Review of experience with real-world data studies in medicines assessment
date: 21/06/2023
The report establishes that real-world evidence (RWE) from studies led by regulators can complement evidence from other sources, such as randomised clinical trials. Furthermore, RWE can support both pre-authorisation and post-approval assessments of EMA’s scientific committees and working parties.
The report covers the period from September 2021 until the first anniversary of the Data Analysis and Real-World Interrogation Network - DARWIN EU® on 7 February 2023. The detailed analysis focusses on the pilot studies and does not include the EMA funded COVID-19 studies nor the routine PRAC impact studies. During this period, 61 real-world data (RWD) research opportunities were identified, 30 pilot studies were initiated and 27 completed.
The review considers studies conducted using EMA’s three pathways for RWE generation:
- 25 studies were performed by a team of pharmacoepidemiologists and data scientists at EMA using six in-house databases containing mainly primary care medical records from European countries;
- Four studies were initiated through DARWIN EU®;
- One study was commissioned to one of the eight research consortia with which EMA has framework contracts in place to procure scientific research studies.
Studies performed included safety, drug utilisation and disease epidemiology studies, as well as studies to inform 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, for example because the medicines or the outcomes of interest was not adequately captured in the available databases, or due to procedural time constraints.
The report provides a set of recommendations to address identified opportunities and challenges. These include the need for wider access to additional and complementary data sources, strategies to further accelerate RWE generation as well as earlier identification of research needs, tools to further build capability and capacity for RWE generation and close collaboration with decision-makers and other stakeholders.
The learnings and recommendations arising from the review will feed into the work of the Big Data Steering Group and inform the further establishment of DARWIN EU®.
The report was discussed during the Multi-stakeholder workshop on Real World Data (RWD) quality and Real World Evidence (RWE) use on 26-27 June 2023.
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