Struggling Uphill to Common European Views on Data and Real-World Evidence

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Applied Clinical Trials

A high-level working group of European regulators is trying to pull together a more coherent approach to real-world evidence-and is running into difficulties with the wide range of initiatives currently underway.

A high-level working group of European regulators is trying to pull together a more coherent approach to real-world evidence-and is running into difficulties with the wide range of initiatives currently underway.

Experts from the European Medicines Agency and the Heads of Medicines Agencies are hoping to develop a common position and some guidance that could bring greater order to the confused European scene. The underlying aim is to increase access to data-for researchers and drug developers, but also for regulators, health technology assessment bodies, and pricing and reimbursement bodies.

But they are confronted by a huge range of initiatives, diverse definitions, multiple sources, a variety of agents collecting data, and a similar variety of players with a need for it.

The group is working from the concept that 'Big data' should denote large data sets from any source, while Real-world data (RWD) should cover any type of health-related data not collected in randomised controlled trials but derived from a diverse population in real life settings.

There is plenty of data out there, it finds-from registries and other types of observational studies, from extensions of clinical trials, from medical health records, from databases held by health insurance agencies or hospitals, and, increasingly, data generated from mobile applications. And it is being collected by many different actors in the healthcare system: manufacturers, learned societies, patients’ organizations, hospitals, healthcare professionals in routine practice, and others.

The new approach is based on addressing the needs of what it identifies as the key target groups-healthcare professionals, regulators, health technology assessment bodies, and policy-makers.  It will be based on ongoing data collection efforts within societies of clinicians and the synthesis of results from EU-funded activities such as the H2020 research programme, projects, or projects run by the Innovative Medicines Initiative. By a process of judicious selection from the many data collection operations in Europe, the group envisages setting up some pilot projects to get a clearer idea – particularly against the background of the digital transformation of health and care of how to connect and share data best for its target audiences.


Out of all of this the end product could-or should-be the development of common data model in Europe that includes criteria for validation for when data is presented in support of regulatory decision making. But so far the interim messages from the project are cautious. They point to the difficulties in getting data rapidly if systems are going to be more flexible to take account of wider sources. "Validation cannot be left to chance", insists EMA every time the question is discussed. There are attractions in the Sentinel system used by the FDA, which provides the authorities with a high level of control, but for the EU such a system would not only be costly, but would hardly fit with the European system, where there is no single authority that can exert the same level of control. Any future European framework must be sustainable with a governance structure that respects data privacy obligations and involves appropriately all stakeholders, say officials involved in the working group. 

Right now, an HMA-EMA joint task force is aiming to report on Big data by the end of this year, and to provide recommendations highlighting opportunities, challenges and risks.

To add to the challenges, the EMA's move to Amsterdam in 2019 complicates things further. Because of uncertainties about staff losses and the many other relocation implications, EMA has already indicated that all activities on real world data, big data, and registries for the next two years will need to be prioritized in the context of the agency's strict business continuity planning so that it can at least maintain its core activities of evaluating medicines.


Peter O'Donnell is a freelance journalist who specializes in European health affairs and is based in Brussels, Belgium