Abstract
Recent discussions about the utilization of real-world data (RWD) and real-world evidence (RWE) have been more focused on drug development for regulatory approval rather than during the post-marketing stage. In Japan, RWD/RWE have been practically utilized as an external control for drug approval. Most cases were related to orphan diseases where the feasibility of conducting randomized controlled clinical trials was generally low. The utilization of RWD/RWE as an external control provides additional information that can support regulatory review for drug approval. However, many points should be taken into consideration through all stages of a study that is based on RWD/RWE, including planning, analysis, and interpretation. In this article, we present our recent review experience focusing on efficacy evaluations with an external control based on RWD/RWE that were submitted as a part of new drug applications in Japan, and we describe our regulatory consideration of the utilization of RWD/RWE for drug evaluation and approval. Points described in this article promote appropriate drug development based on RWD/RWE and facilitate a proper discussion about RWD/RWE utilization with PMDA. Further accumulation of regulatory experience in PMDA with RWD/RWE utilization will enhance our knowledge and contribute to better regulatory decision making for drug approvals based on RWD/RWE.