Negative Makena Confirmatory Data Didn’t Affect Reimbursement
Negative confirmatory data for the preterm birth drug Makena did not affect payers’ decisions to reimburse the drug, according to an analysis published in Health Affairs Forefront. The drug was granted accelerated approval by the US Food and Drug Administration (FDA) back in 2011 based on a small sample size. It was then recommended to be pulled from the market last October by the FDA Center for Drug Evaluation and Research advisory committee based on larger pools of post-market data showing no reduction in pre-term births. The agency finalized Makena’s approval withdrawal in early April this year.
Potential Partnerships Between Retailers and Real-World Data Firms Could Advance Clinical Trials
Major retail chains like CVS, Walgreens, and Kroger are increasingly entering the clinical trials space, effectively creating a swath of new recruitment sites for clinical research partners. According to COTA CEO and president Miruna Sasu, PhD, there is fertile ground for partnerships between retailers and real-world data (RWD) companies which, if capitalized upon, could accelerate clinical research.
ICER Issues Final Report on Alzheimer’s Drug Leqembi
Following a session of public comments, the Institute for Clinical and Economic Review (ICER) has issued its final evidence report on Eisai’s Alzheimer’s drug Leqembi (lecanemab). The independent organization valued the treatment between $8,900 - $21,500 per year. This is notably lower than the current list price of $26,500 per year set by the manufacturer.
FDA Gives Abbvie’s Qulipta the Nod for Chronic Migraine Prevention
The US Food and Drug Administration (FDA) approved the use of Abbvie’s Quilipta as a preventive treatment for chronic migraines. This approval gives AbbVie a leg up on Pfizer, whose migraine drug Nurtec ODT is not officially approved for chronic migraines. The decision was made based on data from a phase 3 trial that found the drug significantly reduced migraine day for patients.
Patient Recruitment Dodges Older Adults to Their Detriment
Despite representing the largest market for new and existing medications, a recent study by the UK-based International Longevity Centre (ILC) finds that adults over 60 are starkly underrepresented or outright excluded from clinical trials. Costs are commonly credited for this disparity, which imperils health outcomes for older adults.
Embracing Patient-Centered Clinical Trials to Facilitate More Representative Recruitment
Despite recent efforts, patient recruitment for clinical trials is expensive, difficult, and time-consuming. Moreover, the vast majority of clinical trials are far from representative of real patient populations, often severely lacking in people from marginalized ethnic and racial backgrounds. In a new sponsored Biopharma Dive article, learn how a patient-centered approach may facilitate cheaper and more representative recruitment.
US FDA Shoots Down Eli Lilly’s Application for Ulcerative Colitis Drug
The US Food and Drug Administration (FDA) has declined to approve Eli Lilly’s ulcerative colitis drug mirikuzimab. The agency credits manufacturing issues for the decision, noting that the drugs efficacy and safety are not in question. A Lilly spokesperson said that they are working closely with the agency to address these problems and bring the drug to market.
FDA Releases Guidance on Patient-Centered Drug Development
A final draft guidance regarding patient-focused drug development (PFDD) has been issued by the US Food and Drug Administration (FDA). The guidance covers data collection methods about patient care experiences and how they can be used in regulatory decision-making processes, specifically clinical outcomes assessments (COA).
NIH Announces Winners for RADx Tech Maternal Health Challenge
The US National Institutes of Health (NIH) announced the next round of winners selected to move forward in the Rapid Acceleration of Diagnostics Technology (RADx Tech) for Maternal Health Challenge, a contest to develop and assess digital health solutions to reduce post-partum maternal mortality. The winners will be awarded $75,000 for their efforts and entered into the next round of competition.
NIA Announces Real-World Data Project to Advance Alzheimer’s Disease Research
Decades of research has generated vast amounts of real-world data (RWD) that can be leveraged to advance research into Alzheimer’s disease. The US National Institute on Aging (NIA) has announced a new effort to create the Real-World Data Platform (RWDP) to consolidate these disparate data sources, which cover the majority of people living with Alzheimer’s and other dementias.
Most Accelerated Approval Confirmatory Studies are Turned in Late
Accelerated approval by the US Food and Drug Administration (FDA) requires drug and device manufacturers to conduct confirmatory studies to support full approval. A newly published paper in JAMA Health Forum found that over half of these studies are turned in late to the agency. These delays were more common in trials for small molecule drugs and non-oncologic therapeutics
Reducing the Financial Impact of Tuberculosis in India
Despite concerted efforts like the END TB Strategy, India faces the highest disease and economic burden for tuberculosis. A newly published study in PLOS Global Public Health, explores the financial burden and consequences for patients with TB in the nation. Regardless of the free diagnostic and treatment provided by the state, patients spent significant funds on care.
