Holly Fernandez Lynch

Leucovorin lacks evidence to use as autism treatment, FDA says The FDA approves leucovorin for a rare brain disorder while retreating from top officials’ earlier insistence that it could benefit many children with autism.

News: Federal health officials are retreating from earlier endorsement of leucovorin as a drug that can treat autism.

www.statnews.com/2026/03/10/f...

As controversial decisions mount, FDA shuns public advisory meetings The FDA is cutting back on public advisory committee meetings, even as the public and other stakeholders seek more transparency.

Despite recent controversial decisions, the FDA has stopped holding advisory committee meetings — which bring together regulators, companies, patients, and a panel of independent advisors to publicly discuss complex regulatory matters. I dug into why:
www.statnews.com/2026/03/09/f...

Institutional Review Board Obligations Regarding Study Funding Sufficiency Given their obligations to ensure ethical research, we argue that institutional review boards (IRBs) bear a responsibility to minimize the possibility that proposed clinical studies will terminate ea...

New pub w/ my fab Penn JD/MBE student, Tasneem Mohammad!
Prompted by an ALS drug sponsor's announcement that it was considering launching a trial before it had all $ in hand, we addressed the ethical role of IRBs in making sure studies have adequate funding.
onlinelibrary.wiley.com/doi/10.1002/...

"If nothing else, one hopes he has learned that the job of being an FDA official is even harder than it looks."

Thank you, @adamfeuerstein.bsky.social, for reporting Prasad's self-identifying statements from that press call (without naming him).

I don't think his departure comes today without it.

With Prasad bouncing from the FDA, now we’re never ever EVER gonna see the data.

Exclusive | FDA’s Controversial Vaccines Chief Will Leave the Agency Dr. Vinay Prasad will depart in April, after a year leading the division that approves vaccines and biotech drugs.

Prasad on press call:
"I serve at the pleasure of the [FDA] commissioner and the [health] secretary and the president, and as long as we’re all in alignment—that me serving is in the best interest of the American people—I will continue to serve happily."
1day later, out:
www.wsj.com/health/healt...

In case you were wondering, this is how a normal FDA responds when it feels it needs to clarify a company's public statements about an investigational product.

www.fda.gov/vaccines-blo...

UPDATED: Senior FDA official refutes uniQure's claims amid controversy over Huntington's gene therapy In a strikingly blunt briefing, a senior FDA official accused uniQure of pushing “distorted” data to mask a “failed” therapy for Huntington’s disease. | In a strikingly blunt briefing, a senior FDA of...

Would the sham procedure Prasad's asking for fool anyone? 10-12 hour surgery + holes in the skull v. 3 scalp nicks in 30 min. If it's not a good sham, it's not ethical to proceed.

Prasad has raised impt concerns about UniQure's data tho. Take it to an adcom.

www.fiercebiotech.com/biotech/seni...

The FDA, urged to avoid controversy, creates a new headache with attack against UniQure Recent decisions at the FDA threaten to turn its disagreements with the drugmaker UniQure into another political headache for the Trump administration.

Confirmed.
www.statnews.com/2026/03/06/f...

The FDA, urged to avoid controversy, creates a new headache with attack against UniQure Recent decisions at the FDA threaten to turn its disagreements with the drugmaker UniQure into another political headache for the Trump administration.

We all knew but here's confirmation the anon official is Prasad:

"You all know that as background, I’m a professor and I’m a practicing [hematology-oncology] doctor. I do miss my clinic and I miss teaching, and I miss being able to run my podcast and speak freely."
www.statnews.com/2026/03/06/f...

An Autism Drug Got a White House Boost. Prescriptions Soon Followed.
Researchers say a 2025 press briefing was followed by a sharp rise in prescriptions for leucovorin, an inexpensive drug now being studied for autism.
Read & subscribe (for free!): www.celinegounder.com/p/an-autism-...

two men hugging each other with the words " i know it was you fredo " on the bottom ALT: two men hugging each other with the words " i know it was you fredo " on the bottom

I know, I was laughing too. Who does he think he's kidding?

2. I don't know if the design is needed here given the existing data. But I wish we had more than a press release on the 3 year results. (Have I missed a publication?)

3. I am blown away by FDA's public comments on the matter.

4. This needs to get in front of an advisory committee stat.

1. It is not *necessarily* unethical to run a sham surgery trial for a serious disease. It depends on whether it's necessary to answer the relevant scientific questions and if risks can be minimized enough for the benefits of answering those questions to outweigh them.

WATCH: 'I'm done,' Hillary Clinton says after learning Boebert posted photo of her at deposition YouTube video by PBS NewsHour

There are three women I NEVER want to infuriate:

1. Elena Kagan
2. Hillary Clinton
3. Mandy Moore - There's a reason my audio was cut from our duet version of "I Wanna Be With You."

