Real-world evidence (RWE) is increasingly gaining prominence in drug approvals and regulatory decisions. In 2019, over half of all US Food and Drug Administration (FDA) submissions for new drugs and biologics included RWE studies, a figure that surged to 78% in 2020. Similarly, Europe incorporated RWE in 40% of marketing authorization applications and 18% of indication extensions submitted to the European Medicines Agency (EMA) in 2018-2019. Japan has also started basing orphan drugs on real-world data (RWD).
As defined by the FDA, RWD includes patient health and healthcare data gathered from various sources, such as electronic health records, medical claims, and registries. While interrelated, RWD should not be confused with RWE, which involves clinical evidence derived from RWD analysis. To advance this field, the FDA has issued draft guidances on RWD and RWE, addressing aspects like electronic health records, data standards, and regulatory considerations. Efforts to harmonize global RWD policies and standards are underway through organizations like the International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use (ICH), the International Coalition of Medicines Regulatory Authorities, and the Council for International Organizations of Medical Sciences.
The European Medicines Agency (EMA) and the Heads of Medicines Agencies (HMA) also reviewed RWD studies between September 2021 and February 2023, aiming to integrate RWD/RWE into regulatory decisions alongside randomized controlled trials. Review the recommendations from this guide regarding the development, authorization, and supervision of medicines, in addition to several benefits of using RWD and RWE in clinical trials by downloading the article below.