Rare disease advocacy group urges Trump administration to restore FDA clarity

April 1 (Reuters) - A rare disease ‌advocacy coalition ​on Wednesday ​urged the Trump administration to restore regulatory clarity as it considers new leadership at the U.S. Food and Drug Administration's Center for ‌Biologics Evaluation and Research.

Dr. Vinay Prasad, the center's current incumbent, is ⁠set to leave the FDA at the end of April after a tenure marked by ‌high-profile disputes over reviews for ‌vaccines, including Moderna's COVID shot, gene therapies such as uniQure's therapy for Huntington's disease and other rare disease drugs.

The Rare Disease Advocacy, Biotechnology, and Investor ​Coalition sent the letter to President Donald Trump, U.S. Health Secretary Robert F. Kennedy Jr, Mehmet Oz, head of the U.S. Medicare agency and FDA ⁠Commissioner Marty Makary.

The coalition, which includes nearly 100 rare disease patient advocacy groups, biotech executives and investors, said ​CBER has become less flexible in overseeing rare disease clinical trials.

The group said 84% of biotech investors surveyed by RDBI ​had reduced, paused or exited rare disease investments ‌because of recent FDA uncertainty. About two-thirds of biotech companies surveyed said the uncertainty had made it harder to raise ⁠capital over the past 12 months.

"We believe it is of the utmost importance that the FDA chooses a leader who understands the unique challenges of rare disease development and ⁠respects and values the views of patients and physicians," the coalition said in the letter.

CBER approved ​five orphan drugs in 2025 while issuing four Complete Response Letters and one comparable setback at the pre-marketing application stage, rejecting about half of late-stage programs, compared with one CRL ‌among 20 programs over the prior two years.

In the first quarter of 2026, CBER approved one orphan drug and issued ‌two CRLs, compared with eight approvals and two CRLs at the FDA's drug evaluation ⁠center over the same period.

CRLs ‌are sent by the FDA ​if the agency determines it will not approve the application in its current form.

(Reporting by Sriparna Roy in Bengaluru; Editing by ‌Tasim Zahid)

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