Enhancing HTA: Surrogate Endpoints, Statistical Standards, and Their Impact on Patients

This session presents the rationale, objectives, and preliminary recommendations from the ISPOR Statistical Evaluation of Surrogate Endpoints for Health Technology Assessment (HTA) Decision-Making Task Force. Attendees will gain insights into existing and novel statistical methods for demonstrating clinical and economic value when surrogate endpoints are used for drug licensing, and how surrogate endpoints affect clinical decisions, patient access and outcomes, and strategies to mitigate uncertainty. Surrogate endpoints can be measured more quickly than target outcomes, reducing trial duration and facilitating earlier regulatory and HTA approvals. This, in turn, may accelerate patient access to potentially life-changing therapies. However, despite the availability of guidelines on use of surrogate endpoints from numerous HTA agencies, few HTA submissions explicitly provide evidence linking the treatment effect on surrogate endpoints to target outcomes. Lack of robust evaluation of surrogate endpoints can lead to increased uncertainties in HTA decision-making and delays to patient access. In this dynamic session, Dr. Heeg (6 min) will introduce the session and its objectives. Dr. Thorlund will discuss statistical approaches for evaluating surrogate endpoints, addressing prediction challenges, uncertainty, and emerging tools for less-than-ideal data scenarios. Professor. Lee will explore how surrogate endpoints are integrated into health economic modeling, detailing how they support predictions of treatment effects and their corresponding uncertainties. Dr. Stefani will discuss the broader implications for clinicians and patients when drugs are approved based on surrogate endpoints, weighing the decision to prescribe these novel therapies, despite uncertainty about their impact on target outcomes. The session will conclude with Dr. Heeg moderating a Q&A segment focused on the task force’s recommendations. This session is designed for researchers, payers, regulators. assessors, and industry professionals, offering a unique opportunity to explore the intersection of surrogate endpoints, statistical innovation, and patient-centered decision-making.