Problem Statement: In July 2021, the Center for Drug Evaluation and Research (CDER) approved a new indication for tacrolimus (Prograf®) using data from Scientific Registry of Transplant Recipients . This approval, based on real-world evidence (RWE), is CDER’s first acceptance of an observational study as an adequate and well-controlled study demonstrating substantial evidence of effectiveness that met the Food and Drug Administration (FDA) regulatory standards. However, several analytical and data issues were identified during the review process.

Description: The 21^st Century Cures Act has been supporting acceleration of medical product development and bringing new innovations faster and more efficiently. Thus, RWE, the clinical evidence generated from the analysis of real world data (RWD), presents great potential to accelerate drug development and has received substantial attention in recent years. On July 16, 2021, the FDA approved a new indication for tacrolimus (Prograf®) based on an observational study demonstrating RWE of effectiveness. This approval reflects how a well-designed, non-interventional study relying on fit-for-purpose RWD can be considered adequate and well-controlled under FDA regulations. This talk will give an overview of the regulatory considerations from a statistical perspective, and the importance of quality fit-for-use RWD for drug applications.

Lessons Learned: Making use of existing data can accelerate the drug development process. To maintain evidentiary standards, the data should be fit-for-purpose and limitations such as data quality issues should be addressed in the early stage of drug development program.

Stakeholder perspective: To be used for drug approval, RWD should be managed transparently and frequent communication with the regulatory agency might be needed when issues arise.