Amyotrophic Lateral Sclerosis (ALS) Intermediate Patient Population Expanded Access (U01 Clinical Trial Required)

The Amyotrophic Lateral Sclerosis (ALS) Intermediate Patient Population Expanded Access program is a federal funding opportunity administered by the National Institutes of Health through the National Institute of Neurological Disorders and Stroke, with participation from the NIH Office of the Director. This initiative is part of a broader federal effort to accelerate therapeutic development for ALS, a rapidly progressive and fatal neurodegenerative disease. The program builds on authorities established under the Accelerating Access to Critical Therapies for ALS Act and leverages the FDA expanded access pathway to generate scientific data while providing investigational treatment access to patients who are not eligible for traditional clinical trials. The primary purpose of this funding opportunity is to support research that utilizes data collected through intermediate-size expanded access protocols involving investigational drugs or biological products for ALS. These protocols are designed to provide treatment access to groups of patients while simultaneously generating valuable data on safety, biomarkers, patient outcomes, and disease progression. The program emphasizes that expanded access activities must not interfere with ongoing or future clinical trials that are intended to support regulatory approval of therapies. Applicants are expected to design protocols that complement existing clinical research and contribute meaningfully to the scientific understanding of ALS. Funding is provided through a cooperative agreement mechanism, indicating substantial involvement by NIH program staff throughout the project lifecycle. The program intends to commit up to forty million dollars annually to support approximately four new awards each year, contingent on appropriations and application quality. Project periods may extend up to four years, and budgets must reflect the actual needs of the proposed research. Allowable costs include expenses related to investigational product acquisition, clinical site operations, biospecimen collection and analysis, and participant support, though certain indirect costs are restricted for specific components. Eligibility for this opportunity is broad in terms of organization types, including higher education institutions, nonprofit organizations, for-profit entities, and government bodies. However, there are critical program-specific requirements: applicants must be clinical trial sites participating in phase two or phase three efficacy trials sponsored by a U.S.-based small business holding an active investigational new drug application. Foreign organizations and components are not eligible. Additionally, applicants must demonstrate compliance with regulatory requirements, including submission of an expanded access investigational new drug application to the FDA and obtaining approval prior to funding. The application process requires submission through federal systems such as Grants.gov or NIH ASSIST, with tracking through eRA Commons. Applicants must adhere strictly to NIH application guidelines and provide detailed research plans, data management strategies, and documentation of partnerships and regulatory readiness. Required components include a research strategy outlining scientific significance, methodological rigor, and plans to avoid interference with clinical trials, as well as documentation of stakeholder engagement and data sharing commitments. Applications are evaluated based on significance, innovation, rigor, feasibility, investigator expertise, and institutional capacity. Key dates include an opening date of May 4, 2026, with multiple submission deadlines including June 8, 2026 and November 10, 2026, and an expiration date of November 11, 2026. Applications must be submitted by 5:00 PM local time on the due date, with no late submissions accepted. Review cycles occur shortly after submission, with funding decisions and project start dates following within months. This funding opportunity does not require cost sharing and is structured as a recurring initiative aligned with federal fiscal cycles, reflecting ongoing national priorities in ALS research and therapeutic development.

Ensure expanded access protocol does not interfere with clinical trials; emphasize data rigor and stakeholder engagement; align with NIH data standards