Real-world evidence (RWE) is valuable in supporting regulatory and health technology assessment (HTA) decisions; however, the actual contribution to approvals remains elusive. This study aimed to review RWE approaches and use in oncology medicine approvals in Europe and understand cohesion and discrepancy in the acceptance of the RWE by the European Medicines Agency (EMA) and European HTA bodies.
This scoping review involved a search of the EMA database, National Institute for Health and Care Excellence (NICE), Gemeinsamer Bundesausschuss (G-BA), and Haute Autorité de Santé (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.
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 suggests discrepancies between EMA and European HTA bodies and among NICE, G-BA, and HAS.
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 the joint European Union 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.
The study explores how real-world evidence (RWE) is used in the approval of cancer treatments in Europe. The research is important because it addresses the growing incorporation of RWE into regulatory and health technology assessment (HTA) decisions, which can help patients gain quicker access to new therapies. The study reviews the practices of the European Medicines Agency (EMA) and several HTA bodies, including the National Institute for Health and Care Excellence (NICE), Gemeinsamer Bundesausschuss (G-BA), and Haute Autorité de Santé (HAS).
The findings show that RWE is often utilized as a supplementary tool to support data from clinical trials, especially in oncology. However, the acceptance of RWE varies widely among the different organizations, leading to inconsistencies in how it is used in decision making. For example, while the EMA often accepts RWE as supportive evidence, other bodies like G-BA are more critical and may reject RWE due to concerns over its methodological quality.
Between 2020 and 2022, RWE appeared in about one-third of EMA's final reports on oncology medicines, and nearly half of the HTA appraisals by NICE included RWE. Despite this progress, the study highlights significant gaps. For instance, there are discrepancies in how RWE is perceived and utilized across different agencies. These differences can hinder the overall effectiveness of RWE in informing treatment decisions and ultimately affect patient care.
The article emphasizes the need for collaboration among regulatory bodies and HTA organizations to establish clearer and more standardized guidelines for using RWE. With the introduction of the European Union's Joint Clinical Assessment in 2025, it becomes even more crucial for these agencies to align their approaches to RWE. This alignment will help ensure that patients have equitable and timely access to innovative treatments.
In summary, while RWE holds promise for enhancing the approval process for cancer therapies in Europe, there are still significant challenges to address. Greater consistency in how RWE is evaluated and integrated into decision-making processes will be vital for improving patient outcomes and improving access to new medicines.