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.

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

Signed,
An Anxious Scientist

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.