OBJECTIVES: Traditionally, RWE is used in post-authorization studies for safety assessments and risk management. However, the interest in RWE in the pre-authorization phase of medicines development is increasing. The recent initiative of the European Medicines Regulatory Network to create an EU-wide distributed network of real world data named the Data Analytics and Real World Interrogation Network (DARWIN EU) has the potential to further amplify this trend. This study aimed at assessing RWE use in new marketing authorization applications (MAA).

METHODS: New MAA assessed by the European Medicines Agency (EMA) which resulted in a recommendation for approval in 2021 were scanned for RWE use and assessed with respect to characteristics such as indication, RWE data source used, and methodological approaches.

RESULTS: A total of 92 medicines recommended for approval in 2021 were included and assessed accordingly. Common indications amongst others were cancer, neurology, and endocrinology. Further, frequently utilized RWE data sources included electronic healthcare records, hospital data and claims data. Overall, RWE studies were used for purposes such as safety and efficacy evaluations.

CONCLUSIONS: In light of the changes in acceptance of regulators, advancements in RWE generation in terms of accessible data sources and statistical methods, it is anticipated that hybrid approaches including clinical and real world data to evidence generation will be required. Further advancing the knowledge on RWE and accelerating the acceptance in the field of early drug development is of utmost importance given the rapidly changing landscape.