Early Phase Clinical Trials: Renewal of PAS-20-160

September 2022 Council

Lead Division/Office


Point(s) of Contact

Tracy Rankin, Ph.D., M.P.H.; Aynur Unalp-Arida, M.D., M.Sc., Ph.D.; Barbara Linder, M.D., 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. Aside from generating preliminary data on intervention efficacy, a key aspect of these early phase clinical trials would be assessing protocol feasibility issues so that operational hurdles can be addressed before the conduct of a larger clinical trial, potentially leading to stronger fully-powered R01 applications. There is also an ongoing need to strengthen opportunities for young, clinical researchers to obtain quality preliminary data that will allow them to develop independent research careers. We propose to reissue PAS-20-160 (Small R01s for Clinical Trials Targeting Diseases within the Mission of NIDDK), 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.