Amyotrophic Lateral Sclerosis (ALS) Intermediate Patient Population Expanded Access (U01 Clinical Trial Required) | RFA NS 23 012

Opportunity Information: Apply for RFA NS 23 012

This funding opportunity, titled "Amyotrophic Lateral Sclerosis (ALS) Intermediate Patient Population Expanded Access (U01 Clinical Trial Required)" (RFA-NS-23-012), is an NIH initiative led by the National Institute of Neurological Disorders and Stroke (NINDS) in partnership with the NIH Office of the Director. Its central goal is to support scientific research that uses data generated when patients with ALS receive investigational drugs or biological products through expanded access (EA), specifically under an "intermediate-size patient population" EA protocol. In practical terms, it is aimed at people living with ALS who cannot enroll in traditional clinical trials, while still allowing structured access to an investigational therapy and enabling researchers to learn from the resulting clinical data.

A key feature of the program is the requirement that expanded access activities must not disrupt, delay, or undermine the standard clinical development pathway. The FOA emphasizes that providing an investigational product via an intermediate EA protocol cannot interfere with the initiation, conduct, or completion of clinical investigations that could support FDA marketing approval. In other words, the expanded access effort has to be designed so it complements ongoing development rather than competes with or compromises pivotal trials, data integrity, enrollment feasibility, or the overall prospects for eventual product approval.

The eligible applicant pool is specialized. The primary intended applicants are clinical trial sites that are already participating in a Phase 3 clinical trial for an ALS investigational product, where that Phase 3 trial is supported by a U.S. small business concern, and that small business is also the FDA-designated sponsor of the ALS drug or biologic under an active Investigational New Drug (IND) application. This structure signals that NIH wants these expanded access research efforts tightly aligned with late-stage development programs and coordinated with the IND sponsor, likely to ensure regulatory consistency, appropriate safety oversight, and well-defined data capture.

The award mechanism is a U01 cooperative agreement, which generally means NIH expects substantial scientific and/or programmatic involvement during the project (for example, coordination around study conduct, milestones, data standards, and oversight). The opportunity is categorized under Health (CFDA 93.853) and is listed as a discretionary funding opportunity. While the full list of potentially eligible applicant types in the source data includes a wide range of domestic entities (such as state and local governments, public and private institutions of higher education, nonprofits with and without 501(c)(3) status, for-profit organizations other than small businesses, and small businesses), the narrative description makes clear that, for responsiveness, applicants should fit the specific scenario of being a Phase 3 ALS trial site linked to a U.S. small business that is the FDA-designated IND sponsor for the product being provided through expanded access.

The FOA also highlights inclusion of certain institution types and organizations that NIH often calls out for eligibility and participation, including Alaska Native and Native Hawaiian Serving Institutions, Asian American Native American Pacific Islander Serving Institutions (AANAPISIs), Hispanic-serving Institutions, Historically Black Colleges and Universities (HBCUs), Tribally Controlled Colleges and Universities (TCCUs), faith-based or community-based organizations, eligible federal agencies, regional organizations, and U.S. territories or possessions. However, foreign participation is explicitly restricted: non-U.S. entities (foreign organizations/institutions) may not apply, non-U.S. components of U.S. organizations are not eligible, and foreign components (as defined by NIH policy) are not allowed.

From an operational standpoint, the initiative is positioned as both an access and a research effort. Applicants are expected to conduct research using data that arise from the expanded access use of investigational ALS therapies in an intermediate-size patient population. The intent is to learn from real-world use in patients who are otherwise excluded from trials, while maintaining appropriate safeguards so that the expanded access program does not compromise the broader clinical evidence base needed for regulatory review. The original notice language indicates NIH provided early visibility so prospective teams could build collaborations and design projects; the source record lists an original closing date of 2023-05-01 and a creation date of 2023-01-09. The award ceiling and expected number of awards are not specified in the provided source text.

• 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 2023-01-09.
• Applicants must submit their applications by 2023-05-01. (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 23 012

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