Compliance Assessment and Consultation on Reliability for the Application for Approval of Pharmaceuticals Using Real World Data

Abstract

In Japan, various studies and efforts are underway to promote the use of real world data (RWD) in applications for approval and reexamination of pharmaceuticals and regenerative medical products. The Ministry of Health, Labour and Welfare (MHLW) and the Pharmaceuticals and Medical Devices Agency (PMDA) have issued a number of notifications and administrative notices on the use of RWD and ensuring its reliability, as well as disseminated information at academic conferences. In addition, from the perspective of ensuring the reliability of RWD, PMDA has established a proactive support system to promote the use of RWD through activities such as establishing a consultation framework, clarifying how the compliance assessment is conducted for the application for approval of pharmaceuticals, and disseminating information that can be generalized based on the experience of consultations. How to handle RWD from the viewpoint of ensuring its reliability was once said unclear but is now considered to be resolved through these activities. Therefore, the utilization of RWD in the application for the approval of pharmaceuticals is expected to be further promoted in Japan in the future.