Watch the full hearing including Dr. Ramachandran’s testimony here: www.help.senate.gov/hearings/the... (5/5)

In the piece, the authors proposed alternatives to having decisions made by single FDA officials that run counter to multiple review teams as had occurred with Elevidys as well as more recently, broader access to COVID-19 vaccines. (2/3)

CRRIT Co-Director @reshmagar.bsky.social provided public comments at the Prescription Drug User Fee Act (PDUFA) VIII Kickoff Meeting @fda.gov yesterday. Dr. Ramachandran discussed how the user fee process could be reformed to be more patient-centered & transparent.

Do companies continue promoting drugs granted
@fda.gov accelerated approval even after failed confirmatory trials? Yes—though less aggressively, finds Dr. Maryam Mooghali’s #ASCO25 Merit Award-winning research.

🔗 meetings.asco.org/abstracts-pr...

...her team found that information published in scholarly journals is becoming more accessible over time (see below ⬇️)—giving patients and their caregivers more agency to make the health decisions best for them. #InfoAsHealth
@jsross119.bsky.social @genpatient.bsky.social

Some more photos of our wonderful CRRIT trainee team presenting their research at #SGIM25! And we managed to get everyone together for dinner right by the beach 🏖️.

So proud of our CRRIT @yaleschoolofmed.bsky.social students & @ncspyale.bsky.social fellows for presenting their research projects on the evidence underlying @fda.gov approvals, FDA safety actions, costs of care, drug pricing, & more at #SGIM25! @jsross119.bsky.social @reshmagar.bsky.social #MedSky

In this study, we reviewed @fda.gov approvals of high-risk therapeutic devices from 2019–2023 to see how often nonconcurrent controls were used—and whether @fda.gov justified their use.

These situations may call for nonconcurrent controls—historical data, objective performance criteria, or preset goals—derived from older clinical data. The limitation of this approach, however, is that such data may not provide a fair comparison of today’s patients or standards of care.

Results: As of July 2023, Medicare had approved 116 studies through the CED program to study 26 products, most of which (19, 73%) were medical devices. Results were available from 57 studies (49%), resulting in 556 research publications.

Check out our public comment submitted to @fda.gov on the accelerated approval pathway that clarifies how FDA can balance faster drug approval with ensuring sponsors confirm clinical benefit afterward – and if not, withdrawing products in a timely manner:
www.regulations.gov/comment/FDA-...

Interestingly, more than half (54.8%) of late reports came from 3 manufacturers (0.1% of the 3028 manufacturers who submitted reports between 2019 and 2022). Just 10 manufacturers accounted for about 83% of late reports.

Moreover, 39 (49.4%) did not report the adverse effects, warnings & precautions, and contraindications of GLP-1RAs.

(Post 5/9)

Ozempic, Wegovy, & other GLP-1RAs need no introduction. Striking demand has caused persistent shortages, legally permitting pharmacies to compound and sell GLP-1RAs without both @fda.gov approval and the agency’s typical oversight over drug advertising.

(Post 2/9)

CED-approved studies proved robust: they are often randomized clinical trials (67%) with large enrollment (median sample size: 1000), especially of US patients (93% of subjects).

In Health Affairs, we analyzed the availability and quality of recent Medicare Local Coverage Determination summaries. Since 2019, the program has dramatically improved its public summary reporting. But the quality of evidence they cite within is...lackluster.

www.healthaffairs.org/doi/full/10....