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

The National Center for Advancing Translational Sciences, with other NIH Institutes and Centers (ICs), intends to publish a Notice of Funding Opportunity (NOFO) to solicit applications for research on projects that address critical needs for clinical trial readiness in rare diseases. The initiative will seek 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. Applications are not being solicited at this time. Notice is being provided to allow potential applicants sufficient time to develop meaningful collaborations and responsive projects. This NOFO will utilize the R21 activity code. Investigators with expertise and insights into this area of clinical trial readiness for rare diseases, disorders, and syndromes are encouraged to begin to consider applying for this new NOFO.