Chairman Scott, Ranking Member Gillibrand to Examine FDA Regulatory Processes, Rare Disease Treatment Delays, and the Need to Improve Patient Access to Innovation

WASHINGTON, D.C. - On Thursday, February 26, Chairman Rick Scott and Ranking Member Kirsten Gillibrand will convene a hearing entitled "From Regulator to Roadblock: How FDA Bureaucracy Stifles Innovation." This hearing will explore how regulatory processes and evolving standards at the Food and Drug Administration (FDA) can unintentionally delay patient access to safe and effective therapies, particularly for individuals living with rare diseases. Members and expert witnesses will share the life-altering impacts of these delays on patients and their families. The hearing will also explore opportunities to improve regulatory clarity and predictability, discuss how an efficient review process that puts patients first can strengthen U.S. biomedical leadership and ensure that lifesaving therapies reach patients faster.

This hearing follows a November 2025 bipartisan letter to FDA Commissioner Dr. Martin Makary expressing the committee's interest in understanding how the agency is supporting innovation in rare disease drug development and increasing the speed and number of rare disease approvals. Chairman Scott and Ranking Member Gillibrand are committed to ensuring that the FDA fulfills its mission to protect patients while fostering innovation and maintaining America's leadership in biomedical research.

Witnesses for the hearing will include:

• Annie Kennedy, Chief Mission Officer, EveryLife Foundation for Rare Diseases, Washington, D.C.

• Jeremy Schmahmann, M.D., Director, Massachusetts General Hospital Ataxia Center, Boston MA

• Bradley Campbell, President & Chief Executive Officer, Amicus Therapeutics, Princeton, NJ

• Dr. Cara O'Neill, M.D., FAAP, Chief Science Officer, Co-Founder, Cure Sanfilippo Foundation, Columbia, SC

WHEN: Thursday, February 26, 2026

9:30AM ET

WHERE: Hart Senate Office Building, SH-216

WATCH: Live broadcast online HERE.

NOTE: Chairman Scott and Ranking Member Gillibrand invite members of the public, patient organizations, researchers, and other stakeholders to submit statements for the record. Written submissions must not include personal identifying information and should be emailed to: RareDiseaseStatements@Aging.Senate.Gov. Please note, all statements must be received by 5:00PM ET Thursday, March 5, 2026, to be included in the official hearing record.

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