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But despite showing a tightening of focus on clinical trials and evidence, the annual report doesn’t paint the full picture.
The coming into force of the EU’s Clinical Trials Regulation (CTR) early this year “represents a major milestone for clinical research in the EU,” according to the just-published annual report for 2021 of the European Medicines Agency (EMA).1 And it should work well for everyone, the agency implies, suggesting that the engagement of national authorities and trial sponsors throughout the development of the legislation will ensure that the new system of harmonization of assessment and supervision processes for trials is in line with user needs. It is backed up by new monitoring and coordination tools for a companion regulation on clinical trial safety that came into effect on the same date, stipulating how member state authorities should cooperate on safety assessment on the basis of the clinical trials regulation.
Clinical trials are also highlighted in the report in relation to the Accelerating Clinical Trials in the EU (ACT EU) initiative, launched in late 2021 with the aim to “transform how clinical trials are initiated, designed and run,” building on “the momentum of the CTR to further promote the development of high quality, safe, and effective medicines.” It is designed, says EMA, to reinforce the European environment for clinical trials while at the same time keeping protection high for trial participants and ensuring data robustness and transparency. What is also clear, although less explicit, is that ACT EU is an attempt to win back studies that are increasingly shifting abroad—or, as EMA more obliquely puts it, “to further develop the EU as a focal point for clinical research and to better integrate clinical research in the European health system.” Ten priority actions identified for 2022/2023 include enabling innovative trial methods, modernizing oversight and supporting the modernization of good clinical practice. Another priority actions will be to establish a multi-stakeholder platform “where all voices, including those of patients, can contribute to enabling better clinical research.”
Elsewhere in the annual report (alongside extensive coverage of EMA’s involvement in tackling COVID-19), a section provides an update on EMA plans to build up European regulatory capability and capacity “to deliver robust evidence for benefit-risk decision-making.” Progress during 2021 included projects to strengthen scientific advice on products under development, support to marketing authorization assessments, and delivery of expert methods advice and data analysis for medicines already on the market. DARWIN EU, the EU’s nascent Data Analysis and Real-World Interrogation Network now being developed to provide real-world evidence from across Europe on diseases, populations, and the uses and performance of medicines, is intended to provide European regulators with data applicable for decision-making throughout the life of a medicine. EMA will request studies to support itsscientific evaluations and regulatory decision-making, providing a crucial link between real-world data studies and benefit-risk decisions. An advisory board is already functioning composed of regulators, patients and healthcare professionals, and payer and health technology assessment organizations.
The “Big Data Steering Group,” set up by EMA and national authorities, is also acting on the agenda it set itself with its work plan through to 2023, including increasing the utility of big data in regulationby boosting data quality and study methods. Its eleven priorities relate in particular to data standardization, artificialintelligence, and real-world meta-data.
The report, with its stilted formal language obligatory in set-piece publications of this nature, provides some signposts to EMA priorities, but fails to convey the vigorous sense of ambition and even excitement that many of its officials display in person in discussing the agency’s future. Nor of course does it dwell on the acute difficulties that the organization and its staff have faced during the twin challenges of COVID and the forced removal from London, or on the real funding challenges it now faces as additional responsibilities are heaped on it by the EU (and more recently by a demanding public). The consequences of all of that unspoken ambition—and exasperation—will be more clearly revealed in what happens in the coming years rather than in a formal report on what happened last year.