digital technology will soon lead to better characterisation of clusters, which may define specific subtypes of the disease, leading to more tailored treatments and better outcomes.”
2019 12 31
Packaging security trends in the pharmaceutical industry
Drug safety is a huge concern for big pharma and tampering and counterfeiting in the market is dangerous for both consumers and brands. In this article, Prakash Shetty shares the latest innovations in packaging design and highlights how tamper proofing and developments in technology can protect all concerned.
THE $20 BILLION pharmaceutical packaging industry is the frontrunner in deploying technology for advanced security through packaging. The rise of e-commerce and efficient logistics have made sophisticated drugs accessible worldwide, irrespective of their manufacturing locations. However, this presents a challenge to protect products throughout their lifecycle and against counterfeiting. Pharmaceutical packaging innovations are important not only for the effectiveness of drugs, but also to ensure authenticity, traceability, product protection and patients’ comfort.
Product security
Pharmaceutical products require responsible packaging, especially for life-saving drugs, medical devices and nutraceuticals. The possible threats to these products include misuse and counterfeiting.
The World Health Organization (WHO) reports that about 10 percent of drugs are counterfeited globally. While counterfeiting is a result of human antisocial practices, tampering can occur due to inefficient handling or storage. However, technology and digitilisation can prevent counterfeiting, as well as tampering, to a great extent.
Pharmaceutical packaging – security through technology
Technology can help identify unauthorised access by making tampering evident and also creating a barrier to it. Tamper-proofing solutions ensure product safety during storage and transit. Tamper-resistant packaging relies on two principles: the requirement for multiple layers of protection and making tampering evident in case of unauthorised access or when the package integrity is compromised. Tamper proofing is challenging, as the package requires protection from external intervention. One strategy for tamper proofing that is popular in the pharmaceutical industry involves the following five layers of protection:
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Primary closure: This involves securing the lid of the container such that any adjustments made after dispatch are recognizable.
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Sealing: The process of making the primary closure permanent against transit pressure
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Isolating: Shrink wraps are used to safeguard the sealed container from moisture and other shipments
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Identification: Unique identification numbers prevent replication. It also makes the product identifiable in the supply chain.
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Secondary packaging: The outer shipping containers that are designed in accordance with the shippers’ guidelines.
Tamper evidence
Tampering can be made evident through overt security features. Overt technology involves authentication through visual inspection without any expert knowledge. Hologram, colour shift inks and tear tapes are scalable and cost-effective systems for tamper evidence. Consumers can refuse to accept the package or report it if they spot damage.
Holograms: Brands usually use hologram techniques on their logo to indicate authenticity. Wear and tear on the hologram indicates rough handling and/or inappropriate storage.
Colour shift inks: Colour shift inks may appear as two or more distinct colours when shifted to different angles and give an illusional 3D effect.
Tear tapes: These are single-use polypropylene tapes. Consumers can identify tampering if the package lacks sealing tape or the tape is broken. Tear tapes are impossible to remove without destroying, hence making unauthorised access evident.
Preventing counterfeiting
Tamper-proofing technology combined with covert security features can help in preventing counterfeiting. Covert technology includes infrared (IR) and ultraviolet (UV) pigments, microtext and microscopic tagging, which are invisible through naked eyes. These are difficult to detect and replicate without specialist detection equipment. Security graphics and digital watermarks are the other techniques widely used in pharmaceutical packaging to confront counterfeiting.
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Security graphics: Design elements like line modulation and line embossing can produce a fine line colour printing similar to currency printing. Furthermore, microtext and latent images can make replication detectable.
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Digital watermarks: Digital watermarking or encoding involves invisible markings within graphics. Special software and readers are available to verify these markers.
Security through digitilisation
Digitilisation refers to the process of converting information into computer-readable format, which is easy to access irrespective of geographical location. The pharmaceutical packaging industry is deploying digital technology to combat counterfeiting and introduce efficient supply chain tracking.
Anti-counterfeiting apps
By simply scanning the medicine, anti-counterfeiting apps can tell the consumer whether the drug is fake or authentic. For example, the Drugsafe app, winner of the Big Data award at the 2018 Microsoft Imagine Cup, uses optical character recognition (OCR) to check the medicine’s authenticity in accordance with the Azure Cosmos database.
