Drug Evaluation Committee
Utilization of Real World Data in Drug Development
(1) First Step to Application for Approval Using an External Control Group
2) Submitting real-world data to regulatory authorities
3) Toward the promotion of RWD utilization in pharmaceutical companies
Clinical Evaluation Subcommittee
May 2020
The use of medical big data (BD) and real world data (RWD) in drug development not only improves efficiency for pharmaceutical companies, but also promotes development in areas where randomized controlled trials are difficult to conduct, such as rare diseases, intractable diseases, and pediatric patients, thereby improving patients' access to new drugs and reducing the burden on medical facilities by utilizing secondary use data. The use of secondary-use data is expected to bring benefits from a variety of perspectives, such as reducing the burden on medical facilities through the use of secondary-use data. The Clinical Evaluation Subcommittee's 2019 Task Force 2 has prepared the following three subcommittee documents for the promotion of medical BD and RWD utilization, which we hope you will find useful.
We have addressed the case of utilizing registry data as an external control group for clinical trials and presented as specifically as possible what needs to be done in the application for approval and the issues involved. We received the cooperation of SCRUM-Japan, Remudy, Intractable Disease Platform, and C-CAT, which are representative registries in Japan, for this review.
(2) Submitting Real World Data for Approval - Recommendations for Promoting Utilization - (1.6MB)
We will present the results of our survey of regulatory and infrastructure projects related to RWD in Japan, Europe, and the U.S., as well as examples of applications for approval utilizing RWD in each country. Based on the results of the survey, we also summarize the issues and recommendations for submitting applications for RWE in Japan.
The information that pharmaceutical companies need to confirm when considering the introduction of RWD and suggestions for more advanced use are summarized, focusing on resources, benefits, and risks. In addition, with the cooperation of the Association of Medical Databases, we will introduce the items that pharmaceutical companies should keep in mind when introducing and utilizing RWDs.
Deliverables of the Drug Evaluation Committee Return to list of all deliverables