OBJECTIVES: In recent years, there has been considerable discussion around the acceptability of surrogate endpoints in oncology. However, other therapeutic areas also use such endpoints to measure patients’ improvement. It is particularly challenging to measure treatment effects when conducting clinical trials in acute conditions in the intensive care (ICU) setting. The objective of this research was to evaluate the acceptability of an endpoint related to arterial pressure as a primary endpoint in HTA submissions

METHODS: Country focus groups with (proxy) payers and leading ICU clinicians across EU4 and UK.

RESULTS: Payers do not consider surrogate endpoints in their clinical assessment. Across countries in scope, payers and clinicians aligned on the low acceptability of the arterial pressure-related endpoint as a primary endpoint as the mortality benefit cannot be demonstrated. Consequently, it cannot be considered a ‘patient-relevant’ endpoint. However, payers in France, Italy, Spain and UK showed an openness to considering alternative endpoints if the treatment demonstrates no detrimental effect on mortality. Economic models showing the reduction in ICU stay could support the value of a treatment, especially in budget impact and cost-effectiveness driven markets, and is considered key in order to understand its price potential.

CONCLUSIONS: Early engagement with European payers and health technology assessment bodies is critical to understand the requirements prior to designing clinical trials in challenging therapy areas to avoid negative pricing and reimbursement outcomes. Surrogate endpoints alone will not meet payers’ requirements as they are not deemed ‘patient relevant’. A cost-offset approach, demonstrating the potential savings to the healthcare system for ICU products, could be an acceptable substitute in some European markets, and is valuable data to collect as part of the clinical development plan.