Utilization of Healthy Volunteers in Oncology Drug Development in Japan: A 10-Year Analysis of Approved Anticancer Agents (2014-2024).

Anticancer agents with favorable safety profiles, such as immunomodulators and molecular-targeted agents, are increasingly used in recent cancer therapy, and clinical trials for these agents increasingly incorporate healthy volunteers as well as cancer patients. Clinical trials in healthy volunteers allow intensive safety monitoring and intensive pharmacokinetic sampling, enabling the acquisition of high-quality, broad-range pharmacokinetic and safety data within a shorter timeframe and at lower cost. Leveraging these advantages by utilizing healthy volunteer trials in place of patient trials may contribute to more rapid and efficient implementation of anticancer drug development and is expected to help eliminate the "drug lag" and "drug loss" that have been concerns in Japan's research and development process in recent years. In this study, we investigated the clinical data packages of newly approved anticancer agents in Japan from April 2014 to March 2024 and evaluated trends in cases in which data from Japanese healthy volunteers were obtained. We then summarized key considerations for anticancer drug development in Japan regarding whether data from Japanese healthy volunteers should be obtained.