Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01 Clinical Trials Required)

The Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01 Clinical Trials Required), opportunity number FOR-FD-25-020, is a forecasted federal grant administered by the U.S. Food and Drug Administration (FDA), an agency within the Department of Health and Human Services. This funding opportunity is designed to promote clinical research that directly supports the advancement of therapies for rare diseases, also known as orphan diseases. It seeks to increase the number of FDA-approved treatments by facilitating trials that demonstrate efficacy and/or safety for products potentially qualifying for new indications or updated labeling. This initiative is grounded in the FDA’s longstanding commitment to supporting development for products addressing rare diseases. The target population includes patients with unmet medical needs due to the lack of approved or effective treatments for their conditions. The opportunity mandates the conduct of clinical trials, making it essential for applicants to have the capacity to conduct studies that will produce actionable safety or efficacy data, especially data that could be submitted to support labeling changes or product approval. While the exact funding amounts—including total program funding, award ceiling, and award floor—have not been disclosed in this forecast, the assistance is offered in the form of discretionary grants. Funding will be drawn from the FDA’s Research Program, listed under Assistance Listing 93.103. There is no cost-sharing or matching requirement, which may help reduce barriers to entry for smaller institutions or investigators. Although the precise project start, award, and submission dates are not listed, the forecast was released on July 11, 2025, indicating that further details will be published in the near future. Eligibility for this opportunity is broad and includes a range of public and private entities, such as higher education institutions (both public and private), nonprofits with and without 501(c)(3) status, for-profit organizations including small businesses, as well as various levels of government (local, state, and federal). Federally recognized and other tribal governments, public housing authorities, school districts, and international entities (both foreign organizations and non-U.S. components of U.S. organizations) are also eligible to apply. The grant is open to foreign organizations and is available nationwide. At present, as a forecasted opportunity, the grant is not yet accepting applications. Interested parties should monitor Grants.gov and the FDA’s funding announcements for updates regarding official release dates, application instructions, and submission deadlines. No additional documents or application questions have been provided in the forecast, nor has the estimated number of awards been specified. For more information or clarification, potential applicants can contact Kimberly Pendleton at 240-402-7610. An email address has not yet been provided in the forecast notice.