Principles for the use of large-scale medical databases to generate real-world evidence

Abstract

A BSTRA CT BACKGROUND The development and expanding accessibility of large-scale medical databases support increasing use of these databases for clinical epidemiological studies in Japan. METHODS Members of the Database Utilization Committee of the Society of Clinical Epidemiology discussed the ways to enhance scientific rigor in the utilization of large-scale medical databases in Japan. The topics covered included what we need to know about the current research environment when conducting large-scale medical database studies, the key points to consider when conducting clinical research using these databases, and the use of real-world evidence in regulatory decision making. RESULTS In generating real-world evidence, several evaluation principles should be noted: the characteristics of data commonly used for clinical epidemiology in Japan; the need for reproducibility and transparency in database studies; the development of research questions for database studies ; the need to assess the quality and validity of data; and legal and ethical considerations. We also introduce the discussions on real-world evidence for drug approval applications. CONCLUSION With rapid technological developments, the characteristics of medical databases and analytical methods will evolve over time. Keeping these principles in mind, we must continuously update the evaluation methods and strategies used to assess the data.