Early Phase Clinical Trials

January 2020 Council

Lead Division/Office

DEM, KUH, DDN

Point(s) of Contact

Barbara Linder, M.D., Ph.D.; Tracy Rankin, Ph.D., M.P.H.; Aynur Unalp-Arida, M.D., M.Sc., Ph.D.

Executive Summary

Improving prevention and treatment of disease requires large clinical trials to ensure efficacy and safety of the proposed intervention. The first step in this process is often exploratory, short-term work to investigate new ideas. Preliminary data are needed about intervention effects and to support feasibility of recruitment/retention and study conduct. NIDDK has used the R21 to support pilot and feasibility clinical trials. However, analysis across all three NIDDK extramural scientific divisions shows that the R21 has not been effective in promoting successful R01 submissions for the conduct of well-designed clinical trials. Overall, the short duration and limited financial support of the R21 is inadequate to garner meaningful preliminary data that can serve as a foundation for larger, adequately powered trials. There is also a need to strengthen opportunities for young, clinical researchers to obtain quality preliminary data that will allow them to develop independent research careers. We propose an FOA to support R01s for 3-year clinical trials having a solid scientific premise and sufficient demonstration of operational feasibility to conduct a pilot. Additional preliminary data would not be required. Applications submitted to this PAS will be reviewed by CSR and assigned a percentile with other R01 applications. A small set-aside will be included to be able to augment the pool of clinical researchers, if there are highly meritorious applications beyond the pay line.