The National Institutes of Health and the Food and Drug Administration awarded roughly $276 million from fiscal years 2022 through 2025 to implement the Accelerating Access to Critical Therapies for ALS Act, according to a Government Accountability Office report published as GAO-26-107691. About 750 individuals with ALS are expected to gain access to investigational drugs through NIH grants funded under the act.

The spending splits into three nearly equal tranches, with one small exception. NIH grants supporting access to investigational drugs — therapies not yet FDA-approved for marketing — plus related research accounted for $125.3 million, or 45 percent of the total. NIH and FDA funding for a public-private partnership focused on rare neurodegenerative disease research totaled $124.4 million, also 45 percent. FDA grants and contracts for research on ALS and other rare neurodegenerative diseases, including clinical development of therapies, made up the remaining $26.5 million, or 10 percent.

The year-by-year distribution shows how the program ramped up. Total awards climbed from $29.4 million in fiscal 2022 to $79.9 million in fiscal 2023, then $80.7 million in fiscal 2024 and $86.2 million in fiscal 2025. The fiscal 2022 figure is the outlier: appropriations were not available until midway through that fiscal year, leaving applicants limited time to respond to NIH grant solicitations. NIH subsequently changed practice, posting requests for grant applications before appropriations were enacted in later years, agency officials told GAO.

The NIH access grants grew from $18.1 million in fiscal 2022 to a peak of $39.2 million in fiscal 2024, then dipped to $35.4 million in fiscal 2025. Partnership funding jumped from $5.5 million in fiscal 2022 to $42.3 million the following year and stayed in the high-$30-million-to-$40-million range afterward. FDA's research grants and contracts doubled from $5.1 million in fiscal 2023 to $10.1 million in fiscal 2025.

For research managers, the audit matters for more than the topline. GAO notes that the awarded funds include money Congress appropriated specifically to implement the act as well as funds FDA identified from other sources. Totals exclude administrative costs and may not sum exactly because of rounding. One structural gap persists: agency officials said FDA received no direct appropriations to support its priorities for other rare neurodegenerative diseases within the public-private partnership — a constraint outside the agencies' control.

What the money has produced so far is only partially measurable. Stakeholder interviews, available literature and NIH data indicate benefits such as increases in the number and geographic diversity of clinic sites providing access to ALS investigational drugs. That geographic spread directly affects trial-eligibility questions for a disease where delayed diagnosis can disqualify patients from clinical trials, since ALS has no cure and limited treatment options. Physicians or drug sponsors can request FDA authorization to make investigational drugs available outside of trials through the expanded access pathway.

GAO draws a clear line between observed and anticipated results. Most of the funded research is ongoing, and the full effects are not yet known, the report states. Anticipated benefits include increased data on ALS and progress on research gaps for ALS and other rare neurodegenerative diseases. Stakeholders expect the data generated to be useful to the wider ALS research community because it will meet high data-quality standards and be available to other researchers.

The evidence base has limits GAO is explicit about. The agency reviewed laws, congressional reports, agency documentation, and grant and contract applications and progress reports, and analyzed NIH database records on funded studies as of November 2025. Its stakeholder sample of 21 respondents — national associations, grant recipients, drug sponsors, clinic sites, partnership entities, and a committee of individuals with ALS and caregivers — was nongeneralizable by design, selected to vary perspectives and organization types rather than to support statistical inference. GAO also reviewed relevant research published from January 2019 through September 2025.

Congress mandated the report in the act itself, which directs GAO to track NIH and FDA funding, implementation challenges, and effects on therapy R&D for ALS and other rare neurodegenerative diseases. The audit closes on an unresolved note: with most funded research still running, the next round of reporting will determine whether the $276 million investment translates into durable data assets and, ultimately, approved therapies.