What question did this study set out to answer?

To evaluate the variability and evidentiary strength of pivotal trials for drug approvals by the European Medicines Agency from 2020 to 2023.

April 10, 2026

Evidence supporting European Medicines Agency drug approvals (2020–2023): A cross-sectional study of trial design and outcomes

Key Points

To evaluate the variability and evidentiary strength of pivotal trials for drug approvals by the European Medicines Agency from 2020 to 2023.
Conducted a cross-sectional analysis of pivotal trials for drug approvals.
Assessed trial designs, including prevalence of randomized controlled trials and surrogate endpoints.
Reviewed primary endpoints of trials to gauge regulatory thresholds.
Majority of trial designs were randomized controlled trials.
Surrogate endpoints were frequently used, indicating a reliance on indirect measures of effectiveness.
10% of drug approvals resulted from trials that did not meet primary endpoints, reflecting regulatory discretion.

Abstract

Our findings reveal substantial variability in the design and evidentiary strength of pivotal trials supporting European Medicines Agency approvals between 2020 and 2023. While the majority of studies were randomized controlled trials, reliance on surrogate endpoints was common. That 10% of approvals were based on pivotal trials with null primary endpoints highlights the nuanced role of regulatory judgment in therapeutic evaluation. These findings prompt reflection on evolving evidence standards in drug regulation and underscore the need for transparency and consistent justifications.

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Cite this study

Siebert et al. (Tue,) studied this question.

synapsesocial.com/papers/69d894ec6c1944d70ce05d55 — DOI: https://doi.org/10.1177/17407745261437363

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Authors

Maximilian Siebert

Harvard University

Laura Caquelin

Karolinska Institutet

Florian Naudet

Inserm

Journals

Clinical Trials

Actions

Institutions

Harvard University

Yale University

Inserm

References and Citations

Connected Papers

Building similarity graph...

Analyzing shared references across papers

Evidence supporting European Medicines Agency drug approvals (2020–2023): A cross-sectional study of trial design and outcomes

Key Points

Abstract

Citation Network

Connected Papers

Discussion

Cite this study

Also consider

Authors

Journals

Actions

Institutions

References and Citations

Citation Network

Connected Papers

Discussion

Also consider