In recent years, many medicines regulatory agencies have expressed increased willingness to consider real-world evidence (RWE), that derives from the review and/or analysis of real-world data (RWD), to support claims of efficacy or effectiveness as well as of safety. This increased willingness is changing the regulatory environment in which RWE is generated and used. This consensus report aims to describe the potential use of RWE for decision making; RWD and data sources; key scientific considerations in the generation of RWE; and ethical and governance issues in using RWD.

The intended audience for this report includes medicinal product regulators, healthcare payers, health care and medicinal products industries, researchers, bioethicists, patients and health care professionals. This report was developed to inform discussions about the use of RWD and RWE for regulatory and health care decision making, including decisions to make a product available for use (authorisation), to cover the costs of its use (reimbursement), and to use a product for a particular patient (clinical use).

This report reflects the opinions of the Council for International Organizations of Medical Sciences (CIOMS) Working Group XIII on Real-world data and real-world evidence in regulatory decision making, and it was finalised after considering comments received during a public consultation.

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