OBJECTIVES: Precision oncology (PO) treatments represent a new frontier in oncology, with targets on specific tumor attributes, like genetic mutations, molecular pathways, and biomarkers, offering specialized diagnoses, tumor-agnostic (TA) treatments, and improved outcomes. Regulatory and health technology assessments (HTAs) of PO treatments are challenging due to limited sample size and expedited trials. Real-world evidence (RWE) is one potential approach to supplement evidence packages, but European HTA bodies may not align on the utility of RWE for reimbursement decisions. This survey updates understandings in payer perspectives on RWE in HTA submissions as new RWE guidance have been released across Europe.

METHODS: Within the last two years, web-based surveys to payers with experience in HTA and reimbursement decision-making in oncology in Europe were administered via the Rapid Payer Response (RPR^TM) platform. Respondents in a new survey of 25 payers in France, Germany, Italy, Spain, and the UK were asked their perceptions of RWE to identify opinions shifts from previous years and to suggest useful RWE study designs in evaluations of PO treatments.

RESULTS: Previous payer responses varied by country and region, but most payers reported limited RWE impact on decision-making, particularly at initial assessment. Supplementing single-arm trial data with RWE-derived external control arms contextualized trial data, but rarely influenced the HTA outcome or price. Payers highlighted the appropriateness of RWE in re-evaluation decisions following conditional reimbursements and expressed preference for national or regional data. This survey will highlight perception shifts based on the greater demand for RWE in regulatory and payer submission packages.

CONCLUSIONS: With more evaluations of PO treatments, RWE may become more common in reimbursement submission packages. Providing high-quality data to HTAs is necessary to improve acceptability of evidence. While hurdles to RWE approaches still exist, sponsors should collaborate with HTA bodies to improve design and execution of studies to maximize acceptability by payers.