Real-world evidence (RWE) from studies led by regulators can complement evidence from other sources including clinical trials. RWE can support both pre-authorisation and post-approval assessments of EMA’s scientific committees, working parties and national competent authorities. 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.

These are some of the findings of a report published today on the Real-world evidence framework to support EU regulatory decision-making: Report on the experience gained with regulator-led studies from September 2021 to February 2023 in the past year and a half. The report is part of the Agency’s efforts, alongside the European Medicines Regulatory Network (EMRN), to enable use of RWD in regulatory decision-making.

In pharmacovigilance, it has become mainstay 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.

Overseen by the EMA-HMA Big Data Steering Group, EMA and EMRN are working towards setting up a sustainable framework that enables the use and establishes the value of RWE in decision-making across the entire product lifecycle.

Since the completion of an earlier pilot with the Pharmacovigilance Risk Assessment Committee (PRAC) in 2021, EMA has engaged in various pilots with its other scientific committees and working parties. The pilots aim to explore the need for and usefulness of RWE generated by EMA to support regulatory decisions. While the pilots continue, the report takes stock of the experience gained so far with regulator-led RWD studies. It focuses on studies conducted in addition to those performed in response to the COVID-19 public health emergency and the Pharmacovigilance impact strategy.

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. During this period, 61 RWD research opportunities were identified, 30?studies initiated and 27 completed.

The review considers studies conducted via EMA’s three pathways for RWE generation:

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

While most research topics were related to scientific assessments of the PRAC, research opportunities were identified across the spectrum of EU decision-makers, including one pilot study in preparation via DARWIN EU® to support health technology assessment bodies and payers organisations.

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 conducted 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 were 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.

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®.

Notes

The report will be discussed during the Multi-stakeholder workshop on Real World Data (RWD) quality and Real World Evidence (RWE) use on 26-27 June. Interested stakeholders are invited to follow the live broadcast of the workshop.

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