Trump Signs ALS Law Requiring FDA Rare Disease Action Plan

President Trump 11/3 signed into law legislation extending federal programs supporting access to investigational amyotrophic lateral sclerosis (ALS) treatments through fiscal 2031 and requiring FDA to develop a new five-year action plan for rare neurodegenerative diseases.

The bipartisan Accelerating Access to Critical Therapies for ALS Reauthorization Act of 2026, HR 8205, reauthorizes programs established under the original 2021 law to accelerate ALS drug development and provide investigational therapies to patients who cannot participate in clinical trials.

The legislation instructs FDA to publish an action plan within 18 months outlining regulatory policy initiatives, program enhancements and regulatory science activities the agency intends to undertake over the next five years. FDA must also publish a report within five years of enactment describing its implementation of the plan.

The law strengthens oversight of grants supporting expanded access to investigational ALS drugs. In reviewing grant renewal applications, HHS must request and assess patient enrollment, safety and available efficacy data from drug manufacturers or sponsors.

Grant recipients also must promptly report new serious adverse events and unexpected safety information associated with Phase 3 trials, in addition to existing FDA safety reporting obligations. The legislation clarifies that certain combined Phase 2/3 trials qualify under the program.

A February Government Accountability Office report found that FDA and NIH awarded approximately $276 million under the original law during fiscal 2022–2025, including $125 million for expanded access grants. About 750 ALS patients were expected to receive investigational treatments through those grants.

The ALS Association welcomed the reauthorization, saying it would preserve treatment access and research programs through 2031.