Real-World Data and Real-World Evidence in Regulatory Decision Making: Report Summary From the Council for International Organizations of Medical Sciences (CIOMS) Working Group XIII.

Data from sources other than traditional randomized clinical trials are known as real-world data (RWD), and the evidence derived from the review and analysis of RWD is known as real-world evidence (RWE). RWD and RWE are used increasingly throughout the lifecycle of medicinal products to provide evidence about their effectiveness and safety. Recent regulatory guidance about RWE and approvals based on the use of RWE to demonstrate beneficial effects of products have created an urgency to develop generally accepted processes that promote trust in the evidence-generation process. A recent report from a working group of the Council for International Organizations of Medical Science (CIOMS) describes the use of RWE for decision making in the lifecycle of medical products, describes RWD and data sources, discusses key scientific considerations in the generation of RWE, and discusses ethical and governance issues related to the use of RWD. This paper provides a high-level summary of this report. More work remains to be done to globally harmonize practices and guidance for using RWD and RWE for regulatory decision making, thereby maximizing the benefits they can bring to patient care and public health.