Aaron Kesselheim
@akesselheim.bsky.social
Professor of Medicine, Brigham and Women's Hospital/Harvard Medical School; Director, Program On Regulation, Therapeutics, And Law (PORTAL)
New in Pediatrics led by @benro.me and John Kim showing increase in leucovorin use after FDA statements/publicity: publications.aap.org/pediatrics/a...
Use of Leucovorin After the FDA Announcement of Its Use for Autism Spectrum Disorders
BACKGROUND AND OBJECTIVES. Leucovorin, or folinic acid (vitamin B9), is approved by the Food and Drug Administration (FDA) to reduce adverse effects from antifolate cancer treatments including methotr...
publications.aap.org
From FactCheck.org -- do RFK's claims of record-breaking new FDA review speeds hold up? Uh, no. www.factcheck.org/2026/05/rfk-...
RFK Jr.’s Muddled Claims on Drug Approval Speed - FactCheck.org
The Food and Drug Administration in recent months has approved a small number of drugs quite quickly under a new expedited review program. But Health and Human Services Secretary Robert F. Kennedy Jr....
factcheck.org
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...
Healing Our De-Commissioned FDA | Health Affairs Forefront
Marty 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.
healthaffairs.org
PORTAL made it to the STAT Madness Sweet Sixteen for best innovation in science/medicine of 2025! Now we're up against the formidable UC-Irvine Blind Mice. Vote here to ensure PORTAL gets to the next round: www.statnews.com/feature/stat...
2026 STAT Madness
2026 bracket Welcome to STAT Madness, a bracket-style contest to find the best innovation in science and medicine. Explore groundbreaking innovations from
statnews.com
Still dancing! By fewer than 2 (percentage) points, made it to the Round of 32 and now we're facing off against the Scripps Rhinos. Once again, vote early and vote often for PORTAL in STAT Madness here: www.statnews.com/feature/stat...
2026 STAT Madness
2026 bracket Welcome to STAT Madness, a bracket-style contest to find the best innovation in science and medicine. Explore groundbreaking innovations from
statnews.com
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...
2026 STAT Madness
2026 bracket Welcome to STAT Madness, a bracket-style contest to find the best innovation in science and medicine. Explore groundbreaking innovations from
statnews.com
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....
One Pivotal Trial, the New Default Option for FDA Approval — Ending the Two-Trial Dogma | NEJM
This 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.
nejm.org
Great coverage of Joseph Daval and my article in JAMA on the highly problematic new FDA use of “expert panels”: www.raps.org/news-and-art...
Experts argue recent FDA expert panels may violate FACA requirements
The US Food and Drug Administration@s (FDA) increasing reliance on holding expert panels, rather than advisory committees, for external expert advice may violate the Federal Advisory Committee Act (FA...
raps.org
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...
Stigma, Scarcity and the Price of Legitimacy: Chronic Pain Advocacy and the Politics of Pharmaceutical Partnerships in Canada
This 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...
onlinelibrary.wiley.com
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...
The 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.
statnews.com
What he said
MedPage Today@medpagetoday.com · 9mo agoGutting 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...
I filed an amicus brief on behalf of 24 professors challenging the interim PTO director's illegal policy of blocking IPRs for any patent that is more than a few years old. drive.google.com/file/d/1NhrG...
drive.google.com
Reprint of my Substack with @ylepidemiologist.bsky.social today in @statnews.com. Check it out if you’re interested in recent “moves” the FDA is making on drug ads. As with most claims this admin makes … IBIWISI: www.statnews.com/2025/09/13/p...
9 answers to burning questions about pharmaceutical ads
Got questions about the FDA’s new plans around pharmaceutical ads? We’ve got answers.
statnews.com
Another fun collaboration with @ylepidemiologist.bsky.social in Substack with a post on DTC drug ads and the new administration announcement claiming to seek greater enforcement: yourlocalepidemiologist.substack.com/p/pharmaceut...
Pharmaceutical ads in the U.S.: Top questions answered
The Administration's order and what can actually be done
yourlocalepidemiologist.substack.com
High brand-name drug prices fall once a generic enters the market. In a new @journalgim.bsky.social article, Ravi Gupta, CRRIT Co-Director @jsross119.bsky.social, and colleagues from @portalresearch.org assess associations between patents, revenue, and generic competition.
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...
Trump’s Ongoing Tariff Chaos Will Make Medications Less Affordable And Harder To Find | Health Affairs Forefront
In 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...
healthaffairs.org
New PORTAL Post from @portalresearch.org for June. Sign up to receive these directly to your inbox! mailchi.mp/eb7d1808132b...
PORTAL Post | July 2025
New faculty members, the ORPHAN Cures Act, “hyaluronidase hopping,” and more from PORTAL
mailchi.mp
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...
Data manipulation within the US Federal Government
A 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 ...
thelancet.com
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...
Congress Should Remove The Rare Disease Carve-Out From Medicare Drug Price Negotiation, Not Expand It | Health Affairs Forefront
Expansion 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.
healthaffairs.org
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...
Legal Challenges for the Advisory Committee on Immunization Practices
This 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.
jamanetwork.com
Thanks to the 2024 peer reviewers for @jlme-journal.bsky.social, with a list here: www.cambridge.org/core/journal...
Thank You to Our 2024 Peer Reviewers | Journal of Law, Medicine & Ethics | Cambridge Core
Thank You to Our 2024 Peer Reviewers
cambridge.org
I helped Your Local Epidemiologist answer some of her pressing Substack subscriber questions on drug prices. yourlocalepidemiologist.substack.com/p/your-top-1...
Your top 10 FAQ on drug prices
Some clarity for this wildly confusing topic.
yourlocalepidemiologist.substack.com
New Health Affairs Forefront post on the Medicare negotiation "biologic bonus" and the massive financial hit to patients and the health care system of delaying small molecule negotiation from 9 to 13 years, led by Chris Cai and @benro.me: www.healthaffairs.org/content/fore...
healthaffairs.org
New op-ed with @drjoshs.bsky.social and Peter Lurie about the agenda-driven reality of 'radical transparency' -- www.washingtonpost.com/opinions/202...
Opinion | It’s not hard to see through RFK Jr.’s ‘radical transparency’ pledge
So far, what’s been released is little more than tactics in pursuit of Kennedy’s own agenda.
washingtonpost.com
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...
Spending After Sacubitril-Valsartan vs Renin-Angiotensin System Blockers for Heart Failure
This 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...
jamanetwork.com
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...
Out of Pocket Getting Out of Hand — Reducing the Financial Toxicity of Rapidly Approved Drugs | NEJM
The 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...
nejm.org
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...
Physician Experiences With and Perspectives on Clozapine Prescribing
This survey study examines physician-prescriber experiences with and perspectives on the clozapine risk evaluation and mitigation strategy.
jamanetwork.com
