EMA report on incorporating RWE in its decisions
MYO Health - Katerina Pilou
Εvidence generation has evolved significantly within the field of medical research and regulation. The traditional gold standard of randomized controlled trials (RCTs) has long been the cornerstone of clinical decision-making. However, a transformative shift is taking place, driven by the integration of Real-World Evidence (RWE).
This shift is reflected in the latest report that the European Medicines Agency (EMA) issued on DARWIN (Data Analysis and Real-World Interrogation Network). The report demonstrates EMA’s and European regulators’ ongoing efforts to establish a robust framework that integrates RWE into the regulatory processes for medicines. This ambitious initiative aligns with the broader European Medicines Regulatory Network (EMRN) and EMA’s Regulatory Science Strategy to 2025.
Since its inception in 2022, DARWIN EU has been a catalyst in generating RWE to support regulatory decisions. It provides a federated network of data, expertise and services, enabling the generation of high-quality evidence from real-world settings. The second year of DARWIN EU’s operation has seen significant expansion and progress. The data is not mathematically anonymized through differential privacy techniques; therefore, its use is limited to few authorized governmental or international public health organizations.
Over the 12-month reporting period, DARWIN EU onboarded 20 data partners, providing access to data from approximately 130 million patients across 13 European countries. This vast data repository includes information from diverse care settings, such as hospitals, national registries, and biobanks. By tapping into these sources, DARWIN EU has become the main RWE generation pathway for EMA, significantly overtaking the in-house route, as well as other frameworks.
The report reveals the impressive breadth of research conducted under DARWIN EU and other pathways, with 41 studies either completed or ongoing. These studies spanned 11 different therapeutic areas and were used to address safety signals, paediatric investigation plans and public health emergencies, among others. Notably, 63% of these studies were descriptive cohort studies, though a growing trend towards more complex analyses has been observed.
One of the key highlights is the increasing diversity of study requesters, including not just EMA’s scientific committees but also national competent authorities (NCAs), the European Centre for Disease Prevention and Control (ECDC) and health technology assessment (HTA) bodies. This indicates a growing recognition of RWE’s value across various decision-making processes.
Examples of impactful studies include those that informed the PRAC (Pharmacovigilance Risk Assessment Committee) on safety signals, such as investigating the association between GLP-1 receptor agonists and suicidal ideation in diabetes patients. Similarly, several studies explored vaccine safety and effectiveness, particularly in response to public health emergencies like COVID-19 and mpox.
Despite the progress made, the journey towards fully integrating RWE into regulatory decision-making is not without challenges. A significant proportion of study requests, particularly in rare disease settings, were deemed unfeasible due to limitations in data availability. For example, some outcomes or medicines were not adequately captured in existing databases, particularly in specialized care settings.
Nevertheless, DARWIN EU has made significant strides in improving the study feasibility assessment process, enhancing the accuracy of early feasibility counts and expediting study timelines. The network’s expansion into more countries and the onboarding of additional data partners from non-primary care settings has also increased the range of research topics that can be explored.
As EMA and its partners continue to refine the use of RWE, the vision remains clear: by 2025, RWE will be an integral part of the EU’s regulatory decision-making framework. DARWIN EU, in its second year, has made tremendous progress toward realizing this goal, expanding access to high-quality, real-world data and producing impactful evidence that informs crucial public health decisions.
With continued advancements in analytical pipelines, data standardization and cross-border collaboration, the future of RWE in Europe is bright. By integrating RWE alongside traditional clinical trial data, regulators are not only enhancing the robustness of their decisions but also paving the way for more responsive, patient-centred healthcare.
In conclusion, the integration of RWE represents a paradigm shift in how regulatory decisions are made. The integration of RWE in the regulatory processes will ensure that Europe remains at the forefront of evidence-based regulation, improving public health outcomes for its population.
You can read the full report at:
Image: Ceescamel, CC BY-SA 4.0 <https://creativecommons.org/licenses/by-sa/4.0>, via Wikimedia Commons