Session (CTI)

Regulatory bodies are increasingly accepting surrogate endpoints to expedite the approval of new therapies, particularly in areas of high unmet medical need. Although these endpoints can streamline drug development, disagreements arise when assessing the value and coverage of such treatments since there may be no direct evidence of benefits to patients. Health technology assessment bodies and payers often remain unconvinced by surrogate data, citing a lack of robust evidence on patient-relevant or long-term outcomes. 

This plenary will examine methodological approaches for ensuring that surrogate endpoints used in accelerated regulatory and HTA decisions are scientifically robust and meaningfully reflect outcomes that matter to patients. This session will explore how early and continuous patient input can inform endpoint selection, validation strategies, and contextual interpretation of surrogate-based evidence. Speakers will discuss practical methods for aligning surrogate measures with patient priorities, as well as the challenges of operationalizing these approaches in time-constrained decision-making environments.