April 1 - A coalition of rare disease patient advocates, biotechnology executives and investors delivered a letter on Wednesday calling on the Trump administration to restore clarity at the U.S. Food and Drug Administration's Center for Biologics Evaluation and Research (CBER) as the agency prepares for a leadership change.
The Rare Disease Advocacy, Biotechnology, and Investor Coalition wrote 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 group, which said it represents nearly 100 rare disease patient advocacy organizations along with biotech executives and investors, argued that CBER has grown less flexible in its approach to overseeing clinical trials for rare conditions.
Dr. Vinay Prasad, who currently leads CBER, is scheduled to leave the FDA at the end of April. His tenure has been marked by public disputes over regulatory reviews for vaccines, including Moderna's COVID-19 shot, and for gene therapies such as uniQure's experimental treatment for Huntington's disease, as well as other drugs for rare conditions.
In their letter the coalition made a direct appeal about the kind of leadership they consider necessary for the center. "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 wrote.
The group also cited survey findings it said reflect growing market reactions to regulatory uncertainty. According to the coalition, 84% of biotech investors surveyed by the organization reported they had reduced, paused or exited investments in rare disease programs because of recent uncertainty at the agency. Roughly two-thirds of biotech companies surveyed said that the uncertainty had made it more difficult to raise capital over the past 12 months.
The coalition pointed to recent review outcomes at CBER as part of its case. In 2025, CBER approved five orphan drugs, the coalition noted, while issuing four Complete Response Letters (CRLs) and one comparable setback at the pre-marketing application stage - a rejection rate that the coalition described as about half of late-stage programs. That compares with a lower CRL rate cited for the prior two-year period, when the agency issued one CRL among 20 programs.
In the first quarter of 2026, CBER approved one orphan drug and issued two CRLs. By comparison, the FDA's drug evaluation center approved eight drugs and issued two CRLs over the same quarter, according to figures the coalition referenced. The coalition included an explanatory note that Complete Response Letters are notifications sent by the FDA when the agency determines it will not approve an application in its current form.
The letter reflects longstanding tensions between developers of treatments for small patient populations - where trial designs and evidentiary standards can differ from larger indications - and regulators seeking to apply consistent standards. The coalition's appeal asks federal officials to prioritize leadership that acknowledges those distinctions and includes patient and physician perspectives in decision-making.
Separately, the article contained a promotional segment about investment tools for identifying opportunities in 2026, mentioning platforms described as combining institutional data and AI-powered insights to help investors. That material was presented as a general note on investment research rather than as reporting on FDA matters.
Summary: A broad coalition representing rare disease patients, biotech executives and investors has urged the Trump administration to appoint CBER leadership that will restore regulatory clarity and be sensitive to the special challenges of rare disease development. Survey results cited by the group show significant investor pullback and fundraising difficulties tied to recent uncertainty. The coalition highlighted recent approval and Complete Response Letter metrics at CBER as a reason for concern.