Aaron Kesselheim

Aaron Kesselheim

@akesselheim.bsky.social

Professor of Medicine, Brigham and Women's Hospital/Harvard Medical School; Director, Program On Regulation, Therapeutics, And Law (PORTAL)

663 followers68 following81 posts

Aaron Kesselheim
@akesselheim.bsky.social

New in @thelancet.com let by T Hwang and K Vokinger, MFN drug pricing in the US would lower prices, with reductions in spending greater than net sales in some countries (but implementation is far away, legally tenuous, and subject to back-room deals/exclusions). www.thelancet.com/journals/lan...

thelancet.comMost-favoured-nation pricing for prescription drugs in US Medicare: a cohort studyMost-favoured-nation pricing for brand-name medicines in Medicare was projected to decrease net spending. Potential reductions in net Medicare spending would be substantially greater than sales in the...
Aaron Kesselheim
@akesselheim.bsky.social

"The FDA traditionally does not make statements about its intention to approve drugs before reviewing data to determine that they are safe and effective, nor do FDA leaders typically appear at press conferences including speculation about drugs’ unproven effects."

Aaron Kesselheim
@akesselheim.bsky.social

"The premature and inaccurate messaging around leucovorin resulted in many young children with ASD being exposed to a treatment with unknown effectiveness and may have perpetuated public distrust in a regulatory process unmoored to scientific evidence."

Aaron Kesselheim
@akesselheim.bsky.social

New from Jerry Avorn and myself in @healthaffairs.bsky.social Forefront: Healing Our De-Commissioned FDA. Marty Makary's legacy will be that he undercut key principles that had made the FDA one of the most important public health institutions in the world. www.healthaffairs.org/content/fore...

healthaffairs.orgHealing Our De-Commissioned FDA | Health Affairs ForefrontMarty Makary's legacy will be that he undercut many of the key principles that had made the agency one of the most important public health institutions in the world.
Aaron Kesselheim
@akesselheim.bsky.social

Vote for PORTAL in the STAT Madness contest for best scientific "innovation" of 2025: discovering improper drug patents listed w/ FDA that FTC later forced mfrs to delist, helping timely generic availability (under MGB logo on the lower right bracket at the bottom): www.statnews.com/feature/stat...

statnews.com2026 STAT Madness2026 bracket Welcome to STAT Madness, a bracket-style contest to find the best innovation in science and medicine. Explore groundbreaking innovations from
Aaron Kesselheim
@akesselheim.bsky.social

This is just needless, self-destructive, and problematic as regulatory policy. Also there never was and isn’t a two trial “dogma.” Bad headline, NEJM. www.nejm.org/doi/full/10....

nejm.orgOne Pivotal Trial, the New Default Option for FDA Approval — Ending the Two-Trial Dogma | NEJMThis Sounding Board announces a new FDA policy that the default requirement for FDA approvals will be one robust pivotal trial plus confirmatory evidence, rather than two trials.

Aaron Kesselheim reposted

Marita Hennessy
@maritahennessy.bsky.social

Stigma, Scarcity & the Price of Legitimacy: Chronic Pain Advocacy & the Politics of Pharmaceutical Partnerships in Canada Daniel Eisenkraft Klein, Benjamin Hawkins, Quinn Grundy, Robert Schwartz onlinelibrary.wiley.com/doi/full/10.... I think findings here potentially transferable to obesity...

onlinelibrary.wiley.comStigma, Scarcity and the Price of Legitimacy: Chronic Pain Advocacy and the Politics of Pharmaceutical Partnerships in CanadaThis study examines how chronic pain advocates in Canada navigated fraught relationships with pharmaceutical companies amid escalating concerns about the adverse public health impacts of opioids. Dra...
Aaron Kesselheim
@akesselheim.bsky.social

With the problematic national priority review voucher in the news again today (FDA staff not involved in decisionmaking? Really?), here’s our overview of how it opens the FDA up to corruption and moves us further and further away from “gold standard” reviews: www.statnews.com/2025/11/21/f...

statnews.comThe Trump administration is turning drug review into make-a-deal“FDA decision-making may be reduced to a sequence of ‘make-a-deals’ with Trump, his FDA, or even political allies,” experts warn.
Aaron Kesselheim
@akesselheim.bsky.social

What he said

MedPage Today@medpagetoday.com

Gutting funding for key health agencies & terminating thousands of workers does nothing to ensure the future of the biotech industry, experts & Democratic lawmakers argued during a Senate health committee hearing on Wednesday. @akesselheim.bsky.social Read more: www.medpagetoday.com/washington-w...

