The Senate held a bipartisan hearing last month to examine why medications are not readily available to patients, particularly those with rare diseases, according to a March 11 statement. Eight days after the hearing, Vinay Prasad resigned as director of the Food and Drug Administration’s Center for Biologics Evaluation and Research.
The issue is significant because many in the industry believe that regulatory processes have hindered sponsors from bringing new therapies to market, especially for rare diseases. The resignation of Prasad is seen by some as an opportunity for needed changes in how these treatments are evaluated and approved.
During the Senate hearing, witnesses including industry leaders, physicians, advocates, and parents described inconsistencies in regulatory guidelines and underuse of available resources. They said these issues have led to delays or denials of treatment options for patients who already face limited choices. "As a healthcare communications professional with two decades of experience in regulatory strategy, I’ve watched sponsors accommodate FDA requests for trial design changes and new datasets. I’ve also seen the subsequent fallout as small biotechs try to figure out how to fund new studies, while patients, families and advocates wait without answers as to why," one participant said.
Testimony highlighted problems such as late-stage reversals on trial designs and endpoint agreements by regulators. Since early 2025, 23 Complete Response Letters have been issued to rare disease sponsors—many with little justification according to witnesses. Dr. Jeremy Schmahmann from Mass General recounted an incident where a regulatory official questioned why they should listen to a patient during a review meeting: "Why should I listen to you?" he quoted.
Witnesses argued that rare disease trials require different standards due to small patient populations and reliance on surrogate endpoints. They noted that Congress had previously created accelerated approval pathways and orphan drug incentives recognizing these challenges. However, at current rates of development and approval it could take over a century just to address half of known rare diseases; currently about 90 percent lack any FDA-approved treatment.
The hearing concluded with calls for four main reforms: establishing a dedicated Rare Disease Advisory Committee; consistently using accelerated approval pathways when criteria are met; issuing clear guidance on surrogate endpoints; and increasing transparency around late-stage review changes or rejections. No one asked the FDA to lower its standards—only that it apply existing tools more consistently under leadership familiar with rare disease science.
