ISSUE: Patient preference studies (PPS) yield evidence about unmet needs and treatment outcomes that matter most to patients, which can inform pharmaceutical companies, health technology assessment bodies, payers, researchers, physicians, and patient organizations in understanding whether a product answers an unmet need and provides benefits that are meaningful to patients. While the potential is clear, there is lack of case examples and understanding on how the implementation and integration of PPS in decision-making along the medical product life cycle (clinical trial endpoint determination, regulatory/HTA assessment) can occur in practice.

OVERVIEW: Drawing upon experiences and results from PPS’ in diverse disease domains (e.g. multiple myeloma, Duchenne muscular dystrophy, inflammatory bowel diseases), speakers from different stakeholder organisations (European Medicines Agency, KU Leuven, Patvocates) will share their views (regulatory, methodological, patient) on how PPS can be robustly designed to provide evidence-based information regarding the unmet needs and treatment outcomes that matter most to patients how much, and how these results could be integrated to inform healthcare decision-making. The session will cover both qualitative (interviews, focus group discussions) and quantitative preference elicitation methodologies (Discrete Choice Experiment, Swing Weighting, threshold technique). Next, the presenters will illustrate how PPS attributes of importance to patients (such as life expectancy and quality of life-related attributes) can be implemented in medical product decision-making and regulatory evaluation. Finally, the session will discuss how patients and patient organizations should be engaged as active research partners throughout the PPS development and dissemination.