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

Puntos clave

• 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.

Resumen