The global use of real-world data (RWD) for drug approval and post-marketing surveillance has increased significantly. During 2023 and 2024, the US Food and Drug Administration issued industry guidance, and the European Medicines Agency released real-world evidence framework reports to support regulatory decision making. In Japan, the Pharmaceuticals and Medical Devices Agency promotes the use of RWD, with collective efforts from the government and related industries. This report focuses on electronic health record (EHR) databases in Japan, aiming to (1) organize the characteristics of data generated from such databases, and (2) identify the challenges when using EHR databases for regulatory decision making. This report conducted an internet-based Google search using the keywords ‘electronic medical record database’ with the condition that the results include any of the keywords ‘Japan’ or ‘real-world data’. Online interviews were conducted with representatives from eight EHR database companies identified as being utilized in Japan, focusing on the characteristics of each EHR database, challenges in their utilization, and future perspectives. The characteristics and limitations of current EHR databases were summarized; they offer broad coverage in terms of patient backgrounds and easier access to clinical laboratory values but are unable to track patients who transfer between hospitals due to lack of data linkage, and there may be loss of information during data abstraction. Data collection methods were mostly retrospective. Critically, there is no overall standardization between the EHR databases. Currently, EHR studies are primarily observational to verify efficacy and safety in real clinical practice and are gradually being used for post-marketing surveillance to support regulatory decision making in Japan. Addressing operational, technical, and methodological challenges in using EHR databases for regulatory approval applications requires efforts from multiple stakeholders, including industry, academia, and government. Real-world data (RWD) are information collected from everyday medical care, not from clinical trials. Around the world, RWD are increasingly being used to verify the effectiveness and safety of medicines. In 2023 and 2024, the US and Europe published regulations to guide this process. In Japan, the government and industry are also working to utilize RWD for drug approval and safety monitoring. This topic matters to the general public because better use of RWD can lead to safer medicines, faster access to new treatments, and healthcare decisions that more accurately reflect everyday patient care, not just clinical trials. This report examines electronic health record (EHR) databases in Japan. These databases store patient information from hospitals. The report explains what these databases can do and what challenges exist. The study used online searches and interviews with database companies. EHR databases have some strengths. They include many types of patients and laboratory test results. But there are challenges. Patients cannot be tracked if they move to another hospital. Some information may be lost when data are processed. Most data are collected after care, not in real time. Also, there is no common standard across databases. Today, EHR data in Japan are mostly used to confirm drug safety and effectiveness after approval. To use these data for new drug approvals, many issues need to be resolved through collaboration between industry, universities, and government.
Piao et al. (Wed,) studied this question.