Presentation (CTI)

OBJECTIVES: Real-world evidence (RWE) is increasingly used to support regulatory decision-making, particularly in oncology where data is sparse and comparative arms may be unethical. This study quantified how RWE has been used to support efficacy evidence in European Medicines Agency (EMA) approvals of oncology medicines.
METHODS: Oncology medicines granted marketing authorization by the EMA after January 1, 2023 were identified. Biosimilars, generics, and supportive therapies were excluded. A structured review of European Public Assessment Reports (EPARs) was conducted to identify instances where RWE was used to support or supplement trial-based efficacy evidence. RWE was then categorized by data source, intended use, and regulatory acceptability.
RESULTS: Of the 35 eligible oncology therapy approvals, real-world evidence was used to support efficacy claims in only 8 cases (23%). While Lung cancer therapies were the most approved (N = 6), RWE to support Efficacy was used only in one instance (17%). Among medicines with orphan drug designation (N =10), 30% incorporated RWE to support efficacy evidence. The most frequently cited RWE data sources included patient registries (N = 3; 38%) and electronic health records (EHRs) (N = 3; 38%). RWE was primarily used either to benchmark trial outcomes (N = 4; 50%) or as formal comparison (N = 3; 38%). In all instances, RWE was accepted as supportive rather than primary clinical evidence. EMA considered RWE evidence to be inadequate in over 35% of the submissions.
CONCLUSIONS: RWE is increasingly leveraged in EMA oncology approvals and accepted as supportive rather than primary evidence. Harmonizing standards and improving evaluation frameworks will be key to increasing RWE acceptance by EMA.