OBJECTIVES: This research aims to understand the payer perception and impact of oncology-related endpoints beyond overall survival (OS) in health technology assessment (HTA) submissions and payer decision-making.

METHODS: Quantitative research was conducted using a web-enabled, 15-minute questionnaire to collect payer insights on the evolving use of non-OS endpoints in oncology drug assessments in France, Germany, Italy, Spain, and the UK from a total of 20 stakeholders. Survey questions evaluated key drivers for payer acceptance of non-OS endpoints, including type of endpoints of interest and circumstances under which they may be accepted.

RESULTS: OS is generally seen as the most patient-relevant and clinically robust endpoint in oncology drug assessments. However, OS data collection is not always feasible as it requires larger and longer trials, delaying access to innovative medicines, and it may not capture the broader priorities of healthcare professionals (HCPs) and patients. Nonetheless, acceptance of non-OS endpoints in oncology trials remains limited among EU payers, and there is a lack of consensus on their value and accuracy to capture clinical benefits. There is a general preference for time-to-event endpoints such as progression-free survival (PFS) and disease-free survival (DFS) among payers, followed by patient-reported outcomes (PRO) and quality of life (QoL) as these endpoints measure additional morbidity benefits. Response rates, particularly biomarker-driven endpoints, are seen as the least meaningful as these are not considered sufficiently robust. There is opportunity to increase payer acceptance of non-OS endpoints by establishing guidance on their use, with initiatives such as the ongoing collaboration between NICE, SMC and CADTH to standardize the use of surrogate endpoints in cost-effectiveness analyses.

CONCLUSIONS: While OS is still considered the ‘gold standard’ endpoint in oncology trials, prioritizing alternative high-impact endpoints in the trial protocol with sufficient statistical power could demonstrate clinical benefits and expedite patient access to novel oncology therapies.