OBJECTIVES: HTA bodies / payers have used overall survival (OS) as the main measure of a medicine’s efficacy. However, reliance on OS poses three challenges. First, time to mature, statistically significant OS is longer in clinical trials for early-stage cancers than for metastatic disease. Second, the vulnerability of OS to confounding during this time may not accurately reflect the benefit of a medicine assessed solely on OS data. Finally, other endpoints more effectively measure important outcomes for patients or outcomes that better characterise the disease and treatment setting. As such, there is a need to improve value recognition of oncology-relevant endpoints (OREs) beyond OS in HTA / payer decision-making.

METHODS: This study investigates perceptions of OREs by 13 interviews with physicians, patient advocacy groups and former payers in US and Europe; 3 roundtable discussions; and a literature review.

RESULTS: OREs beyond OS, including time-to-event, response-related endpoints and patient-reported outcomes, have value as earlier indicators of a medicine’s efficacy and as standalone indicators of clinical and patient benefit. Although many OREs beyond OS are accepted by regulators, HTA bodies / payers may undervalue these endpoints due to perceived uncertainty they translate into long-term benefits for patients and healthcare systems. Several actions are recommended. Early cross-stakeholder engagement should agree the most suitable OREs by disease and treatment setting, evaluate the existing evidence base supporting the surrogate or standalone value and identify research needs to address gaps. Alignment between stakeholders can then inform standardized methodologies to collect them, ensuring data consistency and comparability.

CONCLUSIONS: Increased value recognition of OREs beyond OS in HTA / payer decision-making can improve timely access to life-improving medicines, ensure optimal outcomes for patients. Cross-stakeholder collaboration can support increased ORE incorporation in value assessment frameworks and HTA / payer decision-making that results in timely patient access to innovation medicines.