ISSUE: Patient preference studies (PPS) yield evidence about patients' desirability or acceptability of a particular alternative or choice among all outcomes of a particular drug. Such evidence is considered highly valuable to inform decisions along the medical product life cycle such as in unmet needs identification, clinical trial endpoint selection, regulatory benefit-risk assessment, Health Technology Assessment and reimbursement, and clinical decision-making. While the added value is clear, there is lack of case examples and understanding on how the assessment of PPS should occur to robustly inform decision-making. Methodological questions exist regarding appropriate PPS design and conduct, including regarding the development of the research question (product agnostic or not), method selection, patient recruitment, sample size, attribute and level development and qualitative research in this effort, PPS analysis, availability and transparency of PPS data and methods, and stakeholder engagement (incl. patient, clinician, regulatory and HTA bodies).

OVERVIEW: Drawing upon experiences from PPS’ in diverse disease domains, speakers from different stakeholder organisations (University Medical Center Groningen, European Medicines Agency, KU Leuven, EFCCA) will share their views (regulatory, methodological, patient) on how PPS can be robustly designed to provide evidence-based information considered useful by decision-makers. The session will cover both qualitative (interviews, focus group discussions) and quantitative methods (Discrete Choice Experiment, Swing Weighting, threshold technique). Presenters will illustrate how they approached their PPS design such as the development of attributes and levels (such as life expectancy and quality of life-related attributes), the choice of methods and selection of patients and sample size. The session will discuss how patients and patient organizations should be engaged as active research partners in informing key decisions throughout the PPS development and dissemination. This session is intended to augment best-practice insights and potential improvements regarding the design and conduct of PPS aiming to inform clinical trial design, regulatory and HTA bodies.