Objectives: Real-world evidence (RWE) is valuable in supporting regulatory and HTA decisions, however, the actual contribution in approvals remains elusive. This study aims to review RWE approaches and use in oncology medicines approvals in Europe and understand cohesion and discrepancy in acceptance of the RWE by EMA and European HTA bodies.
Methods: This scoping review involved a search of the EMA database, NICE, G-BA and HAS websites to identify final reports and appraisals for oncology medicines with references to RWE. The selection was guided by research terms associated with RWE study designs, data sources and outcomes. Qualitative analysis was used to systemize the data. Case studies assessed by more than one agency were selected for comparative assessment of RWE approach, use and acceptability.
Results: RWE was mainly leveraged as an external control for indirect treatment comparisons or contextualization to support clinical trial results by the EMA, NICE, G-BA and HAS. However, this approach was mostly rejected due to methodology biases. Comparative assessment of RWE acceptability for the same oncology medicines across agencies suggest discrepancies between EMA and European HTA bodies, and amongst NICE, G-BA and HAS.
Conclusions: There is diverging acceptance of RWE in EMA and European HTA bodies with no clear consensus on the most effective way to leverage RWE in approvals. With the introduction of joint EU Joint Clinical Assessment in 2025, it is crucial for European HTA bodies and EMA to develop synergetic standards for the use of RWE to ensure equitable and timely access to medicines.
Keywords: comparative assessment; decision making; health technology assessment; oncology medicines; real-world evidence; regulatory.
Copyright © 2024. Published by Elsevier Inc.