Value of Innovative Multiple Myeloma Treatments from Patient and Healthcare Provider Perspectives: Evidence from a Discrete Choice Experiment

从患者和医疗服务提供者的角度评估创新型多发性骨髓瘤疗法的价值:一项离散选择实验的证据

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Abstract

BACKGROUND: Although innovation generally provides measurable improvements in disease characteristics and patient survival, some benefits can remain unclear. This study aimed to investigate patient and healthcare provider (HCP) preferences for the innovative attributes of multiple myeloma (MM) treatments. METHODS: A cross-sectional, web-based, discrete choice experiment (DCE) survey was conducted among 200 patients with MM and 30 HCPs of patients with MM in the USA. A literature review, followed by interviews with patients with MM and HCPs, was undertaken to select five attributes (progression-free survival [PFS], chance of severe side effects, how patients live with MM treatments, scientific innovation, and monthly out-of-pocket [OOP] cost) and their levels. A Bayesian efficient design was used to generate DCE choice sets. Each choice set comprised two hypothetical MM treatment alternatives described by the selected attributes and their levels. Each patient and HCP was asked to choose a preferred alternative from each of the 11 choice sets. Mixed logit and latent class models were developed to estimate patient and HCP preferences for the treatment attributes. RESULTS: Overall, patients and HCPs preferred increased PFS, less chance of severe side effects, a treatment that offered life without treatment, scientific innovation, and lower OOP cost. From patients' perspectives, PFS had the highest conditional relative importance (44.7%), followed by how patients live with MM treatments (21.6%) and scientific innovation (16.0%). CONCLUSIONS: In addition to PFS, patients and HCPs also valued innovative MM treatments that allowed them to live without treatments and/or offered scientific innovation. These attributes should be considered when evaluating MM treatments.

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