Adam Raymakers

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...

The FDA is increasingly turning to ad hoc expert panels instead of federal advisory committees for external advice. FDA watchers generally agree these expert panels "are likely unlawful," USC Schaeffer's @profgenkanter.bsky.social tells Regulatory Focus.

New paper just out! Led by Arianna Gentilini and @leah-rand.bsky.social, we performed a systematic review of studies investigating COI in FDA advisory committee meetings.

Full article (in press) here: www.valueinhealthjournal.com/article/S109...

We would like to recruit a stellar junior faculty member in the Division of Child Health Research and Policy @deptpopmed.bsky.social at Harvard. Please circulate this announcement widely!

www.populationmedicine.org/divisions-ce...

New paper just out! Led by Arianna Gentilini and @leah-rand.bsky.social, we performed a systematic review of studies investigating COI in FDA advisory committee meetings.

Full article (in press) here: www.valueinhealthjournal.com/article/S109...

CAR-T for all? Barriers, facilitators, and interventions to commercial CAR T-cell therapy access Chimeric antigen receptor T-cell (CAR-T) immunotherapy has revolutionized the treatment of hematologic malignancies, especially in B-cell acute lympho…

Really interesting new article by Kaparis et al from
@pennmedicine.bsky.social providing an overview of the current barriers/facilitators to accessing CAR-T therapy.

sciencedirect.com/science/arti...

East coast fall.

#capebretonisland

Some more @cnn.com coverage of our study, led by @wbfeldman.bsky.social, on greenhouse gas emissions associated with inhaler use in the US.

Climate pollution from inhalers has the impact of half a million cars per year, study finds | CNN The people who are most vulnerable to the hard-to-breathe air that comes with climate change may inadvertently be adding to the problem, new research finds.

Happy to see that @cnn.com picked up our @jama.com on greenhouse gas emissions and inhaler usage in the US.

Original article: jamanetwork.com/journals/jam...

www.cnn.com/2025/10/06/h...

Really excited to share, and happy have been a part of, this new paper led by @wbfeldman.bsky.social out today in @jama.com!

We conducted a comprehensive analysis of greenhouse gas emissions associated with inhaler usage in the US.

Read it here: jamanetwork.com/journals/jam...

October 2025 | The future of the FDA, pediatric clinical trials, and the budget impact of sickle cell therapies. On September 30, PORTAL hosted a symposium on the future of drug regulation at the FDA, featuring conversations with Peter Marks, former Director of the Center for Biologics Evaluation and Research (C...

New edition of the PORTAL Post (the @portalresearch.org monthly newsletter), featuring lots of great research, including our recent publication about modelling the budget impact of gene therapies to treat sickle cell disease in the US.

Check it out here: www.linkedin.com/pulse/octobe...

CC: @portalresearch.org @hsph.harvard.edu @yaleschoolofmed.bsky.social @yalecancer.bsky.social

Modeling the Budgetary Impacts of Sickle Cell Disease Gene Therapies on State Medicaid Programs - Journal of General Internal Medicine Journal of General Internal Medicine -

Excited to share new research led by Jessica Harvey exploring the budget impact of gene therapies for sickle cell disease, with a focus on Medicaid programs.

Co-As: @ankurfactorial.bsky.social, @leah-rand.bsky.social, @akesselheim.bsky.social, & George Goshua.

🔗: link.springer.com/article/10.1...

The Common Sense Oncology Fellowship program is now accepting applications. Definitely a great opportunity for anyone interested in health services research and oncology!

commonsenseoncology.org/wp-content/u...

Annual trip home to the cottage!

#CapeBretonIsland

Breaking: FDA will ask Sarepta to stop selling Duchenne treatment, following patient deaths FDA to request Sarepta halt shipments of DMD drug Elevidys after two patient deaths, HHS official says. Trading of $SRPT shares paused after decline.

'The FDA will ask Sarepta to stop shipping its Duchenne muscular dystrophy treatment Elevidys, an HHS official said, in what could be a final blow to the company’s hopes for the product following two patients’ deaths.'

via @endpts.com

endpoints.news/fda-asks-sar...

Pharma exports from Ireland climb again as Trump's drug tariffs inch closer New data from Ireland’s Central Statistics Office suggest that pharmaceutical companies are exporting more inventory to the US as the threat of drug tariffs persists.

'Drugmakers [said] they were stockpiling drugs in the US as a strategy to curb the impact of potential pharma tariffs. In March, Ireland saw a massive 243% increase in pharma exports, compared to the same month last year.'

via @annajbrown.bsky.social / @endpts.com

endpoints.news/pharmaceutic...

Fantastic postdoc fellowship opportunity @mcgill.ca!

mcgill.wd3.myworkdayjobs.com/McGill_Caree...

Always a super interesting read!

Notably, amongst the top 20 CEOs, average compensation was greater than $30 million.

Total compensation for the top 20 CEOs was more than $600,000,000.

Also notable that there is only one woman amongst the top 20.

via @andrewedunn.bsky.social / @endpts.com

Just out! The new guidelines from the Canadian Patented Medicine Prices Review Board (PMPRB) have finally been released. Changes to take effect as of January 2026.

#hta #drugprices

www.canada.ca/en/patented-...

New Health Affairs Forefront by @akesselheim.bsky.social and @jerryavorn.bsky.social from @portalresearch.org about the impact of tariffs on the affordability and availability of medications.

www.healthaffairs.org/content/fore...

A little work to make one of the local #Squamish trails a bit more friendly to those riding adaptive mountain bikes.

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.

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...

Vancouver Island family heartbroken as B.C. stops funding $1M drug for girl with terminal condition | CBC News Charleigh Pollock's mother says the family has gone through a roller-coaster of emotions since learning the B.C. government has pulled funding for the drug their daughter needs for the rare and termin...

The clinical aspect & decision aside, it's problematic for health systems that BioMarin priced Brineura at $1 million. If companies didn't charge these prices, decisions would be easier & less restrictive.

Note: Brineura's net sales in 2024 were $170 million (US alone).

www.cbc.ca/news/canada/...

'A 2023 analysis of more than 20 million deaths across the world found that hot days and days with bad air quality both resulted in higher-than-normal mortality rates. But periods in which heat and pollution are combined were even more deadly.'

via @nytimes.com

Looking for a recent paper that reports on HRQoL via EQ-5D-5L/3L scores, that specifically reports on the each of the domains/levels of the EQ-5D.

Preferably in a chronic disease, and also preferably US-based (but not necessary). Anyone? @euroqol.bsky.social

#healtheconomics #hspr #QALYs

Done!

Our special issue, “Advancing Trust in Science: Institutional Obligations to Promote Research Integrity,” is now live in @jlme-journal.bsky.social. Check out the full line up of leading voices on spotting, addressing, and preventing misconduct.

www.cambridge.org/core/journal...

I've attended this event in the past and it's absolutely fantastic! Not to mention Galway is an incredibly fun city to visit. Highly recommend!

Added a few more folks recently so thought I'd re-share.

If I've missed you, reach out and I'll add you to the list!

#healtheconomics

Novo Nordisk's Canadian Mistake

Super interesting story about Novo Nordisk and their Canadian patents.

'... Novo did file a patent there for semaglutide. . .but the last time they paid the annual maintenance fee on it was 2018!'

by @dereklowe.bsky.social

www.science.org/content/blog...