Digital mass serialisation
Counterfeiting in the pharmaceutical market is dangerous for consumers and brands. While consumers may become victims of poor medicines, brands can lose their credibility. Digital packaging solutions are a saviour, especially in supply chain management. Digital mass serialisation is one of the most common and scalable techniques employed; involving generation of a sequence of pseudo code that enters the consumers’ database for verification at later stages.
Security through product design
Ensuring safety for consumers is another important aspect of pharmaceutical packaging. Sophisticated drugs need careful handling and include age restrictions. Patients can become the victim of taking expired drugs, or children might access medicines that are not meant for them. The following packaging solutions are solving these consumer-centric challenges through design:
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Expiry date validation – “A picture speaks a thousand words and a QR code encodes a maximum of 4,296 alphanumeric characters.” A QR code is capable of coding the dose regimen for medicine along with its manufacturing and expiry date. Post consumption, the consumer can rescan the pack, enabling the app to update the regimen and send notifications accordingly.
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Childproofing and senior-friendly packaging – Uflex introduced an innovative packaging known as Child Resistant and Senior Friendly (CRSF) foils. These are available in two variants: push-through and peel-push, based on their operating mechanism. While it requires some amount of pressure for a child to open it, the procedure is easy for a senior citizen. This prevents unintentional handling damage to conventional blister foils.
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Timely drug dosage – Taking the right medicine in the right dosage is essential. Pill Pack, an Amazon company, took the initiative of coordinating with the patients’ drugs requirement and automatically shipping them refills as required.
Closing note
Security in pharmaceutical packaging is essential for both consumers’ safety and maintaining a brand’s integrity. Pharma is a sophisticated sector and its investment in secure packaging is essential. With innovations, technology and consumer acceptance, the serious problems of counterfeiting and tampering can be conquered.
More: https://www.europeanpharmaceuticalreview.com
2019 05 31
Algae could prevent limb amputation
A new algae-based treatment could reduce the need for amputation in people with critical limb ischaemia, according to new research funded by the British Heart Foundation, published today in the journal npj Regenerative Medicine. Researchers at St Thomas' Hospital and King's College London have made small capsules from brown algae which hold macrophages, a type of white blood cell. Tests in mice have shown that these algae capsules may be able to increase blood flow in the limbs where tissue has been damaged. The researchers now hope to progress this research into human clinical trials to help the people visiting hospital with critical limb ischaemia (CLI). It is estimated that there are up to 60,000 new cases of CLI per year in the UK.
Scientists have been experimenting with cells as a treatment to grow arteries in the leg for years, however, these treatments have not been effective in humans. A big challenge is that many of the cells injected into the injured area die, move away to surrounding areas, or are detected as 'foreign' by the immune system and rejected.
In this study, surgeon Professor Bijan Modarai and his team of scientists delivered the new algae-based capsules containing macrophages to areas of injured muscle tissue in the back legs of mice. Alginate from the cell walls of brown algae, which is mainly found in cold waters in the Northern Hemisphere, was used to form the capsules. They found that these macrophages successfully remained in the injured area, new blood vessels formed, and as a result more blood reached the damaged area.
CLI is a serious condition which occurs when the arteries in the limbs become blocked as a result of a build-up of fatty deposits, reducing blood flow to the hands and feet. Smoking, diabetes, obesity and high blood pressure can all lead to CLI. If blood flow is not restored, up to 50 per cent of people with CLI will either die or need amputation within one year.
Currently, to treat CLI and restore blood flow in the limbs, the blocked section of the artery has to be either bypassed during surgery or widened using a small piece of expandable mesh called a stent. However, in up to a third of patients, these methods will eventually fail or are not possible to begin with and amputation is the only option.
Professor Bijan Modarai therefore hopes that this new way of delivering cells could be the key to creating an effective treatment for people suffering with CLI.
Professor Bijan Modarai, Professor of Vascular Surgery and BHF Senior Fellow at King's College London/St Thomas' Hospital, said:
"We hope that this new method of cell therapy will greatly reduce the need for limb amputations in those people whose CLI is untreatable, and would otherwise have no other option.
