Reissue of RFA-FD-22-001 - Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases (R01 Clinical Trials Optional)

The U.S. Food and Drug Administration (FDA), through its Office of Orphan Products Development (OOPD), has reissued its funding opportunity titled "Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases (R01 Clinical Trials Optional)," identified by Funding Opportunity Number RFA-FD-25-017. This grant is a reissue of RFA-FD-22-001 and is designed to support natural history studies that can advance medical product development in rare diseases with significant unmet needs. The initiative falls under the R01 Research Project Grant activity code and aims to provide critical insights that can remove major obstacles to therapeutic innovation and broaden the knowledge base for rare conditions. This opportunity is explicitly intended for both retrospective and prospective observational natural history studies. The research must involve rigorous, standardized, and interpretable data collection methods to be eligible for consideration. The funding will support projects that either incorporate or facilitate collaborative efforts among academia, industry, and patient organizations. Projects may also include the development or validation of biomarkers and clinical outcome assessments (COAs), particularly if conducted in conjunction with prospective studies. Studies employing advanced methods such as machine learning and simulation to improve trial design may be eligible for increased funding, subject to strong justification. Funding amounts vary depending on the study type. Prospective studies can receive up to $400,000 annually for four years, while retrospective studies are limited to $150,000 annually for two years. Applications requesting additional funding of up to $200,000 per year for innovative trial designs may be considered. This could bring the annual total to $600,000 over four years. Applications must detail expected expenses annually and provide comprehensive justifications, especially if requesting increased funding. The number

Engage early with FDA review divisions and adhere to submission guidelines.