Oh I know - lots of cringey statements in that piece. But gotta latch onto the positive where I can.

It makes me sad how instrumental my own 16 year old is sometimes about his education :( It's in the ether, I guess, because I have tried so hard not to let him fall into that trap.

Yes, ethics!

"The technical skills relevant for today are not going to be relevant even two years from now. It really is more soft skills—critical thinking, adaptability. Also ethics—where human judgment comes into play."

Unlocking Hope: Access to Therapies for People with Rare, Progressive, and Serious Diseases | United States Senate Special Committee on Aging United States Senate Special Committee on Aging

More here: www.aging.senate.gov/hearings/unl...

Predictable outcome but important nonetheless.

Market access to unproven drugs can make it much harder to figure out if they (and other drugs) work.

Patient desires for access and willingness to tolerate risk and uncertainty are understandable and reasonable. Yet there are critical tradeoffs.

The bill would allow conditional approval based on weak early evidence of effectiveness (distinct from existing accelerated approval). A survey of cancer pts/survivors found they would be significantly less willing to enroll in trials if there was a conditionally approved drug available.

The Proposed Drug Conditional Approval Pathway and Cancer Survivors’ Responses This survey study assesses understanding of and responses to the proposed US Food and Drug Administration conditional drug approval pathway among individuals who have received prior treatment for canc...

Very interesting survey results re: a bill I've tracked for several years, the Promising Pathway Act.

jamanetwork.com/journals/jam...

@gregggonsalves.bsky.social @reshmagar.bsky.social @alsadvocacy.bsky.social

Announcing the Retraction Watch Research Accountability Reporting Fellowship - The Open Notebook The Open Notebook and Retraction Watch are thrilled to announce a new fellowship program funded by The Center for Scientific Integrity. This six-month program will equip up to six reporters and editor...

Very cool: a new six-month fellowship from @theopennotebook.bsky.social and @retractionwatch.com for reporters and editors at local newsrooms to report stories of scientific integrity unfolding at research universities or institutions in their areas. www.theopennotebook.com/2026/02/26/a...

Ole Skøtt, U. of Southern Denmark's health sciences dean, tells @rollingstone.com: “Your enquiry contains new information in relation to the validity of the ethical approval. I have decided this should be investigated. The Hepatitis B project has been put on hold to assess the new information." /6

The Promise and Perils of FDA’s New ‘Plausible Mechanism’ Pathway With Holly Fernandez Lynch, JD, MBe Podcast Episode · Research Ethics Reimagined · February 20 · 50m

On Monday, FDA is slated to announce draft guidance on its plausible mechanism pathway for individualized therapeutics. Here's a podcast (w/ me from @weareprimr.bsky.social) to get you up to speed - why the pathway is needed, what's great about it + some concerns.
podcasts.apple.com/us/podcast/t...

CNN's Kristen Holmes reports that Trump blew up during a meeting after learning of the SCOTUS ruling on tariffs.

"He started ranting about the decision, not only calling it a disgrace, but started attacking the courts and at one point saying these effing courts, but using the actual language there"

So the Court's decision is not likely to greatly restrict Presidential tariff authority going forward. But the Court's decision is likely to generate other serious practical consequences in the near term. One issue will be refunds. Refunds of billions of dollars would have significant consequences for the U.S. Treasury. The Court says nothing today about whether, and if so how, the Government should go about returning the billions of dollars that it has collected from importers. But that process is likely to be a "mess," as was acknowledged at oral argument. Tr. of Oral Arg. 153-155. A second issue is the decision's effect on the current trade deals. Because IEEPA tariffs have helped facilitate trade deals worth trillions of dollars—including with foreign nations from China to the United Kingdom to Japan, the Court's decision could generate uncertainty regarding various trade agreements. That process, too, could be difficult.

Kavanaugh is attempting to manufacture the legal doctrine of pointus de noreturnis: If the regime’s unlawful actions are too difficult to unwind, we apparently have no choice but to see them through.

Eric Dane, McSteamy on ‘Grey’s Anatomy,’ Dies at 53

This is so sad. He was only 53. ALS is such a cruel disease.

Eric Dane, McSteamy on ‘Grey’s Anatomy,’ Dies at 53 www.nytimes.com/2026/02/19/a...

Today's the deadline for abstract submission. Come join the health law profs (broadly construed!) at Georgia State June 3-5. See you there!

Biotech investor set to lead US National Science Foundation If confirmed by the US Senate, Jim O'Neill would be the first non-scientist or engineer to lead the agency, a big funder of US basic research.

O'Neill, an investor in anti-aging technology who worked on HHS policy, would be the first non-scientist or engineer to lead NSF. His background will be a 'major concern' to the science and technology community, former NSF director Neal Lane told me.

@maxkozlov.bsky.social and I report: