OBJECTIVES: Patient preferences have become an important focus for inclusion in drug development and evaluation. Understanding patient preferences is especially valuable in Multiple Myeloma (MM), where the rapid development of treatments with diverging benefit-risk profiles raises uncertainty about what matters most to patients. This study aimed to investigate which treatment attributes (side-effects, symptoms, efficacy outcomes, uncertainties) are most important to MM patients.
METHODS: A preference survey incorporating Discrete Choice Experiment (DCE) and Swing Weighting (SW) was widely disseminated through the European MM patient population. The survey was developed using a qualitative study during which MM patients (n=24) reached consensus on the attributes and levels included in the survey. MM patients and patient organizations provided extensive feedback during the survey development and piloting.
RESULTS: 393 MM patients across 21 countries participated. Patients were heterogeneous regarding years since diagnosis (M: 6) and prior therapies (M: 3). While life expectancy was most important to patients with the most and least prior therapies, quality of life-related attributes such as pain and mobility problems were most important to participants with intermediary treatment experience. Significant preference heterogeneity was revealed depending on participants’ side-effect and symptom experience. Participants highlighted the difficulty of trading-off between life expectancy and quality of life, and between physical and mental health. Patients demanded psychological support to cope with their symptoms, side-effects and uncertainties.
CONCLUSIONS: Preferences elicited from patients reveal the need for the systematic inclusion and prioritization of quality of life-related treatment outcomes by drug developers, regulators, Health Technology Assessment bodies, and healthcare providers in MM drug development, evidence generation, evaluation, and clinical practice. In order for patients to make informed choices in preference studies, researchers should involve patients and patient organizations during the selection of the attributes, levels, and explanations, how the preference questions are asked, the visuals and survey technology.
