EMA’s real-world evidence progress report highlights expanding role of RWE in medicines regulation

The European Medicines Agency (EMA) has published its fourth progress report on the use of real-world evidence (RWE), highlighting increased study activity and the expanding role of routinely collected healthcare data in supporting medicines regulation and public health decision-making across the EU.

The Baseline

• The EMA’s fourth progress report highlights increasing use of RWE to support medicines regulation and public health decision-making across the EU.
• Regulator-led RWE activity has expanded, with more studies underway and growing use of routinely collected healthcare data through DARWIN EU®.
• The EMA has also updated its guidance on requesting RWE studies, reflecting the expanding role of regulator-led evidence generation in European medicines regulation.

According to the EMA, analyses based on real-world data (RWD) are intended to complement clinical trial evidence by addressing questions that may not otherwise be feasible to investigate. The agency states that:

"RWD can be used to achieve better informed and more efficient regulatory decision-making as a complement to existing evidence,"

while also helping to fill knowledge gaps, provide independent evidence, and generate analyses tailored to specific regulatory questions. The guidance also notes that regulator-led studies can provide “potentially faster evidence generation, reacting immediately to RWE generation needs,"

and avoid procedural delays associated with sponsor-led research.

The latest report indicates that regulator-led RWE generation continues to expand. During the reporting period, 108 research topics were assessed, and 88 studies were completed or remained ongoing, representing almost 50% more activity than during the previous reporting period. The agency also reported the launch of its first highly complex studies involving pregnancy and mother-child data linkage and oncology.

Many of these studies are delivered through DARWIN EU®, which has expanded to 40 data partners across 18 European countries and now provides access to data covering more than 250 million patients. As the EMA's primary infrastructure for RWE generation, the network supports regulator-led studies, including routine repeated analyses that provide updated evidence and monitor trends over time.

The report highlights the increasing contribution of these studies to regulatory activities. Around 30% directly supported a regulatory decision within an active procedure, while 44% contributed to improved clinical or methodological understanding. A further 15% supported preparedness activities relating to medicines shortages, public health, and antimicrobial resistance.

Alongside the progress report, EMA has updated its guidance on requesting RWE studies, describing the processes available to scientific committees, national competent authorities, health technology assessment bodies, payers, and other eligible EU decision-makers. The guidance explains that studies may range from analyses of disease prevalence, treatment patterns, and medicine utilization to more advanced causal inference studies and complex phenotyping approaches, depending on the research question being addressed.

The EMA states that its objective is to "embed regulator-led generation of RWE into the assessment process and to do this in a learning, evidence-based way," while continuing to expand data sources and analytical capabilities to support medicines regulation across Europe.