"The beauty of this new algae-based treatment is that it harnesses the potential of natural materials. Not only does this make it a very attractive solution, but we know we can use it to safely treat people with CLI."
Professor Metin Avkiran, Associate Medical Director at the British Heart Foundation which funded the research said:
"You might associate algae with your garden pond, but our researchers are proving these diverse organisms could hold the key to a new treatment for one of the leading causes of limb amputation - CLI.
"Losing a limb is an all too devastating reality for many patients with CLI. The condition is caused by blockages in arteries that supply blood to the legs and feet, and is triggered by the same process that blocks coronary arteries when someone has a heart attack.
"This research brings us a step closer to finding treatments for the thousands of people affected by this disabling condition in the UK."
More: http://www.worldpharmanews.com
2019 02 15
New pill can deliver insulin
An MIT-led research team has developed a drug capsule that could be used to deliver oral doses of insulin, potentially replacing the injections that people with type 2 diabetes have to give themselves every day. About the size of a blueberry, the capsule contains a small needle made of compressed insulin, which is injected after the capsule reaches the stomach. In tests in animals, the researchers showed that they could deliver enough insulin to lower blood sugar to levels comparable to those produced by injections given through skin. They also demonstrated that the device can be adapted to deliver other protein drugs.
"We are really hopeful that this new type of capsule could someday help diabetic patients and perhaps anyone who requires therapies that can now only be given by injection or infusion," says Robert Langer, the David H. Koch Institute Professor, a member of MIT's Koch Institute for Integrative Cancer Research, and one of the senior authors of the study.
Giovanni Traverso, an assistant professor at Brigham and Women's Hospital, Harvard Medical School, and a visiting scientist in MIT's Department of Mechanical Engineering, where he is starting as a faculty member in 2019, is also a senior author of the study. The first author of the paper, which appears in the February 8 issue of Science, is MIT graduate student Alex Abramson. The research team also includes scientists from the pharmaceutical company Novo Nordisk.
Self-orientation
Several years ago, Traverso, Langer, and their colleagues developed a pill coated with many tiny needles that could be used to inject drugs into the lining of the stomach or the small intestine. For the new capsule, the researchers changed the design to have just one needle, allowing them to avoid injecting drugs into the interior of the stomach, where they would be broken down by stomach acids before having any effect.
The tip of the needle is made of nearly 100 percent compressed, freeze-dried insulin, using the same process used to form tablets of medicine. The shaft of the needle, which does not enter the stomach wall, is made from another biodegradable material.
Within the capsule, the needle is attached to a compressed spring that is held in place by a disk made of sugar. When the capsule is swallowed, water in the stomach dissolves the sugar disk, releasing the spring and injecting the needle into the stomach wall.
The stomach wall has no pain receptors, so the researchers believe that patients would not be able to feel the injection. To ensure that the drug is injected into the stomach wall, the researchers designed their system so that no matter how the capsule lands in the stomach, it can orient itself so the needle is in contact with the lining of the stomach.
"As soon as you take it, you want the system to self-right so that you can ensure contact with the tissue," Traverso says.
The researchers drew their inspiration for the self-orientation feature from a tortoise known as the leopard tortoise. This tortoise, which is found in Africa, has a shell with a high, steep dome, allowing it to right itself if it rolls onto its back. The researchers used computer modeling to come up with a variant of this shape for their capsule, which allows it to reorient itself even in the dynamic environment of the stomach.
"What's important is that we have the needle in contact with the tissue when it is injected," Abramson says. "Also, if a person were to move around or the stomach were to growl, the device would not move from its preferred orientation."
Once the tip of the needle is injected into the stomach wall, the insulin dissolves at a rate that can be controlled by the researchers as the capsule is prepared. In this study, it took about an hour for all of the insulin to be fully released into the bloodstream.
Easier for patients
In tests in pigs, the researchers showed that they could successfully deliver up to 300 micrograms of insulin. More recently, they have been able to increase the dose to 5 milligrams, which is comparable to the amount that a patient with type 2 diabetes would need to inject.
After the capsule releases its contents, it can pass harmlessly through the digestive system. The researchers found no adverse effects from the capsule, which is made from biodegradable polymer and stainless steel components.
