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Accompanied by a press release, the EMA has published a document outlining a framework for establishing Real-world evidence (RWE) data in regulatory decision-making, based on the findings of some 30 studies initiated by the EMA and DARWIN EU®. The document, titled "Real-world evidence framework to support EU regulatory decision-making," describes lessons learned from agency-led studies between September 2021 and February 2023.

Data Analysis and Real World Interrogation Network (DARWIN EU)

In pharmacovigilance, it is common practice to use routinely collected data about a patient’s health status or the delivery of healthcare from a variety of sources other than traditional clinical trials to support decision-making. However, the use of RWE is less established in earlier stages of medicines development.

RWE from studies conducted under the authority's responsibility can complement evidence from other sources, including clinical trials. Moreover, RWE can support both pre-authorization and post-approval assessments. However, more effort is needed to better anticipate the need for such studies and to speed up their initiation to ensure that regulators have access to RWE in a timely manner.

The report covers the period from September 2021 until the first anniversary of the Data Analysis and Real-World Interrogation Network DARWIN EU® (7 February 2023). During this period, 61 RWD research opportunities were identified, 30 studies initiated and 27 completed.

The report on the experience gained with regulator-led studies considers the following studies conducted via the agency’s pathways for RWE generation:

• 25 studies were initiated by EMA through a team of pharmacoepidemiologists and data scientists using databases containing mainly primary care medical records from European countries;
• Four studies were initiated via DARWIN EU®;
• One study was commissioned to a research organization with which the EMA has a framework contract in place to commission research.

Studies performed included safety, drug utilization and disease epidemiology studies, as well as studies on the design and feasibility of clinical trials and clinical management. Most studies focused on conditions diagnosed or medicines used in the primary care setting. For about a third of the research topics, a study was not considered feasible, e.g., because the medicines or the outcomes of interest were not adequately captured in the available databases, or due to procedural time constraints.

However, further work is needed to fulfil the European Union’s vision to enable the use of RWE and establish its value across regulatory use cases by 2025. The learnings and recommendations arising from the review will feed into the work of the Big Data Steering Group and further inform the establishment of DARWIN EU®.