Rare disease advocacy group urges Trump administration to restore FDA clarity
A rare disease advocacy coalition urged the Trump administration to restore regulatory clarity as leadership changes approach at the U.S. Food and Drug Administration’s Center for Biologics Evaluation and Research.
Reuters reported that a rare disease coalition called on the Trump administration to restore FDA clarity as the agency considers new leadership at the biologics review center.
Key facts
- A rare disease advocacy coalition urged the Trump administration to restore regulatory clarity at the FDA.
- The appeal comes as leadership is expected to change at the FDA’s Center for Biologics Evaluation and Research.
- The coalition said 84% of surveyed biotech investors had reduced, paused or exited rare disease investments because of uncertainty.
- About two-thirds of surveyed biotech companies said the uncertainty made it harder to raise capital over the past 12 months.
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.
Coalition says FDA uncertainty is damaging rare disease investment
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. In practical terms, the coalition is arguing that regulatory ambiguity is not just a policy concern, but also a financial and development challenge that may directly affect whether rare disease programs can move forward.
“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.
Rare disease group calls for a leader who understands patient needs
The letter places clear emphasis on the need for leadership at the FDA’s biologics center that understands the realities of rare disease drug development. Because rare disease clinical trials often involve small patient populations, complex endpoints and limited natural history data, advocacy groups and biotech companies have long argued that regulatory flexibility is essential.
By pressing for “clarity,” the coalition is effectively calling for more predictable review standards, better communication with sponsors and a regulatory approach that recognizes the distinctive challenges facing rare disease treatments, gene therapies and other specialized biologic programs.
Approval and rejection trends add to pressure on CBER leadership
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.
Those figures help explain why leadership at the Center for Biologics Evaluation and Research has become a major issue for rare disease investors, patient organizations and drug developers. For the coalition, the concern is not only the number of approvals or rejections, but the broader perception that regulatory expectations have become harder to interpret during a critical period for capital formation and pipeline development in rare disease biotech.