Aaron Kesselheim
@akesselheim.bsky.social

New @healthaffairs.bsky.social Forefront covering the recent EOs on drug prices and tariffs -- little good news for prices or access -- www.healthaffairs.org/content/fore...

healthaffairs.orgTrump’s Ongoing Tariff Chaos Will Make Medications Less Affordable And Harder To Find | Health Affairs ForefrontIn his first term, Trump made a lot of noise about lowering drug prices but accomplished little of consequence. The noise continues, but it is now accompanied by new ill-conceived initiatives, includi...
Aaron Kesselheim
@akesselheim.bsky.social

Unrecorded manipulation of large datasets has been common in the US government since Trump's election -- bad for science, trust, and transparency -- in @thelancet.com from Janet Freilich and myself: www.thelancet.com/journals/lan...

thelancet.comData manipulation within the US Federal GovernmentA US Department of Veterans Affairs dataset compiling veteran health-care use in 2021 was quietly amended on March 5, 2025. A column titled gender was renamed sex, and the words were also switched in ...
Aaron Kesselheim
@akesselheim.bsky.social

Language is now back in the Senate version of the OBBA that would undermine Medicare price negotiation for drugs with Orphan Drug Act designation. Here's our HA Forefront published yesterday (!) from Helen Mooney and @benro.me on why it is a very bad idea: www.healthaffairs.org/do/10.1377/f...

healthaffairs.orgCongress Should Remove The Rare Disease Carve-Out From Medicare Drug Price Negotiation, Not Expand It | Health Affairs ForefrontExpansion of the Medicare drug price negotiation rare disease drug carve-out would unnecessarily limit the number of drugs eligible for negotiation, allow for continued high drug prices for products that earn billions of dollars in Medicare, and introduce a new set of misaligned incentives.
Aaron Kesselheim
@akesselheim.bsky.social

New in @jama.com, Joseph Daval and I discuss legal issues surrounding ACIP including its role in setting government policy and dealing with politically-motivated agendas arising from its new membership: jamanetwork.com/journals/jam...

jamanetwork.comLegal Challenges for the Advisory Committee on Immunization PracticesThis Perspective offers an analysis of the legal foundations in the context of these and other challenges to the role of the Advisory Committee on Immunization Practices.
Aaron Kesselheim
@akesselheim.bsky.social

Analysis from former @portalresearch.org fellow Catherine Hwang and @benro.me showing the extent of out-of-pocket costs associated with starting brand-name Entresto over ACEI/ARB for heart failure and implications for overall spending: jamanetwork.com/journals/jam...

jamanetwork.comSpending After Sacubitril-Valsartan vs Renin-Angiotensin System Blockers for Heart FailureThis cohort study assesses health care costs after initiating sacubitril-valsartan compared to an angiotensin-converting enzyme inhibitor or an angiotensin II receptor-blocker in Medicare patients bei...
Aaron Kesselheim
@akesselheim.bsky.social

New in @nejm.org from Peter Ubel, Astrid Grouls and myself on "financial toxicity" and how to consider it in the context of new drugs @portalresearch.org: www.nejm.org/doi/abs/10.1...

nejm.orgOut of Pocket Getting Out of Hand — Reducing the Financial Toxicity of Rapidly Approved Drugs | NEJMThe FDA often exercises flexibility in deciding whether to approve highly promising drugs for patients in desperate need of treatment options. But it doesn’t consider a drug’s likely financial toxi...
Aaron Kesselheim
@akesselheim.bsky.social

Survey of clozapine prescribers shows satisfaction with the REMS but desire for more educational info, led by @asarpatwari.bsky.social & team at @portalresearch.org in collab with FDA, where world class scientists are sadly being censored and forced out of their jobs: jamanetwork.com/journals/jam...

jamanetwork.comPhysician Experiences With and Perspectives on Clozapine PrescribingThis survey study examines physician-prescriber experiences with and perspectives on the clozapine risk evaluation and mitigation strategy.
Aaron Kesselheim
@akesselheim.bsky.social

Interesting use of modeling to predict the timing of patent challenges seeking generic drug entry in PLoS Medicine, led by Ally Memedovich and @portalresearch.org faculty affiliate Reed Beall at Univ Calgary - journals.plos.org/plosmedicine...

journals.plos.orgPredicting patent challenges for small-molecule drugs: A cross-sectional studyAlly Memedovich and colleagues report on patent challenges within the first year of eligibility among small-molecule drugs approved by the FDA from 2007-2018 and investigate the extent to which market...

Aaron Kesselheim reposted

Program On Regulation, Therapeutics, And Law (PORTAL)
@handle.invalid

Check out the February edition of the PORTAL Post, featuring analysis on: - the promise and risk of drug repurposing, - drug importation to address shortages, - use of subgroup analysis in cost-effectiveness studies - biosimilar patent litigation, - and much more! #medsky #healthpolicy

mailchi.mpPORTAL Post | February 2025Protecting health agency independence, drug repurposing, academic detailing, and more from the PORTAL team.
Aaron Kesselheim
@akesselheim.bsky.social

Criticism of the QALY includes the idea that it'll be used to limit drug access to patient subgroups, but @araymakers.bsky.social shows in J Cancer Policy that no evidence of QALYs in cancer drugs being used to do that -- from @portalresearch.org: www.sciencedirect.com/science/arti...

