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# New CBER Head Should Restore Rare Disease Clarity: Advocates
- URL: https://www.fdaweb.com/new-cber-head-should-restore-rare-disease-clarity-advocates/
- Published: 2026-04-01T12:00:00.000Z
- Updated: 2026-09-14T13:36:37.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5160911

The Rare Disease Advocacy, Biotechnology, and Investor Coalition, which has nearly 100 rare disease patient advocacy groups, biotechnology executives, and investors as members, is urging the Trump administration to use the appointment of a new CBER director to restore regulatory clarity for rare diseases. The new appointee will replace **Vinay Prasad**, who is leaving the agency at the end of April to return to his academic appointment.

Reuters [reports](https://www.usnews.com/news/top-news/articles/2026-04-01/rare-disease-advocacy-group-urges-trump-administration-to-restore-fda-clarity?ref=fdaweb.com) that Prasad’s two stints as Center director have been marked by high-profile disputes over reviews of vaccines, including Moderna’s mRNA Covid-19 shot, gene therapies such as uniQure’s therapy for Huntington’s disease, and other rare-disease drugs.

In a letter to president **Donald Trump**, FDA commissioner **Marty Makary**, HHS secretary **Robert F. Kennedy Jr.**, and Centers for Medicare and Medicaid Services director **Mehmet Oz**, the coalition reportedly said CBER has become less flexible in overseeing rare disease clinical trials under Prasad. The news service says the group pointed out that 84% of biotech investors they surveyed had reduced, paused, or exited rare disease investments because of recent FDA uncertainty. Some two-thirds of the biotech companies surveyed reported the uncertainty had made it harder to raise capital over the past 12 months.

“We believe it is of the utmost importance that 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.

Reuters reports that CBER approved five orphan drugs in 2025, while issuing four Complete Response Letters (CRLs) 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, it says, CBER approved one orphan drug and issued two CRLs, compared to eight approvals and two CRLs at CDER in the same period.