ABSTRACT

Introduction: Despite the increasing role of patients in the US healthcare system, patients have yet been engaged in the value assessment of their treatments, including disease-modifying therapies (DMTs) for multiple sclerosis (MS). The objectives of this review were therefore to summarize existing studies on cost-effectiveness analysis (CEA) with quality-adjusted life years (QALYs) and patients’ preferences of DMTs for MS, and to discuss how to incorporate patients’ preferences into the value assessment of DMTs.

Area covered: We reviewed previous systematic reviews and conducted further search until November 2020 for studies on CEA with QALYs and patients’ preferences of DMTs for MS. We identified the outcomes that were assessed or valued in the CEA studies and the DMT attributes that were important to patients with MS.

Expert opinion: Our literature review showed that the studies using CEA with QALYs failed to capture some important DMT attributes, e.g., route and frequency of administration, identified in the studies on the patients’ preferences. Various approaches were available for incorporating the patients’ preferences in the value assessment of DMTs for MS. We supported this incorporation, which subsequently would increase patient access to preferred DMTs.

摘要

简介：尽管患者在美国医疗保健系统中的作用日益增强，但患者仍参与了其治疗方法的价值评估，包括多发性硬化症（MS）的疾病缓解疗法（DMT）。因此，本次审查的目的是总结有关成本效益分析（CEA）和质量调整生命年（QALYs）以及DMT患者对MS的偏爱的现有研究，并讨论如何将患者的偏爱纳入价值评估中DMT。

覆盖区域：我们回顾了之前的系统评价，并进行了进一步搜索，直到2020年11月，以进行具有QALYs的CEA和患者对MS的DMTs偏好的研究。我们确定了在CEA研究中评估或评估的结果以及对MS患者重要的DMT属性。

专家意见：我们的文献综述表明，将CEA与QALYs结合使用的研究未能掌握一些重要的DMT属性，例如，在研究患者偏爱的研究中确定的给药途径和频率。有多种方法可将患者的喜好纳入MS的DMT的价值评估中。我们支持该合并，随后将增加患者使用首选DMT的机会。