2021 Volume 11 Issue 1 Pages 27-41
The use of real world data (RWD) and real world evidence (RWE) is expected to have various advantages in drug development such as increase in efficiency of development, improvement of patients’ access to new drugs, and reduction in burdens of data collection on medical staff by using secondary data. However, the acceptability of RWD/RWE for regulatory purpose is still unclear in any countries including Japan and the US. We investigated regulations and regulatory authorities’ projects on RWD/RWE and cases of RWE use for drug approval in Japan and the US. Regulations and projects in Japan were for developing unapproved drugs and off-label use, the purpose of post marketing surveillance, and leveraging disease registry. In the US, the FDA provided a framework that will evaluate the use of RWD/RWE to support the regulatory decision making. The FDA has also been working on projects in collaboration with pharmaceutical and health data companies and academic institutions dealing with practical issues of RWD/RWE application. From the review of the cases in which FDA did or did not accept RWE as evidence to support the decision making on approval, key points on reliability and relevancy of RWE required for regulatory use were found. Furthermore, registry data or electronic health record (EHR) -derived data were commonly used for regulatory submission in the US, whereas in Japan, registry data were commonly used. In order to enhance the use of RWD/RWE for regulatory decision making in Japan, it is important to develop an environment where various data sources can be applied and to have a common view of the reliability of RWD/RWE.