The MIT team is now continuing to work with Novo Nordisk to further develop the technology and optimize the manufacturing process for the capsules. They believe this type of drug delivery could be useful for any protein drug that normally has to be injected, such as immunosuppressants used to treat rheumatoid arthritis or inflammatory bowel disease. It may also work for nucleic acids such as DNA and RNA.
"Our motivation is to make it easier for patients to take medication, particularly medications that require an injection," Traverso says. "The classic one is insulin, but there are many others."
More: http://www.worldpharmanews.com
2018 03 28
Medical expansion has improved health - with one exception
While Americans debate the rising cost of health care, a new study of 30 countries over 27 years found that medical expansion has improved overall health - with one major exception. Researchers found that increased spending on health care and increases in specialized care were both associated with longer life expectancy and less mortality in the countries studied. But pharmaceutical industry expansion was linked to negative health effects.
"This study isn't the first to suggest prescription drugs can pose a health risk. But it is the first to find that the growth of the pharmaceutical industry itself may be associated with worse rather than better health," said Hui Zheng, lead author of the study and associate professor of sociology at The Ohio State University.
"The findings were surprising to us."
Zheng conducted the research with Linda George, professor of sociology at Duke University. Their study is published in the March 2018 issue of the Journal of Health and Social Behavior.
The medical industry has undergone a massive expansion all over the Western world since the mid-20th century, Zheng said. In the United States, health care expenditures as a percentage of the gross domestic product increased from 5.1 percent in 1960 to 17.1 percent in 2014.
But it has been unclear whether this expansion has benefited overall public health.
To help answer this question, the researchers used data from 30 countries, including the United States, that are members of the Organisation for Economic Co-operation and Development. This includes most of the world's affluent democracies.
Zheng and George looked at how different types of medical expansion were related to population health between 1981 and 2007. Specifically, they linked expansion to life expectancy at birth; men's and women's life expectancy at age 65; and all-cause mortality rate.
They controlled for a variety of social, economic and demographic variables that also might account for relationships between medical expansion and health.
Medical investment, pharmaceutical expansion and medical specialization increased over time for virtually all 30 countries, although the amount of increase varied substantially, Zheng said.
The United States had the largest increase in medical investment and a steep increase in pharmaceutical expansion, but one of the flattest trajectories over the 29 years in the increase of medical specialization.
All three types of medical expansion were associated with two or more indicators of population health, results showed.
Increased medical investment and increased medical specialization were related to growth in all three life-expectancy measures and a decrease in overall mortality.
Two measures of expansion in the pharmaceutical industry - increased sales and more money spent on research and development - were linked to lower life expectancy among women aged 65 and older, and with increased mortality rates. The pharmaceutical measures were not associated with the other health outcomes studied.
The researchers ran tests to confirm that it wasn't the other way around - that lower life expectancy and increased mortality were causing an expansion of the pharmaceutical industry. But that wasn't the case.
That wasn't the only negative finding about the growing drug industry.
"We found that as the pharmaceutical industry expands, there is a decrease in the beneficial impact of medical specialization on population health," Zheng said.
This study can't say why expansion in the pharmaceutical industry is leading to negative population health effects, Zheng said.
"It could be due to toxic side effects of drugs, doctors' prescribing practices, patients' misuse of prescription drugs, reasons related to pharmaceutical industry's marketing strategies or some combination of these factors," he said.
He said they plan on studying this issue further.
More: http://www.worldpharmanews.com
2017 08 24
A startling new cost estimate for new medicines is met with skepticism
In the pharmaceuticals business there are few issues more loaded than the cost of developing a new drug. For a number of years estimates from industry groups on either side of the Atlantic have put it at $1.2 billion-1.8 billion. A new study by the Centre for the Study of Drug Development at Tufts University in Massachusetts reckons the average cost for drugs developed between 1995 and 2007 was $2.6 billion. Among those rejecting this new figure as highly misleading are Médecins Sans Frontières, a charity, and the Union for Affordable Cancer Treatment, a patients’ group.
The main point of controversy over such estimates is that they roll in the costs of those drugs that failed to win approval and, for good measure, the cost of capital required for the R&D. Tufts’s estimate includes $1.2 billion for the return on capital forgone while a drug is in development, on the assumption it would have otherwise earned a generous 10.5% a year. The remaining $1.4 billion is the average R&D cost of a random selection of drugs, multiplied by risk factors that account for the chances of failure at each stage.
