Measuring What Matters: Practical Pathways for Personalized Endpoints and Patient-Centric Approaches in HTA Evaluation

Purpose: This workshop aims to discuss how personalized endpoints and patient-centric approaches can be effectively designed, implemented, and integrated into clinical trials, and explore their incorporation into health technology assessments (HTA). The session will offer practical tools, concrete examples, and expert insight to support broader adoption of patient-centered evidence in value assessment. Description: As healthcare systems move toward more patient-centric approaches, it is crucial to reflect individual patient experiences and treatment goals in evidence generation. Traditional clinical endpoints may fail to capture what matters most to specific patient subgroups, particularly in rare diseases, neurodevelopmental and chronic conditions, due to heterogeneity in disease burden and treatment response. HTA bodies face the task of balancing the need for robust, population-level evidence with the growing demand for patient relevance in value assessment. While the importance of incorporating the patient voice is widely recognized, practical implementation of personalized endpoints in clinical trials and HTA evaluations remains limited. This workshop will discuss both conceptual and practical aspects of personalized endpoints and patient-centric approaches. The panel will share qualitative and quantitative examples, including the Goal Attainment Scale (GAS), which captures individualized treatment goals, quantifies efficacy, and reflects patient priorities. Other examples will include adapting generic measures to specific diseases and patient populations. We will also discuss how such endpoints could be integrated into HTA frameworks. •Danny Yeh: Moderator, sharing an overview of personalized endpoints in value assessment. •Gunes Sevinc: Introduction to GAS as a structured, patient-centered endpoint used in drug development and beyond. •Hannah Staunton: Industry perspective on opportunities and challenges in implementation. •Dalia Dawoud: HTA methodologist stance on the use of personalized endpoints in current and future value frameworks. The session will include polling and live Q&A to engage the audience and advance patient-relevant HTA.