October 2024 | Drug price negotiation abroad, biosimilar approval pathways, PTAB reform, and more.
Program on Regulation, Therapeutics, and Law (PORTAL)
Part of the Division of Pharmacoepidemiology at Harvard Medical School and Brigham & Women's Hospital
New from PORTAL
Click on article titles to view the full text. Please contact Matt Martin (mmartin55@bwh.harvard.edu) for access to specific publications.
Original Analysis
Cai CL, Kesselheim AS, Rome BN | JAMA Internal Medicine
Cai et al. assess the potential patient impact of the Center for Medicare & Medicaid Innovation’s (CMMI) High-Value Drug List Model, a voluntary model under which Medicare Part D plans cap patient out-of-pocket costs for 150 generic drugs at $2 per month. They found that 4 in 10 Medicare beneficiaries would experience modest savings under this model, with an annual savings of $11 per person.
Syversen ID, Schulman K, Kesselheim AS, Feldman WB | Milbank Quarterly
Syversen and colleagues compare the processes that health systems in seven other countries (Belgium, Canada, France, Germany, Netherlands, Norway, UK) and the US Department of Veterans Affairs use to negotiate brand-name prescription drug prices. Health systems differ across several axes, including how added health benefit is assessed, whether explicit willingness-to-pay thresholds are employed, and how specific approaches for priority disease areas are taken, but they largely coalesce around a set of practices that are absent from Medicare price negotiation.
Knox RP, Desai V, Sarpatwari A | Journal of Law and the Biosciences
In this analysis, Knox and colleagues compare the regulatory approval process for biosimilars in five jurisdictions (Australia, Canada, EU, UK, and US), noting differences in biosimilar testing requirements and policies regarding indication extrapolation, exclusivities, and biosimilar substitution. Understanding the impact of these policies on biosimilar approval and uptake is important to strengthen the global biosimilar market.
Liu ITT, Kesselheim AS | Pediatrics
Liu and Kesselheim analyze the impact of expanded pediatric testing requirements for adult cancer drugs under the Research to Accelerate Cures and Equity (RACE) for Children Act. Though they identify an increase in pediatric post-approval testing requirements, early pediatric trial rates appear unchanged. As more evidence emerges, policymakers may consider using the RACE Act as a model to ensure the timely completion of pediatric studies.
Feder NM, Hernandez I, Feldman WB, Murali M, Kesselheim AS, Elmunzer BJ, Gellad WF | JAMA Network Open
Feder et al. examined price changes for generic indomethacin suppositories. New clinical uses for the drug in 2012 fueled a price increase by the drug’s manufacturer from $13 in 2008 to over $720 in 2023, with a median hospital gross charge price over $1800. The authors indicate that Medicare patients may be particularly vulnerable to this price increase, as the drug is not covered under Medicare Part B or Part D, suggesting that government intervention may be needed.
Viewpoint & Commentary
Freilich J, Kesselheim AS | JAMA
Freilich and Kesselheim discuss efforts to reform the US Patent Trial and Appeals Board (PTAB) via the Promoting and Respecting Economically Vital American Innovation Leadership (PREVAIL) Act. They argue that the PREVAIL Act threatens to worsen drug patent thickets by impeding efforts to challenge patents as improperly granted. Instead, allowing patent challenges at the PTAB to trigger the 180-day generic exclusivity period and for successful challenges to end the 30-month stay on generic entry would be better solutions to strengthen the PTAB and promote generic competition.
Bendicksen L, Kesselheim AS, Feldman WB | JAMA
Bendicksen and colleagues highlight the need for stronger reporting and compliance mechanisms to track federal investments in pharmaceutical research and development, as current disclosure of federal funding in drug patents and government databases is inadequate. Developing a more robust system to monitor the government's pharmaceutical investments would facilitate efforts to quantify federal contributions to drug development, data that would be informative to both federal and state prescription drug programs.
Liu ITT, Kesselheim AS | Journal of the National Comprehensive Cancer Network
Liu and Kesselheim comment on an analysis by Benedict et al. that quantifies the life years gained from the accelerated approval program in oncology. Instead of using 37 months after accelerated approval as the counterfactual date in the authors' analysis, they recommend using a shorter period between 7 and 19 months to reflect prior research and more accurately assess the benefits of accelerated approval.
Recommended by LinkedIn
Raymakers AJN, Kesselheim AS, Feldman WB | JAMA Dermatology
Raymakers and coauthors respond to a letter from Chen et al. commenting on a previous PORTAL estimate of the budgetary effects of beremagene geperpavec (B-VEC) therapy for dystrophic epidermolysis bullosa, agreeing that the therapy’s high cost may be offset by reductions in other health care costs. However, these offsets are likely to vary by disease subtype. The authors also reinforce the value of conducting a QALY-based cost-effectiveness analysis of B-VEC to further inform reimbursement decisions.
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PORTAL in Action
Helen Mooney and Matt Martin attended the National Academy for State Health Policy annual conference in Nashville, which brought together state policymakers to discuss state efforts to improve prescription drug affordability and access, among other topics. September 9-11, 2024
William Feldman and Gregg Furie presented on the environmental impact of metered-dose inhalers during a meeting with the Ohio Hospital Association. September 12, 2024
Aaron Kesselheim participated in a panel discussion hosted by the University of Pennsylvania Leonard Davis Institute of Health Economics on the access and payment challenges for high-cost cell and gene therapies. September 13, 2024
Sean Tu spoke during a Congressional briefing hosted by the ERISA Industry Committee on the potential impacts of patent reform on the drug costs self-insured employers and their employees face. September 17, 2024
Sean Tu and Aaron Kesselheim joined other patent law scholars in sending a letter to Senate Judiciary Committee leadership cautioning them against advancing the Patent Eligibility Restoration Act, which would significantly alter current law on patent-eligible subject matter. September 18, 2024
Aaron Kesselheim joined Consilium Scientific for a conversation about the drug development pipeline in oncology and the treatment of Alzheimer’s, including the use and implications of accelerated approval and other expedited review mechanisms at the FDA. September 19, 2024
Leah Rand presented research on patient and public participation in the Medicare drug price negotiation process at the American Society for Bioethics and Humanities annual conference in St. Louis. September 19, 2024
Aaron Kesselheim participated in an event convened by the Federation of American Scientists (FAS) to discuss the role of advisory committees and patient advocacy at the FDA. He also joined a Greenwall Foundation panel on the cost and resource allocation of GLP-1 drugs for weight loss. September 23, 2024
Benjamin Rome joined a discussion on the payment policy landscape for high-cost cell and gene therapies at a policy summit hosted in Washington DC by the American Society of Gene & Cell Therapy. September 23, 2024
Aaron Kesselheim and Sean Tu spoke alongside other legal scholars and practitioners at an NYU Engelberg Center on Innovation Law & Policy event commemorating the 40th anniversary of the Hatch-Waxman Act. September 25, 2024
Benjamin Rome was part of a symposium at the North American Cystic Fibrosis Conference in Boston to discuss challenges posed by the high cost of new cell and gene therapies to treat rare diseases like cystic fibrosis. September 28, 2024.
About PORTAL
The Program On Regulation, Therapeutics, And Law (PORTAL) is one of the largest research centers in the U.S. studying drug prices, drug policy, and the clinical, economic, and legal aspects of medication use and costs. PORTAL is housed within the Division of Pharmacoepidemiology and Pharmacoeconomics in the Department of Medicine at Brigham and Women’s Hospital (BWH) and Harvard Medical School. To assure independence, no members of PORTAL have any personal financial relationships with pharmaceutical or medical device manufacturers.
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