Darwin EU Seeks New Partnerships After Successful First Year
The European Medicines Agency (EMA) has issued a call for applications for new partnerships with Darwin EU. The move comes after a successful first year that saw the implementation of four real-world data (RWD) projects that investigated rare blood cancers, the use of certain medications tied to pregnancy issues, antibiotic prescription patterns, and severe asthma.
Study Finds Most Employers Provide Multiple Options for Health Insurance Plans
Results from Alight’s 2023 Benefits and Enrollment Trends report show that the vast majority of employers who provide health insurance give employees multiple options for plans. Over 90% offered more than one plan and three-quarters put money into employee health savings accounts for those with high-deductible plans.
Real-World Evidence Explores Inpatient and Outpatient Delivery of CAR T-Cell Therapy
Real-world evidence (RWE) presented at the 2023 Transplantation and Cellular Therapy Meeting compared and contrasted outcomes for patients receiving inpatient vs. outpatient CAR T-cell therapy for mantle cell lymphoma (MCL) and follicular lymphoma (FL). Patients with FL fared better with outpatient treatment, but those fighting MCL had slightly more adverse events (AEs) with outpatient infusions.
Improving Clinical Trials for Rare Disease Therapeutics
With millions worldwide living with rare diseases that have few or no current therapeutic options, clinical trials are essential in treating the conditions faced by this diverse patient population. However, running these trials are expensive, hard to recruit for, and often require novel trial formats to account for small participant pools. Regardless, hope is on the horizon for patients.
Housing Instability Worsens Health Outcomes
Astronomical rent and home prices are driving the ever-worsening affordable housing crisis. A newly published article in JAMA Network Open explores the link between housing instability and health outcomes, finding that displacement and unsafe living situations related to unaffordable housing are tied to worse mental and physical wellbeing.
FDA Allows Dose Increase in Dravet Syndrome Clinical Trial
The US Food and Drug Administration (FDA) has released a hold blocking a Phase I/IIa clinical trial testing an increased dose of STK-001, Stoke Therapeutics’ drug candidate for Dravet syndrome. Dravet syndrome is a rare form of severe childhood epilepsy that comes with a short life expectancy and developmental delays.
Research Details Health Disparities in Childhood Asthma and Eczema Based on Race and Ethnicity
Health disparities in asthma and eczema have been observed in Black children, but little research has explored the combination of the two. In research presented at this year’s American Academy of Allergy, Asthma, and Immunology (AAAAI) meeting, researchers explored the social determinants of health (SDoH) underlying this heath disparity. In addition, another abstract investigated the increased rate of childhood asthma-related respiratory infections in Black and Mexican-American patients.
Merck Announces Foundry DevTools Data Library is Now Free on Github
Merck KGaA has announced that its Foundry DevTools library, created with Palantir Technologies, will be freely available to users on Github, an open-source repository of code and software development resources. Merck and other pharma giants have used the platform for drug development and real-world evidence (RWE) analysis. This move marks a first for the platform.
Transparency, Oversight, and Opt-in Consent Makes Patients More Open to Sharing Data
A newly published paper in JAMA Network Open found that patients are more likely to participate in patient data sharing programs if certain conditions are met. Survey data from 3,500 hundred patients showed that patients valued transparency, opt-in informed consent, oversight mechanisms, and privacy.
Using Real-world Data to Take Better Baseline Patient Measurements
After recruitment and on-boarding, patients in clinical trials undergo a battery of baseline measurements. However, measurements can vary significantly from moment to moment and “normal” ranges are often wide. Therefore, an approach that uses multiple measurements could help reduce this uncertainty and improve clinical trials.
Improving Health Equity Through More Representative Clinical Trials
Patients from marginalized or rural populations often have less access to clinical trials or distrust based on a long-standing history of medical exploitation. However, this disparity in patient populations is detrimental to health equity efforts, biasing research by skewing heavily toward primarily white, upper-middle class, urban participant populations.
Sanofi Launches “This is Rare” Campaign on Rare Disease Day
Sanofi is marking this year’s Rare Disease Day, February 28th, with its new “This is Rare” campaign, which will feature the voices of patients living with rare diseases. The campaign is part of a large push by the company and others to develop therapeutics for the rare disease community, which accounts for one in seventeen people in the UK.
Value in Health Review: Applying Machine Learning to Wearable Data to Generate HEOR Insights
An article in the latest issue of Value in Health breaks down how machine learning can be applied to data from wearable devices to advance health economics and outcomes research (HEOR). Wearables are a font of real-world data (RWD) that can be analyzed by machine learning (ML). By addressing existing issues of data quality and effective usage, such a trove of information could accelerate research and decision-making process for payers, regulatory bodies, and pharma companies.
