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

Abstract

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.