New EMA report on real-world evidence to support EU regulatory decision-making
The European Medicines Agency (EMA) has published a report presenting the experience they have gained over the past year and half in conducting studies using real-world evidence (RWE), containing new findings about the steps that need to be taken to improve feasibility of RWE studies on rare diseases at the European level.
RWE is an increasingly popular data source which can complement findings from more traditional studies, such as clinical trials, to support decision-making by regulatory bodies such as the EMA’s scientific committees and national competent authorities. It holds particular promise for areas like rare diseases, where it is particularly difficult to conduct robust clinical research. However, there are specific barriers to integrating RWE into such processes. This report examines studies conducted between September 2021-February 2023 to understand existing pathways for RWE generation within the EMA, as well as what challenges still need to be overcome.
The report identified three different RWE generation pathways through which studies were conducted. These were studies initiated by the EMA using databases containing European primary care medical records; studies undertaken within the framework of DARWIN EU®; and those commissioned to research organisations and consortia.
Despite these multiple pathways, it was found that none were sufficient to meet the data needs of rare disease research. Such studies were often deemed not feasible due to a lack of adequate data captured in databases, combined with procedural time constraints which were prohibitive of other pathways.
In order to overcome these challenges, the report recommends further work on widening access to more diverse and complementary data sources, such as hospital, claims and registry data. Additionally, efforts should be made to accelerate RWE generation, such as through increasing the use of a common data model, or developing a catalogue of standard data analyses. Doing so would broaden the possible applications of RWE for regulatory decision-making, thereby allowing a better realisation of the potential it holds for improving the lives of Europe’s rare disease community.
