Patient Preferences in Drug Development and Evaluation: Issues and Recommendations

Stakeholders involved in drug development and evaluation (patients, patient organizations, regulators, Health Technology Assessment (HTA) bodies, payers, drug developers, academic researchers, clinicians) are presently calling for evidence-based insights into ways for assessing and including patients’ preferences in a meaningful and systematic manner. This thesis offers recommendations and insights into best practices for patient preference study design, conduct, analysis and use in drug development and evaluation. Methodological learnings and proposed ways for systematically integrating preference studies in drug development aim to foster targeted multi-stakeholder discussions, internationally accepted recommendations and guidelines (such as those by IMI-PREFER and the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) respectively), joint horizon scanning activities, and (parallel) scientific advice on preference study design and use. In doing so, the preference study methodologies outlined in this thesis aim to complement existing methods that capture patients’ preferences, views, and experiences in drug decision-making such as Patient Reported Outcomes (PROs) and direct patient engagement. A wide-spread cross-stakeholder adoption of robustly designed and patient-centered preference studies will amplify patients’ unique voices in the medicinal product life cycle and intensify patients’ impact in decisions that ultimately affect them.