OBJECTIVES: The use of real-world evidence (RWE) and real-world data (RWD) may play an important role in complementing clinical trial data to support the clinical effectiveness of a medicinal product in regulatory submissions. This review identified the prevalence with which RWE was evaluated in regulatory decisions.

METHODS: European Public Assessment Reports (EPARs) for human drugs published by the European Medicines Agency (EMA) during the last year (20/06/21 - 20/06/22) were identified. Only documents reporting RWE-related terms were included.

RESULTS: During the study period, 76 EPARs were first published or updated by the EMA for authorised drugs, and among these, 43 (57%) included RWE-related terminology. Drug therapeutic areas were most commonly cancer (n=11), followed by neurological conditions (n=8), and infections (n=7). RWD were reported for 27 (36%) drugs, more frequently in the Product Assessment Report (PAR; n=23) than in the Product Information (n=8). A lack of long-term clinical efficacy and/or safety data and small patient numbers, particularly in paediatric and pregnant populations, were referenced as examples of a common evidence need for RWE. However, some concerns were raised by the EMA regarding the interpretation of RWD, due to methodological limitations, such as small sample sizes, risk of bias, and non-randomised study designs. Furthermore, 17 drugs were approved subject to post-authorisation obligations relating to the long-term collection of RWD, most commonly post-authorisation safety studies (PASS; n=10).

CONCLUSIONS: RWE was used to support product labelling claims in more than a third of EMA decisions in the last year. However, these data were mostly reported in the supplementary PAR, not the Product Information, which suggests that the data did not significantly contribute to the regulatory decision-making. The frequency of drugs gaining conditional authorisation indicates that large, long-term observational studies can provide valuable data, particularly in the post-authorisation period.