Evolving Real‐World Data and Evidence Use for New Drugs and Regenerative Medical Products Approvals in Japan—An Analysis of the 6‐Year Trend

Several regulatory initiatives have been made to clarify the acceptability and requirements of real-world data and real-world evidence (RWD/E) for the benefit/risk assessment of new medical products in Japan. The objectives of this review were to characterize the use of RWD/E in regulatory applications of new medical products and to describe the longitudinal trends of how use cases evolved in conjunction with regulatory initiatives in the most recent 6 years in Japan. New drugs and regenerative medical products applications approved by the Ministry of Labour, Health, and Welfare between January 1, 2019, and December 31, 2024, were included (N = 674). Of these, 158 (23.4%) applications contained RWD/E. The number of RWD/E use cases increased continuously over the period from 21 (18.1%) of applications in 2019 to 38 (30.4%) of applications in 2024. 75% of use cases categorized as the main study were used to provide comparator arms in clinical trials. Most of the cases were employed because a randomized controlled trial was ethically difficult or infeasible. In pediatric drug applications, RWD/E were leveraged to address the low feasibility of clinical trials and ethical concerns, with a relatively higher use for safety purposes (40%) observed. Alongside the development of regulatory initiatives, the findings confirm the increasing number and variety of RWD/E utilization. The requirements for the regulatory use of RWD/E differ based on its purposes and context. Insights from accumulated cases may help clarify regulatory requirements and deepen our understanding of the value of RWD/E use in developing new medicines.