A new report from the E.U. Big Data Steering Group (BDSG), EMA, and the European Medicines Regulatory Network (EMRN) shares experience obtained from real-world data (RWD) based studies between September 2021 and February 2023. The aim of the study was to establish a workable framework for using real-world evidence (RWE) for regulatory cases within the E.U., building off the EMA’s Strategy through 2025.
Real-world data (RWD) is data generated by the healthcare system outside of clinical trials, including electronic health records, claims data, and data from registries. RWD can be used to generate real-world evidence (RWE), which is increasingly used for regulatory decision-making. In the E.U., there has been a focus on advancing RWE in regulatory decision-making, with the publication of the Operational, Technical, and Methodological (OPTIMAL) Framework for RWE in 2019. This framework outlined the challenges and possible solutions for using RWD in regulatory decision-making and acknowledged the changing digital data landscape.
The EMA’s Pharmacovigilance Risk Assessment Committee (PRAC) conducted a pilot program in September 2021 to explore the use of RWE generated by EMA to support regulatory decisions. This pilot program included work from various committees, such as the Pediatric Committee (PDCO), the Committee for Orphan Medicinal Products (COMP), and the Committee for Medicinal Products for Human Use (CHMP). Regulators at EMA aim to establish the value of RWE for regulatory decision-making by 2025.
The EMA’s Big Data Steering Group (BDSG) has been leading the efforts on RWE issues. In July 2022, the BDSG adopted a new work plan outlining its goals through 2025. Several pilot programs are currently ongoing, with the aim of strengthening the regulatory system’s ability to integrate data analysis and improve decision-making. The pilots aim to assess RWE’s ability to improve treatment outcomes, facilitate early patient access to new treatments, and accelerate drug development.
The recently released status report from BDSG and EMA provides an update on the RWD pilots. The report categorizes the different studies and explores their use and progress. The pilots use different data sources, including in-house databases, the DARWIN EU network, and a framework contract network. The majority of the studies were conducted via the in-house pathway, using EMA’s access to patient medical records. However, there were challenges in finding feasible topics for the RWE pilots, with only about half of the selected topics being executed.
The report highlights the pros and cons of each RWE generation method. The in-house studies were the most utilized method, as they provided accessibility to patient records from various care settings across the E.U. However, limitations in the data, such as lack of specificity and coding granularity, were acknowledged. The DARWIN EU network and the framework contract network had fewer studies but provided access to a larger network of data sources.
Overall, the report provides a status check on the progress of the E.U.’s efforts to establish an RWE framework for regulatory decision-making. While there have been challenges in finding feasible topics for the pilots, the ongoing programs aim to generate valuable evidence for decision-making. The findings from these pilots will contribute to the official 2025 regulatory report on RWE decision-making.
