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Legislative efforts seek a common framework in accessing and applying real-world data and evidence in clinical studies.
Discussions on current European legislative initiatives edge forward in drug approval and the use of health data, and the potential for exploiting real-world evidence (RWE) is receiving increasing attention from researchers and regulators. European innovators are looking forward to what they see as "a large number of high-quality data sets that can be accessed and shared for research and innovative purposes that ultimately improve the lives of European patients." That is the view of The European Confederation of Pharmaceutical Entrepreneurs (EUCOPE), the body representing smaller biopharmaceutical research companies in Europe.
The legislators' intention is clear enough as a concept. The draft regulation on reforming the EU authorization process1 says that the companion proposal on the European Health Data Space (EHDS)2 "will provide a common framework across member states for access to high-quality real-world health data (RWD)," it says, going on to speak of promoting “progress in R&D, comparative clinical assessments, and regulatory decision-making."
For industry, there is evident merit in the use of RWE to complement clinical studies, and particularly in the case of many novel therapies to provide answers to questions that cannot be addressed by randomized clinical trials. A EUCOPE working group on digital health has been energetic in its exploration of the possibilities—and pitfalls—of regulation in this area, and the group's Leander Vranken recently outlined his view of how the opportunities can be best taken advantage of.
As he puts it, EHDS could optimize and widen the secondary use of health data through its envisioned electronic health data sharing mechanism, which would allow industry innovators to request access to health data from the new category of “data holders,” such as hospitals, for the purposes of science, research, and innovation. And because EHDS will set up a health data ecosystem with clear data collection standards, it will—if it comes into effect in an optimal fashion—provide for the robust RWE generation requires from the routine collection and analysis of large numbers of high-quality data sets. For the smaller firms in EUCOPE's membership, the common network of health data sharing would lower the barriers for the use and reuse of high-quality health data.
Another of the prominent examples of this growing engagement is a group styling itself RWE4Decisions, created by the Belgian health insurance body INAMI, but comprising a broad range of the European healthcare community, including notably more than a dozen national health technology assessment (HTA) bodies, payers, and regulatory agencies, but also extending to clinicians, patient groups, researchers, registry-holders, data analysts, industry, and academic experts. Its focus is on the sharp end of using RWD—where decisions are made on whether to reimburse new medicines. Its extensive stakeholder membership is allowing it to explore what RWD can be collected for highly innovative technologies—when, by whom, and how—so that it can be used to generate RWE for decision-making by healthcare systems, clinicians, and patients. It works on the basis that RWE generation is a shared responsibility and should be pre-specified and planned with all stakeholders.
The prospects for using RWE have also been welcomed by leading members of the European Parliament. Stelios Kympouropoulos, a Greek psychiatrist who is one of the center-right European People’s Party (EPP) group—and who, as a spinal muscular atrophy patient, understands well the challenges of rare diseases. A champion of RWD as a source of "invaluable insights into the effectiveness, safety, and value of medical treatments and interventions when applied in real-world settings," he sees the combination of RWD within EHDS as a valuable support for research.
Specifically, he recognizes the benefits that can occur in rare disease research, notably in the creation of rare disease registries, to improve rare disease diagnosis and treatment and to enhance patient engagement and participation in research. But he raises what he calls a "pivotal question": are the member states adequately prepared to confront the challenges of infrastructure, policies, and regulations on data sharing and technical capabilities? "It is crucial," he concludes, "for stakeholders to join forces and work together."
It is a conclusion that, regrettably, seems to encapsulate so many of the dilemmas in European health policy.
Peter O'Donnell is a freelance journalist who specializes in European health affairs and is based in Brussels, Belgium.