Successful drugs cost far less than even the lower, $1.4 billion figure. But the road to approvals is littered with casualties such as the $800m that Pfizer blew on torcetrapib, a potential treatment for high cholesterol, before giving up in 2006. Jeff Williams, the boss of Clinipace, a contract-research organisation, has said that the small to medium-sized drugs firms his company works for manage to get their candidate drugs through development for less than $500m.
Another criticism of Tufts’s work is that it is based on secret data provided by a self-selected group of drug companies. The Tufts study group gets much of its funding from the industry; it says the group’s members are independent academics. Another criticism is that although such estimates embrace all the risks of developing drugs, they say little of the rewards.
The industry inevitably quotes such figures whenever it is suffering criticism for the high price of patented drugs. James Love, the head of Knowledge Ecology International, a group that studies and comments on issues of social justice, says drugs giants have used these big estimates of average costs to try to talk developing countries like India out of breaking the patents on specific medicines that, in practice, cost a lot less to develop.
Joseph DiMasi, director of economic analysis at the Tufts centre, says the most useful aspect of the $2.6 billion figure in his study is that it is comparable with previous figures. In 2003 his centre put the cost of drug development at $802m. This implies that in real terms costs have risen by 145%. Mr DiMasi says the increase has been caused by larger and more complex trials, a greater focus on chronic and degenerative diseases, and higher failure rates.
However, the life-saving cancer drugs that feature in many of the most emotional disputes over pricing are far from typical. Regulators often pass them after far smaller clinical trials than for other, less urgent medicines, thereby greatly reducing the most costly element of their development. Such drugs are also likely to qualify for “orphan drug” tax credits—an issue the Tufts study does not consider. It may be that the average is being inflated by other types of new drug, such as psychotropics, which may be only a bit more effective than existing ones but require big, expensive trials to gain approval.
More: http://www.economist.com
2017 08 09
Drug costs vary by more than 600% in study of 10 high-income countries
In a study of 10 high-income countries with universal health care, costs for prescription drugs in 6 of the largest categories of primary care medicines varied by more than 600%, according to research published in CMAJ (Canadian Medical Association Journal). All countries except Canada offered universal coverage of outpatient prescription drugs.
The study looked at data on the volume and daily cost of primary care prescriptions in 10 high-income countries with universal health care: Australia, Canada, France, Germany, the Netherlands, New Zealand, Norway, Sweden, Switzerland and the United Kingdom. Because of the high cost of pharmaceutical drugs and the lack of universal health care, the United States was not included.
Researchers focused on 6 categories of widely used primary care drugs usually purchased at retail pharmacies rather than hospital pharmacies. These included hypertension treatments, pain medications (nonsteroidal anti-inflammatory drugs as well as opioids), cholesterol-lowering drugs, noninsulin diabetes treatments, gastrointestinal medications and antidepressants. They measured frequency of use of the medications by average number of days of therapy purchased per capita.
Medications for treating high blood pressure accounted for the largest number of days of therapy in all countries.
In the 5 countries with universal, single-payer coverage of prescription medications, the average per-person cost was $77. Average costs were $99 in the 4 countries with universal social insurance for prescription drugs and $158 in Canada, which has a mixed system of private and public financing. Higher costs of drugs and the mix of therapies chosen accounted for most of the cost differences between countries.
"The volume of therapy purchased in Canada was about the same as that in the comparator countries; however, Canadians spent an estimated $2.3 billion more than they would have in 2015 if these primary care treatments had had the same average cost per day in Canada as in the 9 comparator countries combined," writes Dr. Steven Morgan, School of Population and Public Health, University of British Columbia, with coauthors.
"Average expenditures are lower among single-payer financing systems, which appear to promote lower prices and selection of lower-cost treatment options within therapeutic categories," the study authors conclude.
In a related commentary, Dr. Joel Lexchin, York University, Toronto, Ontario, writes "Canada is not doing well when it comes to ensuring that its population has access to prescription medications; we can and must get to a better place."
He says that we need universal pharmacare to reduce drug prices so that Canadians are not deterred from taking their medications.
More: http://www.worldpharmanews.com