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Dissecting the EMA’s new annual report on milestones reached in clinical research and public health.
It was "another transformative year for the agency," says Lorraine Nolan, the recently-elected chair of the European Medicines Agency's (EMA) management board, in her foreword to its just-published annual report covering 2022.1 That's true enough—but it's really a matter of primus inter pares: so many years in the life of the agency have been transformative since Fernand Sauer became the first executive director of what was initially called the European Medicines Evaluation Agency in 1995.
Those with long memories will recall the excitement of the first multi-state applications for marketing authorization; the conflicts over quality of expertise in the CSP (the Comité de Spécialités Pharmaceutiques as the predecessor to the CMPH—before it became the CPMP); the subsequent challenges of integrating a wave of new member states—many with very distinct traditions of regulation—into the agency and its work; the revolving-doors shadow cast by the departure of a subsequent executive director into an industry-related job and the travails of restoring respectability when the appointment of the anointed savior-successor was annulled by a legal challenge that took many months to resolve; the disruption of Brexit and the hotly-contested process of choosing a new location for the agency; as well as many other dramas and a long record of solid work that built the international reputation the agency has acquired.
So in addition to the 89 new human medicines recommended for approval (including the first drug for the prevention of respiratory syncytial virus in newborns and infants), what claim can 2022 make to being a transformative year?
For those with a professional interest in clinical trials, quite a lot. 2022 was the year the Clinical Trials Regulation was implemented and the Clinical Trials Information System (CTIS) came into force, "setting the building blocks to reinvigorate and facilitate clinical research in the EU," as EMA's executive director Emer Cooke described it. "This is a major change, a move from entirely national approaches to a system with a single EU submission, coordinated assessment between the member states, and high levels of transparency never seen before for clinical trials," she claims. And although the use of CTIS remained voluntary in 2022, more than 200 initial clinical trial applications were authorized, and more than 200 were under evaluation with the new system at the end of the year.
2022 was also the year of the launch of the Accelerating Clinical Trials in the EU initiative, known as ACT EU, and aimed at "transforming how clinical trials are initiated, designed, and run." The official EMA line is that ACT EU will help "develop the EU as a focal point for clinical research and better integrate clinical research in the European health system," exploiting the momentum of the Clinical Trials Regulation. Simultaneously, the agency has been deepening the integration of data and analytics into its work, with new approaches to assessment and approval of therapies, and the evolution of DARWIN EU—the Data Analysis and Real World Interrogation Network designed to collect real-world evidence (RWE) from across the EU, and seen by Cooke as "a sea change for medicines regulation, feeding into a future European health data space."
As part of the Big Data Steering Group workplan, a two-year pilot project was launched last July to assess the merits of regulators analyzing raw data from clinical trials—both for speeding evaluations, with fewer questions being raised with applicants, and for better defining the target population. And data quality was the focus of new guidance, with a list of metadata to help developers identify and use real-world data sources and studies, and a good practice guide for using the planned metadata catalogue of real-world data sources.
2022 was also the year that EMA started operating under an extended mandate built on the role the agency played during the COVID-19 pandemic in crisis coordination and response. Building on the work in 2020 and 2021 to leverage collaborations with academics in observational research, EMA used real-world data from routine clinical practice to monitor the safety and effectiveness of COVID and mpox vaccines to generate RWE supporting the response to both outbreaks. Building on this experience, a process has been developed for the rapid procurement of studies in emergency situations, where rapid evidence is required to support public health and regulatory actions.
One transformation that has not yet happened is for EMA's funding to match its ambitions, with new legislation still under discussion in the EU’s legislative machinery. So Nolan, whose day-job is as the chief executive of the Irish Health Products Regulatory Authority, concludes her remarks with a plea that echoes what is now a refrain from her compatriot Cooke: "I would like to see continued investment in the sustainability of our network to ensure we have the resourcing and capacity to meet the regulatory needs of the EU population, both in normal times, and in a crisis."
That is one transformation that is not in hands of EMA itself.