Treatment preferences of patients with relapsed or refractory multiple myeloma in the United States, United Kingdom, Italy, Germany, France, and Spain: results from a discrete choice experiment.

Introduction: Newer treatment options for relapsed/refractory multiple myeloma (RRMM) with efficacy and safety profiles that differ from traditional therapies have facilitated personalized management strategies to optimize patient outcomes. In the context of such personalized management, understanding how treatment characteristics influence patients' preferences is essential. This study assessed patients' preferences for RRMM treatment attributes and determined trade-offs between potential benefits, administration procedures, and adverse effects.
Methods: Patients' preferences were evaluated using a discrete choice experiment (DCE). Patients with RRMM who reported failing two lines of anti-myeloma treatment (immunomodulatory agent and a proteasome inhibitor [PI]) or ≥ 3 lines (including ≥1 PI, immunomodulatory agent, or anti-CD38 monoclonal antibody), were recruited across the US, UK, Italy, Germany, France, and Spain. DCE attributes and levels were identified using a targeted literature review, a review of clinical data for relevant RRMM treatments, qualitative patient interviews, and input from clinical and myeloma patient experts. The DCE was administered within an online survey from February-June 2022. Preference data were analyzed using an error-component logit model and willingness to make trade-offs for potential benefits, and relative attribute importance scores were calculated.
Results: Overall, 296 patients from the US ( n = 100), UK ( n = 49), Italy ( n = 45), Germany ( n = 43), France ( n = 39), and Spain ( n = 20) participated in the DCE. Mean (standard deviation) age was 63.8 (8.0) years, 84% had a caregiver, and patients had a median of 3 (range: 2-8) prior lines of therapy. Efficacy attributes most influenced patients' preferences, with increasing overall response rate (25-85%) and overall survival (6 months to 2 years) contributing to ~50% of treatment decision-making. Administration procedures were also considered important to patients. Avoiding individual side effects was considered relatively less important, with patients willing to tolerate increases in side effects for gains in efficacy. Patient characteristics such as rate of disease progression, sociodemographics, or clinical characteristics also influenced treatment preferences.
Conclusion: Patients with RRMM were willing to tolerate increased risk of side effects for higher efficacy. Preferences and risk tolerance varied between patients, with preference patterns differing by certain patient characteristics. This highlights the importance of shared decision-making for optimal treatment selection and patient outcomes.
Competing Interests: CT, MR, SM, NK, and HG are salaried employees of Evidera who received funding from GSK to conduct this study. MR and HG hold shares in Evidera. SA reports consultancy for Amgen, AstraZeneca, Beigene, Bristol Myers Squibb, Janssen, GSK, Pharmacyclics, Regeneron, Sanofi, and Takeda and has research funding from, AbbVie, Amgen, Ascentage Pharma, Bristol Myers Squibb, Cellectar, Janssen, GSK, Medimmune, Pharmacyclics, and Xencor. RP reports personal fees and non-financial support from Janssen, Celgene, and GSK and personal fees from AbbVie. DK reports consultancy for GSK and Triphase Accelerator Corporation and has management, advisory roles, and an ownership interest in Calm Water Therapeutics LLC. BG, MH, and SP are employees of and hold stocks and shares in GSK. AO’N and AM are employees of GSK. PP was an employee of GSK at the time this analysis was completed and currently hold stocks and shares in GSK.
(Copyright © 2023 Thomas, Ailawadhi, Popat, Kleinman, Ross, Gorsh, Mulnick, O’Neill, Paka, Hanna, Krucien, Molinari, Gelhorn and Perera.)