Program on Regulation, Therapeutics, and Law (PORTAL)

Interested in the latest research from the PORTAL team? Check out the October edition of the PORTAL Post newsletter, with analysis and commentary on: - the CMMI High-Value Drug List Model - Drug price negotiation processes in the US and abroad - Biosimilar approval pathways - Pediatric testing requirements for cancer drugs - Federal investments in drug development - Proposed reforms at the Patent Trial and Appeals Board (PTAB) - And more! View the newsletter below and share with your colleagues! #drugs #pharmacy #pediatric #FDA #patents #pharma #healthpolicy #Medicare #biosimilar #ptab #generics #drugdevelopment

Excited to share the first output of my The Commonwealth Fund Harkness Fellowship. Appreciate the collaboration with William Feldman, MD, DPhil, MPH, Kevin Schulman and Aaron S. Kesselheim, M.D., J.D., M.P.H. on this. The paper compares frameworks for drug price negotiations in 8 different health systems (Belgium, Canada, France, Germany, the Netherlands, Norway, UK (England) and the US Veterans Affairs Health System). We hope this paper will be useful for policymakers in identifying best practices for drug price negotiations and provide valuable insights into the ongoing development of an effective drug price negotiation process in the US. Stanford Clinical Excellence Research Center Program on Regulation, Therapeutics, and Law (PORTAL) Reginald D. Williams II Molly Fitzgerald

We have a new paper out led by Iselin Dahlen Syversen that compared how 8 different health systems (Belgium, Canada, France, Germany, Netherlands, Norway, UK, and the VA) negotiate prescription drug prices. Iselin conducted interviews with negotiators in each system and synthesized government documents and published academic studies to compare how systems differed across several axes of interest. Nearly all systems negotiate the prices of prescription drugs immediately after approval. Frameworks for drug price negotiation differed on several key parameters, including who performs the clinical assessments, how added health benefit is quantified, whether explicit willingness-to-pay thresholds are employed, and how specific approaches for priority diseases areas are taken. We hope this article will be useful for policymakers in the US and elsewhere as they think about how to refine frameworks for drug price negotiation. Appreciate the chance to collaborate with Iselin Dahlen Syversen, Kevin Schulman, and Aaron S. Kesselheim, M.D., J.D., M.P.H. on this work. Program on Regulation, Therapeutics, and Law (PORTAL) https://lnkd.in/g4zS8HZz

Led by Adam Raymakers, we recently published a piece in JAMA Dermatology on the potential budget impact of the first approved topical gene therapy--B-VEC for dystrophic epidermolysis bullosa. We are grateful for the thoughtful comments we received on the manuscript. We amplify 2 key points in the attached response. 1. B-VEC therapy will be associated with important cost offsets, but these offsets are likely to differ substantially between those with autosomal recessive disease (RDEB) and autosomal dominant disease (DDEB). The pivotal GEM-3 trial only included 1 patient with the milder DDEB. Yet, the drug is approved for both forms of the disease. The health gains associated with B-VEC therapy in patients with DDEB are uncertain but likely to be substantially less than in RDEB. 2. Budget impact studies like the one we completed are important, but formal cost-effectiveness analyses remain vital to inform reimbursement decisions. Unfortunately, unlike other industrialized countries, the US lacks a centralized HTA body to conduct these analyses for newly approved drugs. Developing such a mechanism for centralized assessment should be a public health priority. https://lnkd.in/gv75qjHw

Check out the latest research from PORTAL in this month's newsletter, featuring analysis on: - Biosimilar use in the US - Expanded Medicare coverage for Wegovy - The legal and policy landscape of patient assistance programs - The high price of sickle cell gene therapies - And more! Aaron S. Kesselheim, M.D., J.D., M.P.H. William Feldman, MD, DPhil, MPH Benjamin Rome S. Sean Tu Hussain Lalani, MD, MPH, MSc Edward Cliff #drugs #pharmacy #FDA #patents #pharma #healthpolicy #Medicare #GLP1s #biosimilar #patient

New research in Annals of Internal Medicine by Alexander Chaitoff, Liam Bendicksen, William Feldman, Alexander Zheutlin, and Hussain Lalani evaluates the potential impact of expanded Medicare coverage of semaglutide (Wegovy) for patients with elevated BMI and "established cardiovascular disease." They find that 3.6 million Medicare beneficiaries would likely be newly eligible for Wegovy based on this coverage decision, costing Medicare up to $34.3 billion annually if all patients began treatment. If "established cardiovascular disease" was more narrowly defined, only 1 in 7 Medicare patients with elevated BMI would likely be eligible, with an added cost of $10 billion per year. And, if the broadest definition was used, 15.2 million beneficiaries may qualify, adding up to $145 billion in annual costs to Medicare. Read the full analysis: https://t.co/wjE1oQakN9 #GLP1s #Medicare #obesity #pharmacy #healthcare #healthplans

With today’s news from Pfizer and Eli Lilly, re-upping this JAMA viewpoint I wrote earlier this year about Direct-to-Consumer Drug Company Pharmacies. https://lnkd.in/eh_-8Q6Y

Excited to share a new grant that we received today from the FDA! Over the next few years, we’ll be collaborating with folks at the FDA's Office of Generic Drugs to develop tools for analyzing the performance of complex generic products—eg, inhalers, transdermal patches, and injector pens—using real-world evidence. The FDA continues to take measures aimed at streamlining the entry of generic drugs in the Unites States. A robust generic drug market is key for lowering health care costs for patients and payers. Looking forward to working on this w/ co-PI Shirley V Wang and folks at the Program on Regulation, Therapeutics, and Law (PORTAL), Brigham and Women's Hospital, and Harvard Medical School. You can learn more about the grant here: https://lnkd.in/gK7kztXJ

New analysis in JAMA from S. Sean Tu, Aaron S. Kesselheim, M.D., J.D., M.P.H., and Bernard Chao on the prevalence of drug #patents with terminal disclaimers and obviousness-type double patenting rejections over the last two decades.

New research by Dongzhe Hong, Aaron Kesselheim, Ameet Sarpatwari, and Benjamin Rome in Health Affairs analyzes the patient & prescriber factors impacting US #biosimilar use from 2013-2022, including geography, patient age, prescriber type, & where the drug was administered (hospital outpatient facility vs. physician's office). Though the proportion of patients who initiated a biosimilar increased from 1% to 34% over the study period, variations in uptake across these factors highlight the need for targeted policy intervention to increase biosimilar use, which can generate patient and health care system savings. https://lnkd.in/ef4Dn2zD