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Real-world evidence reported for clinical efficacy evaluation in European Public Assessment Reports of authorised

J W G Derksen1, D Martins-Branco2, A Valachis3

  • 1Julius Center for Health Sciences and Primary Care, Department of Epidemiology and Health Economics, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands.

ESMO Real World Data and Digital Oncology
|February 6, 2026
PubMed
Abstract

Insights

Real-world evidence (RWE) was used in 21% of European regulatory evaluations for targeted cancer therapies, showing an increasing trend. Most RWE served as supportive evidence, though studies often had a high risk of bias.

Area of Science:

  • Oncology
  • Regulatory Science
  • Health Economics and Outcomes Research

Background:

  • The precise role of real-world evidence (RWE) in the regulatory assessment of clinical efficacy for targeted therapies remains incompletely defined.
  • This study focuses on targeted therapies authorized in Europe for solid tumors.

Purpose of the Study:

  • To assess and describe the reported utilization of RWE for the clinical efficacy evaluation of authorized targeted therapies for solid malignancies in Europe.
  • To analyze the frequency, role, characteristics, and risk of bias of RWE used in regulatory submissions.

Main Methods:

  • Systematic review of European Public Assessment Reports (EPARs) for targeted therapies approved by the European Medicines Agency (EMA) between 2018 and 2022.
  • Data retrieval and analysis conducted in March 2023.
  • Evaluation of RWE frequency, role, study design, origin, and risk of bias.

Main Results:

  • Real-world evidence (RWE) was reported in the EPARs of 16 out of 75 (21.3%) authorized indications, with a rising trend observed from 2018 to 2022.
  • RWE was more frequently utilized for lung and breast cancer indications, antibody-drug conjugates, and conditional approvals.
  • In most cases (75%), RWE functioned as supportive or confirmatory evidence, primarily from retrospective, non-international, analytical studies, predominantly originating from the US, and often exhibiting a high risk of bias.

Conclusions:

  • Real-world evidence (RWE) is increasingly being incorporated into the regulatory evaluation of targeted therapies for solid tumors in Europe.
  • While RWE plays a supportive role, the methodological quality and risk of bias in existing studies necessitate careful consideration during regulatory decision-making.
Keywords:
European medicines agencycancermarketing authorisationoncologyreal-world evidencetargeted therapy

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