Through the lens of marketing authorization holders: experience in use of real-world data and real-world evidence in drug development and regulatory submissions in EU

AIM: The aim of this study was to assess the general and product-specific experiences of MAHs use of RWD/RWE in medicines development and in their regulatory submissions, and to explore organizational aspects of MAHs related to RWD/RWE.

METHODS: An electronic survey was conducted, and information collected directly from MAHs. All analyses were performed using IBM SPSS Statistics 2023 (version 29).

RESULTS: Fifteen MAHs filled in an electronic survey in 2021 (40% response rate, n = 15/38) and reported vast experience with routinely utilizing RWD/RWE. RWD/RWE was mainly used to supplement the efficacy (87%, n = 13/15) and safety (80%, n = 12/15) data or defining the target population and making relevant comparisons to current clinical practice (73%, respectively, n = 11/15) in drug development. The products approved in 2019 and on which we received information (n = 22/46), 36% (n = 8/22) had utilized RWD/RWE in drug development, of which 63% (n = 5/8) included RWE as part of the approved Marketing Authorization Application (MAA). Main motivation to use RWE in MAAs was the availability of RWE (63%, n = 5/8), and its use due to low population size or rarity of target disease (38%, respectively, n = 3/8). A total of 60% of MAHs (n = 9/15) had a specific department for RWE, and 67% (n = 10/15) of MAHs outsourced at least parts of the RWD/RWE tasks.

CONCLUSION: The research shows that, already by 2021, the use of RWD/RWE in pre-approval drug development was widespread and systematic among large pharmaceutical companies, even if this is not fully visible in public regulatory documents. Overall, the findings provide rare, direct industry evidence that RWD/RWE are already embedded in pre-marketing development and utilized to support EU regulatory submissions.