European Medicines Agency Publishes Second Report on Real-World Data Studies: Key Insights and Future Directions

The European Medicines Agency (EMA) has released its second comprehensive report on studies utilizing real-world data (RWD) between February 2023 and February 2024. This report highlights EMA's ongoing efforts, in collaboration with the European Medicines Regulator Network (EMRN), to integrate real-world evidence (RWE) into regulatory decision-making, supplementing the gold standard of randomized controlled trials (RCTs).

Key Highlights of the Report

Increasing Integration of Real-World Evidence (RWE): The EMA is committed to enhancing the role of RWE in regulatory decisions concerning the development, authorization, and supervision of medicines. This aligns with EMA's 2025 RWE Vision, which aims to better incorporate RWE alongside traditional RCTs.

Peter Arlett, Co-Chair of the Big Data Steering Group, emphasized this commitment, stating, “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.”

Expansion of DARWIN EU Network: The Data Analysis and Real World Interrogation Network (DARWIN EU) has become the primary RWE generation pathway, conducting the majority of studies during the reporting period. The network has expanded to include 20 partners and now has access to data from approximately 130 million patients across 13 European countries. This expansion has allowed for a broader scope of research, particularly in disease epidemiology, medicine use, safety, and effectiveness.

Study Output and Focus Areas: During the reporting period, 41 studies were conducted, with 22 completed and 18 ongoing. These studies predominantly used the DARWIN EU network (19 studies), followed by studies commissioned to external research organizations via the Agency’s research framework contract (14 studies), and in-house studies conducted by EMA (7 studies).

Most studies (63%) were descriptive cohort studies, addressing crucial research questions in disease epidemiology, clinical management, and the safety and effectiveness of medicines.

Challenges in Study Feasibility: The report identified several challenges in conducting studies, with approximately one-third of new research topics deemed infeasible. The primary reason for this was the lack of adequate data capture for the outcome of interest (52%). Other challenges included the absence of the medicinal product of interest in the database, insufficient granularity of data, and tight timelines.

RWE Generation Timelines: Among the RWE generation pathways, in-house studies were the fastest, with a median duration of just over three months. DARWIN EU studies had a median duration of eight months, while studies commissioned to external research organizations took the longest, with a median of 25 months.

Survey of National Competent Authorities (NCAs)

The report also included a survey of NCAs, revealing disparities in access to and capability for using RWD. While six of the eleven respondents had access to electronic health data, only three had conducted non-interventional studies. The survey highlighted the need for building capabilities within the network and improving data quality and completeness.

Future Considerations

Looking ahead, the EMA is preparing to enhance its ability to process personal health data, a capability that is expected to be bolstered by the European Health Data Space Regulation and revised EU pharmaceutical legislation. These advancements aim to improve the robustness of scientific assessments and support the verification of claims made by applicants or marketing authorization holders.

Conclusion

The EMA’s second report on real-world data studies underscores the agency’s progress in integrating RWE into regulatory processes, with DARWIN EU emerging as a central pillar of this effort. As the EMA continues to enhance its data processing capabilities, it is poised to play a pivotal role in shaping the future of medicine regulation in Europe, ensuring that regulatory decisions are informed by the most comprehensive and relevant evidence available.

For more detailed information, the full report can be accessed here.

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About Stuart McCully...

Stuart is a real world research (RWR) regulatory scientist and thought leader.?With his background as a pharmacologist working on traditional pre-approval clinical trials, he became frustrated with the operational inefficiencies and support gaps for non-interventional (observational) studies. As a result, Stuart created CHCUK which became a global leader in supplying regulatory and operational intelligence for non-interventional studies. Currently, Stuart is a co-founder of RWR-Regs, a regulatory compliance solutions platform for real world research.