Forecast for Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed)

The National Center for Advancing Translational Sciences (NCATS), part of the U.S. Department of Health and Human Services under the National Institutes of Health (NIH), plans to issue a Notice of Funding Opportunity (NOFO) to support clinical trial readiness for rare diseases, disorders, and syndromes. This initiative is designed to foster research that facilitates the efficient and effective progression of candidate therapeutics or diagnostics into clinical trials and enhances their probability of success. The program will leverage the NIH R21 activity code and will not allow clinical trials under this opportunity. The overarching goal of this funding opportunity is to support the development and testing of rigorous biomarkers and clinical outcome assessment measures. Another focus area is improving understanding of the presentation and progression of rare diseases, which is essential for the design of future clinical trials. The NIH encourages applications from researchers with relevant expertise who can contribute to advancing clinical trial readiness in this field. The forecast indicates that the NOFO is intended to give potential applicants ample time to develop meaningful collaborations and prepare competitive projects. Applications are not being solicited at this time, but the estimated post date for the opportunity is November 14, 2025, with an estimated application due date of February 16, 2026. Eligibility for this grant includes a broad range of entities such as Native American tribal governments (Federally recognized), state governments, private and public institutions of higher education, nonprofits with 501(c)(3) status, independent school districts, county governments, public housing authorities/Indian housing authorities, Native American tribal organizations (other than Federally recognized tribal governments), for-profit organizations (excluding small businesses), and small businesses. No cost sharing or matching is required for this funding opportunity. The expected award date is December 15, 2026, with funded projects anticipated to start on the same date. As this is a forecasted opportunity, certain details such as total program funding, award ceiling, and award floor have not yet been disclosed. For more information, interested applicants can contact Alice Chen Grady, M.D. at the National Center for Advancing Translational Sciences. She can be reached by phone at 301-827-2015 or via email at alice.chen2@nih.gov.