Real-world evidence (RWE): A challenge for regulatory agencies discussion of the RWE conference with the network of the european medicine agencies, patients, and experts

Apart from its traditional use in the post-authorization phase for safety assessment, risk management, and life cycle benefit-risk evaluation (Flynn et al., 2022), the interest in Real-World Evidence (RWE) in the pre-authorization phase of medicines development is increasing exponentially (Li et al., 2021; Leufkens et al., 2022; Purpura et al., 2022). In this context, regulatory agencies should adapt their strategy (Heads ofMedicines Agencies and European Medicines Agency, 2019a). RWE is defined as “the clinical evidence about the usage and potential benefits or risks of a medical product derived from analysis of RealWorld Data (RWD)”; the latter includes “big data”, i.e., “extremely large datasets which may be complex, multi-dimensional, unstructured and heterogeneous, which are accumulating rapidly and which may be analyzed computationally to reveal patterns, trends, and associations” (Heads of Medicines Agencies and EuropeanMedicines Agency, 2019b). RWE has a great potential not only to complement evidence generated through randomized controlled trials (RCTs) (Eichler et al., 2021) but also to provide valuable opportunities unavailable through RCTs (Chodankar, 2021). RCTs rightfully remain the fundamental method to establish the safety and efficacy of drugs for licensure (Slattery and Kurz, 2020). Nevertheless, the growing production and accessibility of digital health data support RWE to close the evidentiary gap between clinical research and real-world practice for better disease management (Corrigan-Curay et al., 2018; Lasky et al., 2021).