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Europe: EMA study reveals need for RWE framework, submission structure

2021/09/16  RAPS Convergence

The European Medicines Agency (EMA) has identified a need for more consistency in how real-world evidence (RWE) is submitted, Xavier Kurz told attendees at RAPS Convergence 2021.

Kurz, the head of surveillance and epidemiology at EMA, said the agency reached the conclusion after analyzing the use of RWE in applications for marketing authorization and extension of indication in 2018 and 2019. The study, preliminary findings from which were shared at RAPS Convergence 2021, found RWE was used in 40% of marketing authorization applications (MAAs), mainly at the post-approval stage, and in 18% of extension of indication filings.

Immunosuppressants and oncology drugs made up more than one-third of the MAAs and extensions submitted with RWE. Kurz said the predominance of the two therapeutic areas may reflect the use of single-arm trials to seek approval and the reliance on post-authorization studies to show long-term safety and effectiveness.

Disease registries and hospital data were the two most frequently used data sources in applications for marketing authorization and indication expansions. Kurz said the position of registries at the top of the list was “a bit surprising” but it potentially validates the work EMA has done to increase use of the data source.

“The fact that disease registries were used in many studies is a bit reassuring to us. Several years ago, we started a project on registries in order to support and to encourage the companies to use these disease registries as a source of data and real-world evidence,” Kurz said.

While the study shows RWE is frequently being used in filings to EMA, it also revealed ways the agency thinks it can improve the use of the data source. Kurz identified the need for further work to evaluate the concrete impact and usefulness of RWE in evaluations and decision making as one of the lessons of the study. EMA plans to further analyze the criteria used to evaluate RWE and whether it took a consistent approach.

EMA has also identified a need for consistency at other steps in the process. Kurz highlighted the lack of a framework for using RWE in submissions and the absence of a structure for the way information is submitted as other lessons of the study.

“What is the purpose of real-world evidence? How is it presented? What is the information that is needed? There is a need for perhaps more consistency,” Kurz said. “And also the structure and the way real-world evidence is submitted needs attention and probably to be given more guidance in terms of standard definitions … or assessment of the quality by the applicants.”

EMA responded to the identification of limitations in the documentation covering RWE by starting a new phase of the study. In the second phase, a person at EMA is working full time on the additional objectives identified in the original work.

Kurz’s presentation was followed by a talk by John Concato, the associate director for real-world evidence analytics in FDA’s Office of Medical Policy. Concato touched on some of the same themes as Kurz, such as the challenges of even defining what submissions are deemed to include RWE.

“Tracking and counting real world evidence is actually not straightforward, for reasons linked back to terminology issues … and for specific reasons. Should FDA rely on a sponsor’s designation of what is RWE? Or should we make our own determination based on an assessment of the actual methods? Should we include safety studies in that count? Or should we focus only on effectiveness studies?” Concato asked.

Concato went on to give examples of applications FDA deemed to include RWE, emphasizing that the agency began reviewing the data source well before the 21st Century Cures Act tasked it with setting up a program focused on real-world evidence.

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