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 2021 m. I quarter is already generated with the forecast for 2022.
2021 07 28
New LT data
Data for period July 19-25, 2021 is uploaded to PharmaZOOM LT.
2021 07 12
New LV data
Data for June 2021 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
Every month together with regularly updated wholesalers sales, we are importing VVKT sales of packages which sales we did not have before.
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
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.
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.
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:
Primary closure: This involves securing the lid of the container such that any adjustments made after dispatch are recognizable.
Sealing: The process of making the primary closure permanent against transit pressure
Isolating: Shrink wraps are used to safeguard the sealed container from moisture and other shipments
Identification: Unique identification numbers prevent replication. It also makes the product identifiable in the supply chain.
Secondary packaging: The outer shipping containers that are designed in accordance with the shippers’ guidelines.
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.
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.
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.
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.
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:
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.
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.
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.
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.
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."
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.
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."
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.
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.
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.