Chairman Rick Scott led a Senate Special Committee on Aging hearing titled 'From Regulator to Roadblock:
How FDA Bureaucracy Stifles Innovation' to examine how FDA regulatory processes delay patient access to rare disease therapies2
The FDA denied approval in November 2025 for Biohaven's troriluzole for spinocerebellar ataxia (SCA), a progressive genetic disease with no existing treatments, despite the drug winning on its primary and eight secondary endpoints in its priority review study1
Senator Kirsten Gillibrand stated that Congressional mechanisms like accelerated approval and real-world evidence use are 'not working how it should be,' citing a pattern of FDA hesitation, limited sponsor communication, and last-minute regulatory position shifts1
FDA advisory committee meetings dropped 65% from 2024 to 2025, reducing opportunities for external expertise and patient input on rare disease product decisions1
Approximately 1 in 10 Americans live with a rare disease, and senators emphasized that rare diseases can progress rapidly, causing irreversible harm and premature death1
Witnesses testified that patients currently stable on experimental drugs face removal from treatment, with one ataxia specialist calling proposed FDA alternatives 'unethical' and suggesting patients on placebo 'will die'1
The hearing examined regulatory clarity, predictability, and efficiency to strengthen America's position as a biomedical leader while maintaining safety standards2
Sources:
1. https://www.fiercebiotech.com/biotech/talking-brick-wall-senate-hearing-takes-aim-fdas-rare-disease-review-process
2. https://www.aging.senate.gov/press-releases/chairman-rick-scott-addresses-regulatory-hurdles-delaying-rare-disease-treatments-and-limiting-patient-access-to-innovation