製薬企業における日本のRWD/RWEの薬事利用について

抄録

The utilization of Real World Data (RWD) and Real World Evidence (RWE) in clinical development for drugs has gained significant attention, particularly following the enactment of the 21st Century Cures Act in the United States in 2016. This act encouraged the use of RWD for regulatory purposes and RWE have been used in the development of treatments for rare diseases, regenerative medicine, and gene therapy, where randomized controlled trials (RCTs) are challenging to conduct. Various attempts have been made to use RWE as an alternative to RCTs, and recent evaluations have highlighted both the strengths and limitations of RWE. This paper summarizes case evaluations of RWD for regulatory use and explores potential applications of RWD beyond evidence generation in clinical development. The discussion includes the role of RWD in regulatory decision–making, the challenges of using RWD for non–interventional evaluations of drug efficacy and safety, and the evolving landscape of RWD utilization in Japan's healthcare system. The paper also addresses the integration of RWD with traditional RCTs to complement evidence generation and improve decision–making in drug development.