sciencedirect.comAn empirical investigation into concerns over quality-adjusted life-years: a review of cost-effectiveness analyses in oncologyHealth care payers often use cost-effectiveness analyses (CEA) using the quality-adjusted life-year (QALY), as the measure of benefit, to inform reimb…
Aaron Kesselheim
@akesselheim.bsky.social

Use of Doxepin for insomnia -- example of potentially useful drug repurposing that seems to be under-prescribed among patients who could benefit because of a problematically high pricing point -- from Chris Cai and @benro.me at @portalresearch.org: jamanetwork.com/journals/jam...

jamanetwork.comTrends in Fills, Spending, and Prices of Doxepin for InsomniaThis cross-sectional study explores US prescription fills, retail spending, and prices of low-dose doxepin for insomnia and the possible cost savings to patients had they used liquid doxepin, a less e...
Aaron Kesselheim
@akesselheim.bsky.social

New in @jlme-journal.bsky.social from co-first authors Liam Bendicksen and Mirre Scholte as well as others at @repo4eu.bsky.social, a review of results from research and roundtables on regulatory pathways for drug repurposing in the US & EU: www.cambridge.org/core/journal...

cambridge.orgA Regulatory Roadmap for Repurposing: Comparing Pathways for Making Repurposed Drugs Available In The EU, UK, And US | Journal of Law, Medicine & Ethics | Cambridge CoreA Regulatory Roadmap for Repurposing: Comparing Pathways for Making Repurposed Drugs Available In The EU, UK, And US - Volume 52 Issue 4
Aaron Kesselheim
@akesselheim.bsky.social

New piece led by Anushka Bhaskar @portalresearch.org in STAT First Opinion on protecting public health agencies from damaging political interference -- even the threat of which can greatly affect people's behavior: www.statnews.com/2025/01/31/p...

statnews.comHow to protect HHS, FDA, NIH, and other health agencies from political interferenceWhen politicians subordinate scientific institutions to electoral interests, they undermine immediate public health efforts and inflict lasting damage on institutional credibility.
Aaron Kesselheim
@akesselheim.bsky.social

From Rosa Ahn-Horst @portalresearch.org -- psychiatric drug innovation over the last decade shows widespread use of clinical endpoints but few new mechanisms of action and 3/16 drugs for which < half the trials submitted to FDA were positive (in JAMA Network Open): jamanetwork.com/journals/jam...

jamanetwork.comTrials Preceding FDA Approval of Novel Psychiatric DrugsThis cross-sectional study reports on the study design characteristics of clinical trials for novel psychiatric drugs approved by the US Food and Drug Administration.

Aaron Kesselheim reposted

The Journal of Law, Medicine and Ethics
@jlme-journal.bsky.social

Due to the Trump administration freeze on communication from government agencies, an article JLME planned to publish this spring on research misconduct has been placed on hold. Read more: www.statnews.com/pharmalot/20...

statnews.com'This is censorship': Trump freeze on communications forces medical journal to pull HHS authors' articleThe article had to be pulled even though HHS Office of Research Integrity officials gave a public talk on the topic last fall
Aaron Kesselheim
@akesselheim.bsky.social

Also out this week a review of new EU pharma policies and their potential impact on repurposed drugs with the excellent Mirre Scholte and Manuela Joore who are collaborating with @portalresearch.org on the REPO4EU project: pubmed.ncbi.nlm.nih.gov/39756647/

pubmed.ncbi.nlm.nih.govRevising EU pharmaceutical legislation: will it foster drug repurposing? - PubMedRepurposing off-patent drugs can be a potential source of low-cost treatments for patients with unmet medical needs. Here, we review the proposed new European Union (EU) pharmaceutical legislation in ...
Aaron Kesselheim
@akesselheim.bsky.social

In @nejm.org this week led by @tombollyky.bsky.social with Sarosh Nagar of @portalresearch.org and Chloe Searchinger - how a rational regulatory approach to supporting importation of generic drugs can be used to respond to essential medicine shortages: www.nejm.org/doi/full/10....

nejm.orgThe Role of Importation in Remediating U.S. Generic Drug Shortages | NEJMU.S. shortages of critical drugs are a worsening problem, and the FDA has been too slow to authorize temporary importation of drugs from other well-regulated markets to meet pressing U.S. needs.
Aaron Kesselheim
@akesselheim.bsky.social

Dry powder inhaler shows similar effectiveness and safety to metered-dose inhaler, w numerous implications including that environmentally-friendly prescribing in this case is also clinically equivalent -- led by @wbfeldman.bsky.social of @portalresearch.org in BMJ: www.bmj.com/content/387/...

bmj.comComparative effectiveness and safety of single inhaler triple therapies for chronic obstructive pulmonary disease: new user cohort studyObjective To compare the effectiveness and safety of budesonide-glycopyrrolate-formoterol, a twice daily metered dose inhaler, and fluticasone-umeclidinium-vilanterol, a once daily dry powder inhaler,...