The Food and Drug Administration (FDA) has announced a forecasted funding opportunity titled "Clinical Trials Addressing Unmet Needs of Rare Neurodegenerative Diseases (R01) Clinical Trials Required" under opportunity number FOR-FD-25-001. This grant focuses on funding clinical trials that evaluate the safety and efficacy of medical products intended to address unmet needs in rare neurodegenerative diseases in both children and adults. The trials supported by this initiative will contribute to regulatory advancements, such as new indications or changes in product labeling, with the ultimate aim of increasing the number of approved treatments for these debilitating conditions.

The primary goal of this opportunity is to support clinical trials that explore innovative or collaborative approaches to developing treatments for rare neurodegenerative diseases. These diseases, often overlooked in mainstream drug development, present significant challenges in terms of treatment options, particularly for conditions affecting smaller patient populations. By funding clinical trials that meet these unmet needs, the FDA seeks to encourage research that could lead to groundbreaking treatments, significantly impacting the field of rare disease drug development.

The program is expected to offer awards ranging between $650,000 and $900,000, although the total number of awards and overall program funding have not yet been determined. This flexibility in award amounts allows applicants to tailor their clinical trial proposals to the scope and scale required by their specific research. Notably, there is no cost-sharing or matching funds requirement, which makes this opportunity more accessible to a wider range of applicants, including smaller organizations or institutions with limited resources.

Eligible applicants for this opportunity are diverse and include state and local governments, public and private institutions of higher education, for-profit organizations (excluding small businesses), nonprofits with or without 501(c)(3) status, Native American tribal organizations and governments, public housing authorities, and other entities. This broad eligibility ensures that various institutions with the capability to conduct clinical trials are able to apply. The focus is on ensuring that the proposed clinical trials address significant gaps in treatment options for rare neurodegenerative diseases.

The timeline for this grant, including the application due date and project start date, is still to be determined. Applicants should expect further details closer to the estimated post date, with funding decisions anticipated in fiscal year 2025. The FDA will assess the clinical trials based on their potential to generate meaningful data that could lead to new treatment approvals or expanded indications for existing products. Applications will need to demonstrate a clear plan for assessing the efficacy and safety of the products being studied in the context of rare neurodegenerative diseases.

Researchers and institutions interested in applying for this grant should begin preparing their proposals by identifying specific unmet needs within the rare neurodegenerative disease space and developing robust clinical trial designs. For additional information or clarifications, the FDA’s contact for this opportunity is Terrin Brown, who can be reached at (240) 402-7610 or via email at terrin.brown@fda.hhs.gov. This opportunity represents a significant step forward in addressing the urgent need for effective treatments for rare neurodegenerative diseases, offering substantial funding support for innovative clinical trials.