Technical Assistance Webinar RFA-NS-26-001: NINDS ALS Expanded Access Program

Why It Matters

By linking expanded‑access treatment with structured research, the RFA accelerates data collection on investigational ALS therapies, potentially shortening the time to regulatory approval and expanding options for patients lacking trial eligibility.

Key Takeaways

• RFA NS-26-001 funds ALS expanded‑access for intermediate‑size patient groups.
• Applications due June 8 2026; second deadline November 10 2026 pending reauthorization.
• Applicants must be clinical sites in Phase 2/3 trials by US small businesses.
• FDA “may proceed” letter required before NIH funds; IND submission same day.
• Cooperative agreements include NIH program scientist partnership and biospecimen repository support.

Summary

NIH’s NINDS held a technical assistance webinar to explain RFA‑NS‑26‑001, the ALS Expanded Access program authorized under the 2021 ACT for ALS law. The RFA targets an intermediate‑size patient population—more than one individual but fewer than a typical treatment IND—and provides grant funding to clinical sites that can deliver investigational drugs while generating research data.

Key dates include a June 8 2026 application deadline with a July peer‑review and a September 2026 start, plus a second November 10 2026 deadline contingent on ACT for ALS reauthorization. Eligible applicants must be clinical trial sites participating in Phase 2/3 efficacy studies sponsored by a U.S. small‑business, and the investigational product cannot already have an NDA or BLA. Applicants must submit an FDA “may proceed” letter and an IND on the same day as the grant application, and obtain IRB approval before patient treatment begins.

Dr. Amy Tsou emphasized that the program is a re‑issue of the original ACT for ALS funding, noting that five studies have been funded since FY 2022. The webinar introduced competitive renewal applications, which must propose distinct scientific aims and report progress toward regulatory approval. NIH will award the grants as cooperative agreements, assigning a program scientist to the primary leadership committee and providing access to NINDS biospecimen repositories and the ALS Knowledge Portal for data sharing.

The initiative creates a pathway for ALS patients who are ineligible for standard trials to receive potentially life‑saving therapies while contributing valuable safety, biomarker, and patient‑experience data. For sponsors and sites, the program offers NIH partnership, funding, and infrastructure support that could accelerate drug development and improve the evidentiary base for future ALS approvals.

Original Description

Chapters:

0:00 Introduction

1:02 Purpose and Background

2:59 Key Requirements and Considerations

5:23 Eligibility Criteria

6:46 Additional Eligibility Information

7:36 Competitive Renewals

9:29 How to Apply- Resources

9:57 How to Apply- Tips

10:30 Peer Review of Applications

11:28 Cooperative Agreement Awards

12:33 Cooperative Agreement Awards- Roles and Responsibilities

14:26 Sharing of Biospecimens and Data

16:00 Conclusion