Current Status, Challenges, and Future Perspectives of Real-World Data and Real-World Evidence in Japan

Abstract

The aim of this article is to help develop a common understanding of the current status, challenges, and future perspectives of real-world data (RWD) and real-world evidence (RWE) in Japan. RWD and RWE are very widely used terms, but standardized definitions are lacking. Given broad and growing applications of RWD/RWE from the perspective of clinical development and medical affairs, the PhRMA Japan Medical Affairs Committee Working Group 1 have proposed the following definitions: "RWD are the data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources" and "RWE is the evidence derived from analysis of RWD." The key challenges for RWD and RWE in Japan include restricted access and linkage of RWD, as well as a lack of universally accepted methodological approaches, which reduces the potential for patient and healthcare benefits. These challenges for RWD/RWE are by no means unique to Japan and similar challenges exist for countries in Europe and the USA. The quality of data and analysis, study design, and the transparency of reporting should be discussed more to ensure credibility and acceptance by decision-makers as the demand for RWD and RWE increases. The future developments around Japan's RWD and RWE are expected to include improved RWD access, data linkage, and increased acceptance by decision-makers, all supported by innovative technology. Improvements in RWD access and database linkage will enable both public and private sectors to assemble more comprehensive health information in Japan.

Fig. 1

Real-world data and real-world evidence utilization by pharmaceutical companies. (1) for development strategy; (2) for clinical trial design; (3) for promotion of enrolment of study participants; (4) for drug price calculation; (5) for expansion of indications; (6) for new or additional indications; (7) for identification of unmet medical needs, closing data gaps, and informing clinical practice. RWD real-world data, RWE real-world evidence

Fig. 2

Real-world data definitions and data sources. Adapted from Makady et al. [16] with permission from Value Health 2017; 20(7):858-65. Copyright© 2017 International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc. All rights reserved. EMR electronic medical record, LST large simple trial, Obs. Observational, PAES post-authorization efficacy study, PASS post-authorization safety studies, PCT pragmatic clinical trial, RCT randomized clinical trial, RWD real-world data, EMA Europe Medical Agency, FDA US Food and Drug Administration, ISPOR the International Society of Pharmacoeconomics and Outcomes Research, ISPE the International Society of Pharmacoepidemiology