Amyotrophic Lateral Sclerosis (ALS) Intermediate Patient Population Expanded Access (U01 Clinical Trial Required) | RFA NS 25 024

Opportunity Information: Apply for RFA NS 25 024

This funding opportunity (RFA-NS-25-024) is a National Institutes of Health (NIH) cooperative agreement (U01) led by the National Institute of Neurological Disorders and Stroke (NINDS) in partnership with the NIH Office of the Director. Its core purpose is to support research that uses data generated through expanded access (EA) to investigational drugs or biologics for people living with amyotrophic lateral sclerosis (ALS). The emphasis is specifically on an "intermediate-size" patient population: individuals who do not qualify for traditional clinical trials, but who may be able to receive an investigational product through an intermediate expanded access protocol. In practical terms, the program is trying to create a structured, research-ready way to learn from expanded access use in ALS, rather than treating EA as purely compassionate use with limited systematic data collection.

A central requirement of the program is that providing an investigational drug or biologic under this intermediate EA approach must not disrupt or slow down the clinical development pathway that could lead to FDA marketing approval. NIH is signaling that expanded access must be conducted in a way that does not interfere with initiation, enrollment, conduct, or completion of the pivotal trials (or other studies) needed to establish safety and effectiveness. The goal is to strike a balance: broaden access for patients who lack trial options while protecting the integrity and feasibility of the clinical investigations that ultimately determine whether a therapy becomes widely available.

Eligibility is relatively specific. The intended applicants are clinical trial sites that are already participating in a Phase 3 clinical trial for an ALS investigational product. That Phase 3 trial must be supported by a U.S. small business, and that small business must also be the FDA-designated sponsor of the investigational drug or biologic and hold the relevant Investigational New Drug (IND) application. This structure effectively ties the expanded access research to a product that is already in late-stage testing and to an established sponsor responsible for regulatory oversight, safety reporting, and IND obligations. The notice also indicates NIH wanted to give potential applicants time to build meaningful collaborations, which matters here because these projects require close coordination among clinical sites, the product sponsor, regulatory teams, and data management groups.

The mechanism is a U01 cooperative agreement, which typically means NIH expects substantial scientific and programmatic involvement during the project, not just a hands-off grant. While the text does not spell out the full set of required activities, the intent is clear: applicants would be expected to implement or operate an intermediate expanded access protocol for ALS and simultaneously conduct scientific research using the data produced through that access. That often implies more formalized data collection than is typical in ad hoc compassionate use, such as consistent clinical outcome measures, safety monitoring, standardized case report forms, and plans for data quality and analysis that can produce interpretable evidence (while still recognizing that EA is not the same as a randomized controlled trial).

From an applicant-type standpoint, the opportunity is listed under NIH with a health focus (CFDA 93.853) and is categorized as discretionary funding using a cooperative agreement instrument. The eligibility list in the source data is broad and includes many domestic entity types, such as state and local governments, public and private institutions of higher education, nonprofits (with or without 501(c)(3) status), for-profit organizations (including but not limited to small businesses), public housing authorities, and Native American tribal governments and organizations. It also explicitly mentions certain institution types (for example, HBCUs, Hispanic-serving institutions, tribally controlled colleges and universities, Alaska Native and Native Hawaiian serving institutions, and AANAPISISs), as well as faith-based and community-based organizations and U.S. territories or possessions. However, foreign organizations are not eligible to apply, and foreign components of U.S. organizations are not allowed, consistent with NIH restrictions stated in the notice.

Key administrative details from the listing include the funding opportunity title "Amyotrophic Lateral Sclerosis (ALS) Intermediate Patient Population Expanded Access (U01 Clinical Trial Required)," the funding opportunity number RFA-NS-25-024, and the NIH as the issuing agency. The original closing date in the provided source data is 2025-01-24, and the record shows a creation date of 2024-09-26. The award ceiling and expected number of awards are not specified in the excerpt you provided, so applicants would need to consult the full FOA package for budget limits, project period expectations, and the anticipated number of funded sites or awards.

Overall, this opportunity is aimed at building a careful bridge between patient need and product development in ALS: enabling access for patients who cannot join trials, while producing usable, responsibly collected data and ensuring expanded access does not undermine the clinical trials needed for eventual regulatory 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 2024-09-26.
• Applicants must submit their applications by 2025-01-24. (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 25 024

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