Sponsors (104)
Tightens grant reporting and renewal review standards for ALS investigational drug grants and requires FDA to publish a 5‑year rare neurodegenerative disease action plan and later report.
Official title: To amend the Accelerating Access to Critical Therapies for ALS Act to reauthorize the provisions of such Act through fiscal year 2031, and for other purposes.
Introduced April 6, 2026 by Mike Quigley · Last progress April 6, 2026
The bill aims to speed and better-coordinate development and oversight of therapies for ALS and other rare neurodegenerative diseases through clearer authority, reporting, and FDA planning — at the cost of higher spending, more compliance burdens, and potential regulatory uncertainty if implementation falls short.
Patients with ALS and other rare neurodegenerative diseases gain clearer federal support and timelines—potentially faster access to therapies because HHS program authority and FDA planning are modernized and made more transparent.
Trial participants and the public receive stronger safety protections and faster awareness of problems — grant renewals require closer monitoring of investigational drug safety and prompt reporting of serious unexpected adverse events beyond current FDA-only reporting.
Researchers and drug developers gain predictability to align development plans — FDA's required 5-year plan and public reporting increases transparency about agency priorities and can accelerate clinical programs.
Taxpayers and other HHS programs may face higher costs because expanding or implementing new programs and FDA planning could require additional federal spending or reprioritization of resources.
Researchers, sponsors, hospitals, and FDA staff will face increased administrative and compliance burdens — new reporting, monitoring, and planning requirements may slow grant processing, raise costs, and divert agency staff time from reviews.
Patients and researchers could see fewer funding opportunities if amendments instead narrow grant authority or add restrictive conditions, reducing development support for ALS and rare-disease therapies.
Based on analysis of 4 sections of legislative text.
Requires updated grant program rules and reporting to speed development of drugs for ALS and other rare neurodegenerative diseases. It tightens grant reporting and renewal review standards for investigational ALS drug trials and directs the FDA to publish a five‑year "rare neurodegenerative disease action plan" within 18 months and a follow-up report within five years. The bill amends existing authority for grants and contracts under 21 U.S.C. § 360ee to require sponsors to provide enrollment, safety, and efficacy data for renewal reviews, to promptly report new serious unexpected adverse events, and to clarify that certain combined phase 2/3 trials can qualify as "phase 3" when they begin within grant-defined timeframes. It also adds required timebound planning and public reporting by the FDA Commissioner about actions on rare neurodegenerative disease regulatory and program priorities.