Europe’s Regulators Plan Real Action on Exploiting Real-World Evidence


Newly unveiled workplan focuses on harnessing the impact of big data on drug development—and ultimately public health.

Given the central importance of data in clinical trials, it may seem perverse to be apologetic for focusing yet again on the subject in this column, but every journalist is naturally and professionally reluctant to test readers’ patience. However, here we go again…another element in Europe’s rapidly developing moves to make sense of new opportunities in drug development, and to bring some order to impending chaos.

A new workplan from Europe’s top drug regulators sets ambitious targets for getting much more out of data analysis over the next three years.1 “Coupled with rapidly developing technology, big data can complement the evidence from clinical trials by filling knowledge gaps on a medicine, and can help to better characterize diseases, treatments, and the performance of medicines in individual healthcare systems,” says the plan.

The plan, designed by the “Big Data Steering Group” set up by the European Medicines Agency (EMA) and the Heads of Medicines Agencies (HMA) in Europe, aims at using the evidence generated from big data to benefit public health by speeding drug development and improving treatment outcomes. It takes a decidedly positive view of the potential contribution of big data—in contrast to many more skeptical opinions currently circulating in European regulatory circles. “Knowing when and how to have confidence in novel technologies and the evidence generated from big data will benefit public health by accelerating medicines development,” it states, while laying out explicit deliverables and clear timelines.

The EMA’s flagship DARWIN program—the embryonic Data Analysis and Real-World Interrogation Network that is expected to transform the use of real-world evidence (RWE) when assessing medicines—is scheduled to conduct more than 100 studies per year by 2025. DARWIN will provide results of its analysis of real-world data (RWD) not just to regulators but also to bodies responsible for health technology assessments (HTAs) and payers. Four such studies, involving 10 partners, are to be initiated this year, with 16 studies and 10 additional data partners during 2023, rising to 50 studies by 2024.

To help the EU medicines regulatory network to make an informed decision on the place of raw data in regulatory decision-making, a proof-of-concept pilot will be performed to clarify the benefits and practicalities of access to individual patient data from clinical trials in the scientific assessment of medicines. The plan also envisions installing a data quality framework by the end of 2022 for the use of regulators, after some intensive consultation between now and then with patients, healthcare professionals (HCPs), regulators, the pharmaceutical industry, and academia. Training curricula on biostatistics, pharmacoepidemiology, and data science will be developed as from 2023, and a curriculum on genomics data will be adopted. Some of the modules will be made accessible topatients, healthcare professionals and academics.

A good practice guide on real-world metadata and a public catalogue of European RWD are on the list of deliverables, along with the development of analytics tools and standardized clinical trial protocols. Guidance will be strengthened through expert groups of specialists, initially in artificial intelligence and RWE, and collaboration in analysis of raw data from clinical trials will be promoted through a specifically created “cluster of excellence.” Reports on pilot studies on the use of RWE by EMA scientific committees will be published incrementally and concluded in 2025 with a report on RWE in regulatory decision-making.

There is more to come on data use—and not just on EMA and HMA planning. The legal framework that the EU is proposing for the use of all health data, the European Health Data Space, is now generating significant comment—and some significant pushback from the European health community. And that—I almost regret having to inform you—will have to form the subject of yet another column on data. Enjoy, meanwhile, the summer while it lasts.


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