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

This Notice of Funding Opportunity (NOFO) invites researchers to submit applications for support of clinical projects that address critical needs for clinical trial readiness in rare diseases. The initiative seeks applications that are intended to facilitate rare diseases research by enabling efficient and effective movement of candidate therapeutics or diagnostics toward clinical trials, and to increase their likelihood of success. This could be through the development and testing of rigorous biomarkers and clinical outcome assessment measures, or by defining the presentation and course of a rare disease to enable the design of upcoming clinical trials. The program supports projects that prepare the groundwork for future clinical trials in rare disease populations, focusing on pre-clinical trial activities rather than conducting actual clinical trials. Eligible applicants include a wide range of organizations including educational institutions, government entities, nonprofits, and for-profit organizations, though foreign entities are not eligible.