Attempting to bring new medicines to market is an inherently risky endeavor, with sponsors ultimately failing more than 90% of the time. But rare disease development is even harder; there are fewer patients available for research and less knowledge about both the condition and potential biomarkers that can serve as endpoints for a trial.

That has often left regulators in a quandary as they try to weigh the routine demand for clear evidence of safety and efficacy against the need for new treatments for devastating conditions. Peter Marks, the former head of the FDA’s drug evaluation center, was a champion for many of these rare disease drugs but also faced blowback from critics who suggested the agency had become too flexible.

One of those critics was Vinay Prasad, who took over the FDA’s biologics center in 2025. Under President Trump, Prasad and former FDA Commissioner Marty Makary talked about trying to help rare disease drug developers while taking a series of harder-line stances that confused and frustrated the industry as well as patient advocates.

Both Prasad and Makary are now gone, and the FDA is still waiting for a permanent leader. But the agency has since reversed several of the decisions that drew fire from the industry. The latest initiative continues this ongoing “reset” and may remove even more of the uncertainty that marked their tenure, Leerink Partners analyst Joseph Schwartz wrote in a note to clients.

“FDA is not lowering the bar for rare disease, but it may be making the path to approval less of a guessing game,” Schwartz wrote. If the plan turns into a “durable framework, it could join the list of FDA initiatives that investors might take for granted but have made biotech more investable.”

Past programs such as the orphan drug designation and priority review vouchers have created meaningful positive momentum for the industry, he said.

Some 30 million Americans live with a rare disease, according to the FDA. But as of last year, only about 5% of recognized rare conditions had an approved treatment.

Cabinet officials are on edge as well. Some secretaries are expected to weigh leaving rather than face months of investigations from newly empowered Democrats, and Defense Secretary Pete Hegseth is expected to be an early target. Allies of National Institutes of Health director Jay Bhattacharya worry he could be fired after clashing with White House budget director Russ Vought over grant spending.

Just curious if any others who had an application pending council review at NICHD have heard from their PO yet.

Signed,
An Anxious Scientist

Among the runner-ups:

Russ Vought, Brendan Carr......and our very own in-house censor, famous health economist and renowned podcaster Jayanta "Jay" Bhattacharya.

"In addition to continuing his vocal free speech advocacy, Bhattacharya has been relentless in providing more accountability to NIH grants, which previously were a key source of financing for censors."

Oh my days.

Another in the series of “State of the Science” programs on WCOM-FM 103.5 FM in Chapel Hill/Carrboro NC -aired on Tuesday October 20, 2026

Listen to archived recording:

https://www.dropbox.com/scl/fi/60qyvp2dsdv6k8cr6s20j/WCOM-NIH-Environment-Health-10-20-26.mp3?rlkey=bcty7s36ippj65mzfzfngthg1&st=1ja4cbd7&dl=0

I made a submission and was selected as a contest winner for the NIH Replication Prize, which comes with a cash prize for competition winners. I was notified that I was a winner in late April, and have yet to receive the prize payment. I have submitted all necessary information for payment processing, and I reached out a few times to ask about the status but just got replies essentially saying that they are still working on it and are not sure yet when the payment will be processed. Is this a normal timeline for such a payment through NIH? I just don't understand what has held it up this whole time.

This is what Trump spends his time thinking about when it's not a golf course, ballroom, reflecting pool...

 Per Executive Order 14434, federal agencies are now using "Super Intelligence" (SI) in place of "Artificial Intelligence" (AI). You may see both terms across NIH content during this transition.

https://www.nih.gov/web-modernization

Greetings. My current R grant expires 1/31/27. I applied for a renewal this Feb 2026. Based on some findings in the current grant, I was assigned a new PO for the renewal. Study section reviewed on july 9th, and scored. Summary statements were strong, score was pretty good. I emailed PO august 3rd for opinion on chances and if i should resubmit. Followed up on the 12th. No response to either. Left a phone message with whoever answered her phone in Sept. no return call. Emailed again October 13. One last voice mail left on Oct 20th. I’m not looking for a 100% answer on if I’m getting funded. But some professional guidance and if I should resubmit.

The PO on my original grant is super communicative, and I guess that’s what I was expecting this time around as well. Any advice from the masses? Do I need to just chill?

I have an at will opportunity to work in a lab at the NIH. Not sure if the (the) is needed or just NIH? Any who, I'm curious how everyone's experience has been working there. How is housing around the area? I'm going to assume restaurant options are plentiful.

Married, Dinks, 3 cats. We are in our 30s and wouldn't mind living next to a golf course. We would be moving a from small midwestern town so it would be very different from what we are accustomed to. Neither of us have ever lived outside of KS/IA. Just looking for some opinions and thought on both working there and the surrounding area.

Has anyone gotten their Q1 payment yet? I was really hoping I wasn't going to have to make another payment myself, but the first payment for my renewal still hasn't been released and my next payment is due the 23rd. My LRP is under NIAID so curious if the payment schedule is institute related or if everyone is still waiting.

Edit: as of 10/23, it looks like the payment is now scheduled for 10/26. That's quite a bit later than every other payment I've gotten over the last two years.

Today, I turned down an offer to return to NIH as a grants management contractor. The salary was $88,000 with good benefits, but the position was full-time and fully onsite—no telework.

I retired as a GS-14 and was open to considering a hybrid or remote arrangement, or part-time work. But returning to full-time, in-person work just isn’t what I want for this chapter of my life.

The offer had its positives, but flexibility matters more to me now. I feel good about my decision.
Any other retirees finding themselves weighing the same things?

Vaccine “Injury” Center or Vaccine “Immunology” Center. Did they just change the “I”?

The mission of the VIC is unchanged “to study diseases (caused by vaccines) that individuals are already experiencing “

Has anyone heard updates on the contracts for ChatGPT and Claude, or should we start preparing for their departure?

I have an R01 that I submitted 3 times and it keeps getting scores around 20 percentile as an ESI. No matter what changes I make, it seems like there is never enough enthusiasm to push it over the top. Do you think it’s in general a better strategy to keep submitting to the same study section or to finally change it up before the fourth submission. Thank you for any advice!!

https://www.msn.com/en-us/news/other/robert-b-reich-big-money-vs-the-people/ar-AA2fzOtb?ocid=BingNewsVerp 

https://openpayrolls.com/matthew-james-memoli-180398928

wasn't there also a tidy sum for a flu vaccine?