Updated report summarizes EMA's progress in utilizing real-world data for EU regulations

The European Medicines Agency (EMA)’s latest report highlights progress in using real-world data (RWD) for EU regulations, noting 41 studies, expanded data partners, and advancements in integrating real-world evidence (RWE) into regulatory decisions.

Building on their previous report, the EMA has shared their experience with regulatory-led studies using RWD to support EU regulatory decisions for the period from February 8, 2023, to February 7, 2024. This report encompasses the second year of DARWIN EU, reflecting continued advancements in the integration of RWE into regulatory frameworks.

The report, “Real-world evidence framework to support EU regulatory decision-making: 2nd report on the experience gained with regulator-led studies from February 2023 to February 2024,” highlights significant progress in EMA's efforts to enhance the use of RWD for regulatory decisions. Peter Arlett, co-chair of the Big Data Steering Group, underscored this progress:

“We are in the business of excellent clinical evidence. Bigger, better, and more impactful clinical trials are one part of this while enabling the use and establishing the value of RWE is another. That is our vision, and we are on track to achieve it.”

Key findings and developments

During the reporting period, EMA conducted a total of 41 studies, with 22 completed and 18 ongoing. This represents a broad array of research topics, including safety studies, drug utilization, disease epidemiology, and vaccine effectiveness. Notably, the DARWIN EU network, which has expanded from 10 to 20 data partners, now provides access to data from approximately 130 million patients across 13 European countries. This expansion reflects EMA’s commitment to leveraging extensive RWD to support regulatory decisions.

Among the significant studies, 13 focused on vaccine safety and effectiveness, including investigations into COVID-19 and Mpox. For example, one study examined the risk of suicidal ideation associated with GLP-1 receptor agonists, providing crucial insights that helped EMA conclude there was no causal link between these medications and increased risk of self-harm. Additionally, two pioneering studies addressed the demand and stock levels of critical medicines, aimed at improving the management of medicine shortages.

Emer Cooke, EMA’s Executive Director, highlighted the progress made:

"We’ve come a long way since 2021, when we set out a vision to enable the use and establish the value of RWE in regulatory decision-making by 2025. We’re on track to achieve this, and the work continues."

DARWIN EU and future directions

DARWIN EU has proven to be a central component in EMA’s RWE generation strategy. In its second year, the network has refined its processes, including the development of standard analytical pipelines and the onboarding of new data partners. The network’s ability to conduct studies rapidly – such as those using primary data collection for vaccine safety – demonstrates its growing efficiency and impact.

However, the report also notes challenges. Approximately one-third of new research topics were deemed infeasible due to limitations in available data, particularly in rare disease settings. This underscores the ongoing need for enhanced data access and integration across diverse healthcare settings.

Conclusions and future outlook

The report underscores EMA’s continued efforts to integrate RWE into regulatory decision-making, aligning with their broader strategic goals. As stated in the report,

“Overall, there is an increased diversity of requesters and use cases compared to the previous reporting period.”

The EMA's initiatives are expected to further enhance the value of RWE in regulatory practices, fostering more informed and timely decisions in the evolving landscape of medicine regulation.