Drug Evaluation Committee
—For Decision-Making Based on Patients’ Voices—
April 2025
In 2021, the ICH published a Reflection Paper on “Patient-Focused Drug Development,” advocating for the use of patient-derived information (patient experience data) in drug evaluation. However, there are currently no guidelines in Japan that provide an overview of how to handle patient experience data—specifically, what types of data to collect, how to collect them, and how to utilize them.Therefore, this task force has prepared a report providing an explanation of patient experience data—such as patient preference information and patient-reported outcomes—and summarizing recent proposals from various regulatory authorities as well as the current status of its utilization.This report is intended for statisticians, clinical staff, and other professionals involved in clinical development who are considering and discussing the use of patient experience data from a data science perspective. We hope this report will serve as a useful resource for considering how patient experience data can be applied to drug development in Japan.
Japan Pharmaceutical Manufacturers Association (JPMA) Drug Evaluation Committee
Data Science Subcommittee FY2024 Task Force 6