Current Status and Examples of US FDA or EMA’s Regulatory Decision using Real World Data/Real World Evidence

Abstract

Lately, the use of Real-World Data (RWD)/Real-World Evidence (RWE) have been actively expanded in regulatory decisions. We aimed to investigate how RWD/RWE were used in pursuit of supporting regulatory actions of FDA and EMA. We selected 9 examples of regulatory decision making with the support of RWD/RWE. To identify information included in RWE submission, we referred to the official website of US FDA or EMA. In addition, we used ClinicalTrials.gov and Medline database to collect information on study design using RWE and RWD source used to generate RWE. Our study drugs were classified into 3 parts according to regulatory context; primary approval, label expansion, and regulatory response to safety signal after marketing. Among these examples, 5 biologic products including 1 vaccine had gone through accelerated review supported by RWE. To summarize, we investigated several examples where RWD/RWE has already been used in the process of making regulatory decisions for assessing the safety of drugs as well as their effectiveness. We recommend further discussions on advancing the use of RWD/RWE in regulatory context to improve patient access to novel rare disease treatments and provide more efficient safety assessment.