Drug Evaluation Committee Examination of Study Design and Statistical Evaluation Methods in the Development of Regenerative Medicine Products
May 2021
The markets for industries related to regenerative medicine, both in Japan and abroad, are projected to experience rapid growth in the future. In recent years, Japan has been working to establish regulations and systems tailored to the characteristics of regenerative medicine products and to promote their practical application through financial support. In this fiscal year 2020 TF3 initiative, we will organize Japanese, U.S., andEurope regarding the development of regenerative medicine products and other related products. After carefully examining the application packages and review records for products approved in Japan and the U.S., the task force made recommendations on trial designs to be considered for improving the level of evidence and statistical evaluation methods to be explored for streamlining clinical trials.We hope this report will serve as a new catalyst for lively discussion among those involved in the development of regenerative medicine products and “Launch Excellence,” and that it will assist in the formulation of scientific and efficient strategies.
Japan Pharmaceutical Manufacturers Association (JPMA) Drug Evaluation Committee,
Data Science Subcommittee, FY2020 Task Force 3