Chairman Rick Scott Addresses Regulatory Hurdles Delaying Rare Disease Treatments and Limiting Patient Access to Innovation |
| Chairman Rick Scott led a hearing entitled “From Regulator to Roadblock: How FDA Bureaucracy Stifles Innovation.” This hearing examined 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. This hearing also explored opportunities to improve regulatory clarity and predictability and discussed how an efficient review process that puts patients first can strengthen America’s place as a biomedical leader. The hearing followed a November 2025 bipartisan letter to FDA Commissioner Dr. Martin Makary, which expressed 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 is committed to ensuring that the FDA fulfills its mission to protect patients while fostering innovation and maintaining America’s leadership in biomedical research. Chairman Scott’s witnesses for today’s hearing included Annie Kennedy, the chief mission officer for the EveryLife Foundation for Rare Diseases; Jeremy Schmahmann, M.D., the director of the Massachusetts General Hospital Ataxia Center; Bradley Campbell, the chief executive officer at Amicus Therapeutics; and Dr. Cara O’Neill, M.D., FAAP, the chief science officer and co-founder of the Cure Sanfilippo Foundation. These witnesses highlighted the real-world impact these regulatory hurdles can cause on patients and how the federal government and the FDA can work to improve care and ensuring that lifesaving therapies reach patients faster and safer. NOTE: Chairman Scott invites 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. All statements must be received by 5:00PM ET Thursday, March 5, 2026, to be included in the official hearing record. Watch Chairman Scott's full remarks HERE or below. Read more HERE. Lea más AQUÍ. — The U.S. Senate Special Committee on Aging is committed to helping seniors and victims of fraud. If you or someone you know has been a victim of fraud, suspect you or someone you know have been targeted by a scammer, or have questions about common scams, please call the Fraud Hotline at 1-855-303-9470 (open weekdays from 9 a.m. to 5 p.m. Eastern Time) or fill out the web form HERE. If you are looking for materials to help you avoid frauds and scams, the most recent copy of the Committee’s Fraud Book is HERE. — To be the first to find out about the U.S. Senate Special Committee on Aging's actions and statements, follow on X. — |
