Real-world data in drug regulation-current developments and outlook

Background: Real-world data (RWD) and real-world evidence (RWE) derived from RWD through statistical-epidemiological analyses play a promising and increasingly relevant role in regulatory decision-making along the product life cycle of medicinal products.Objectives: To provide an overview of the current status, development potential and initiatives to increase the use of RWE in European and international drug regulation.Materials and methods: The review is based on original papers and reviews from the current international literature (including our own research results), current examples from regulatory practice, and involvement in European and international initiatives to increase the use of RWD/RWE in regulatory decision-making processes.Results: To date, RWE from RWD is primarily used for supportive evidence in regulatory decision-making. In addition to the established use in postauthorization phases (e.g., monitoring of drug safety), RWD are also increasingly used in the preauthorization phase and in evaluation. Currently, a health data infrastructure is being established through increased networking of data sources at the national and international level to create new opportunities for RWD use.Conclusions: In addition to a growing importance of RWD/RWE in European and international drug regulation, there are also new challenges to accessing and analyzing RWD. The variability and heterogeneity of RWD sources makes the development of new and optimized methods for RWD analyses essential. New guidance and training concepts for the stakeholders involved are also crucial.