Amyotrophic Lateral Sclerosis (ALS) Intermediate Patient Population Expanded Access (U01 Clinical Trial Required) | RFA NS 24 029

Opportunity Information: Apply for RFA NS 24 029

This NIH funding opportunity (RFA-NS-24-029) supports research that leverages data generated when patients with amyotrophic lateral sclerosis (ALS) receive investigational drugs or biologics through expanded access (EA), specifically under an intermediate-size patient population EA protocol. The core idea is to study outcomes, safety signals, and other clinically meaningful information from ALS patients who cannot enroll in traditional clinical trials, while still generating useful scientific evidence that can inform ALS drug development and patient care. The program is run by the National Institute of Neurological Disorders and Stroke (NINDS) in partnership with the NIH Office of the Director (OD), and it uses the U01 cooperative agreement mechanism, meaning awardees should expect substantial NIH scientific and programmatic involvement compared to a typical research grant.

A central requirement is that the expanded access activity and the resulting research must not undermine or slow down the standard clinical development pathway. In practical terms, the investigational product being provided under intermediate EA cannot interfere with the start, conduct, or completion of clinical trials intended to support FDA marketing approval, and it cannot compromise broader development of the product for ALS. This reflects FDA and NIH priorities: expanded access can provide an option for patients who lack trial eligibility, but it should not become a substitute for well-controlled studies or create operational, enrollment, or data-integrity problems that weaken the evidence needed for approval.

Eligibility is more specific than many NIH opportunities. The intended applicants are clinical trial sites that are already participating in a phase 3 clinical trial for ALS that is supported by a U.S. small business, where that small business is also the FDA-designated sponsor for the investigational drug or biologic and holds the relevant Investigational New Drug (IND) application. In other words, the opportunity is designed to connect an established phase 3 development program (run by a U.S. small business sponsor under an IND) with clinical sites that can also implement an intermediate-size expanded access protocol and conduct research using the data produced through that pathway. While the listing includes a wide range of organizational types that can be eligible for NIH grants (state and local governments, universities, nonprofits, for-profits, small businesses, tribal entities, and others), the practical eligibility for this specific FOA hinges on being a qualifying phase 3 trial site aligned with an eligible U.S. small business sponsor that is the FDA sponsor of the IND product in ALS.

The patient population focus is also explicit: intermediate-size cohorts of people living with ALS who are not otherwise eligible for clinical trials. That typically means patients who fall outside protocol inclusion criteria due to disease stage, comorbidities, prior treatments, geographic constraints, or other clinical and logistical reasons. The research supported under the FOA is intended to use data from providing the investigational product via EA in this intermediate group, which sits between single-patient (individual) expanded access and large-scale treatment protocols. The goal is to responsibly broaden access while learning as much as possible from real-world use under a structured protocol, such as characterizing safety in a broader ALS population, understanding feasibility of administration and monitoring, and exploring outcomes that may be hard to capture in tightly controlled trials.

Administratively, this is a discretionary health funding opportunity under CFDA 93.853, with the NIH as the issuing agency. The funding instrument is a cooperative agreement (U01), signaling closer coordination with NIH on milestones, data elements, oversight, and execution. The listing shows an original closing date of 2024-02-22. The notice text also references an earlier planning timeline (FOA anticipated in May 2022 with applications due June 2022), which appears to be legacy language from the pre-announcement; the authoritative dates for applicants should be taken from the posted FOA and the active closing date in the listing. Foreign organizations are not eligible to apply, and foreign components of U.S. organizations are not allowed, consistent with the stated restriction that non-domestic entities and foreign components (as defined by NIH policy) are excluded.

Overall, the opportunity is aimed at a very specific intersection: ALS expanded access programs run in coordination with an active phase 3 development effort, with the dual purpose of enabling access for trial-ineligible patients and producing analyzable data that advances scientific understanding without disrupting the evidence-generation needed for potential FDA approval.

• The National Institutes of Health in the health sector is offering a public funding opportunity titled "Amyotrophic Lateral Sclerosis (ALS) Intermediate Patient Population Expanded Access (U01 Clinical Trial Required)" and is now available to receive applicants.
• Interested and eligible applicants and submit their applications by referencing the CFDA number(s): 93.853.
• This funding opportunity was created on 2023-11-21.
• Applicants must submit their applications by 2024-02-22. (Agency may still review applications by suitable applicants for the remaining/unused allocated funding in 2026.)
• Eligible applicants include: State governments, County governments, City or township governments, Special district governments, Independent school districts, Public and State controlled institutions of higher education, Native American tribal governments (Federally recognized), Public housing authorities/Indian housing authorities, Native American tribal organizations (other than Federally recognized tribal governments), Nonprofits having a 501 (c) (3) status with the IRS, other than institutions of higher education, Nonprofits that do not have a 501 (c) (3) status with the IRS, other than institutions of higher education, Private institutions of higher education, For-profit organizations other than small businesses, Small businesses, Others.

Apply for RFA NS 24 029

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