Clinical Trials Addressing Unmet Needs of Rare Neurodegenerative Diseases (R01) Clinical Trials Required

The FDA’s Clinical Trials Addressing Unmet Needs of Rare Neurodegenerative Diseases (R01) grant, managed by the Office of Orphan Products Development (OOPD), aims to fund clinical trials that evaluate the safety and efficacy of medical products designed to address rare neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS). This initiative, part of the Accelerating Access to Critical Therapies for ALS Act, supports the development of new therapies and aims to facilitate product approval processes. This funding opportunity invites research proposals across all stages of clinical trials (Phase 1-3) and prioritizes trials that could lead to new indications or labeling changes for drugs and devices to benefit patients with rare neurodegenerative diseases. A particular focus is given to innovative trial designs, such as adaptive, seamless, basket, and platform trials, to improve efficiency and increase the likelihood of patient access to new therapies. Eligible applicants include institutions of higher education, nonprofit and for-profit organizations, local and state governments, tribal governments, and foreign institutions, among others. Non-U.S. entities and international organizations are also eligible to apply. Applications may be submitted as new proposals, resubmissions, or revisions. Budgets are capped at $650,000 annually for up to four years, with a maximum total award of $2.6 million. Additional funding of up to $250,000 per year may be available for projects using innovative clinical trial designs, provided a strong justification is included. The application process involves multiple steps, including required registrations in the System for Award Management (SAM), eRA Commons, and Grants.gov. Key dates include the earliest submission date of August 23, 2024, with letters of intent due by September 22, 2024, and final applications due by October 22, 2024. FDA expects the award process to commence by July 2025. Applicants must include a research strategy with sections addressing the study’s rationale, design, patient input, and potential impact. Early engagement with the FDA review divisions is encouraged to discuss innovative trial design options and potential challenges. Proposals will be evaluated based on several criteria: scientific merit, relevance to FDA’s goals, the innovative nature of the trial design, and the applicant’s ability to execute the project effectively within the proposed budget and timeline. Applications must demonstrate a clear benefit to rare disease treatment development and are expected to provide a compelling case for how the trial will address the current treatment gaps for neurodegenerative diseases. Documentation of patient input in trial design, such as feasibility studies or outcome prioritization, is highly encouraged. FDA requires applicants to follow strict submission guidelines, including a 12-page limit on the research strategy. Additional documents, such as letters of support, data management plans, and protocols, must be included as appendices. The program mandates compliance with Good Clinical Practice (GCP) guidelines, protection of human subjects, and provisions for IRB or IEC review. Monitoring and safety plans are critical components, and selected projects must undergo regular FDA oversight to ensure progress toward therapeutic goals. For inquiries, applicants can contact the FDA OOPD program staff, Grants Management Office, or consult the eRA Service Desk for submission support.