Patient-Centered Research, Evidera, Bethesda, MD, USA.
formerly with Value Based Medicine, Biogen, Cambridge, MA.
Med Decis Making. 2020 Oct;40(7):897-911. doi: 10.1177/0272989X20951782. Epub 2020 Oct 5.
The Neuro-QoL is a standardized approach to assessing health-related quality of life in people with neurological conditions, including multiple sclerosis (MS). Item banks were developed with item response theory (IRT) methodology so items are calibrated along a continuum of each construct. The purpose of this study was to develop a preference-based scoring algorithm for the Neuro-QoL to derive utilities that could be used in economic modeling.
With input from neurologists, 6 Neuro-QoL domains were selected based on relevance to MS and used to define health states for a utility elicitation study in the United Kingdom. General population participants and individuals with MS valued the health states and completed questionnaires (including Neuro-QoL short forms). The Neuro-QoL Utility Scoring System (NQU) was derived based on multi-attribute utility theory using data from the general population sample. Single-attribute disutility functions for 6 Neuro-QoL domains were estimated using isotonic regression with linear interpolation and then combined with a multiplicative model. NQU validity was assessed using MS participant data.
Interviews were completed with 203 general population participants (50.2% female; mean age = 45.0 years) and 62 participants with MS (62.9% female; mean age = 46.1 years). Mean (SD) NQU scores were 0.94 (0.06) and 0.82 (0.13) for the general population and MS samples, respectively. The NQU demonstrated known-groups validity by differentiating among subgroups categorized based on level of disability. The NQU demonstrated convergent validity via correlations with generic measures (0.66 and 0.63 with EQ-5D-5L and Health Utilities Index Mark 3, respectively; both < 0.001).
With the NQU, utilities can be derived from any MS treatment group, subgroup, or patient sample who completes items from 6 Neuro-QoL domains. Because the Neuro-QoL is frequently used with MS patients, the NQU greatly expands the options for quantifying outcomes in cost-utility analyses conducted to inform allocation of resources for MS treatment.
Neuro-QoL 是一种评估神经科疾病患者(包括多发性硬化症患者)健康相关生活质量的标准化方法。通过项目反应理论(IRT)方法开发了项目库,以便根据每个构建的连续体对项目进行校准。本研究的目的是开发一种基于偏好的 Neuro-QoL 评分算法,以得出可用于经济建模的效用值。
根据神经病学家的意见,选择了 6 个与多发性硬化症相关的 Neuro-QoL 领域,用于定义英国效用评估研究中的健康状态。一般人群参与者和多发性硬化症患者对健康状态进行了评估,并完成了问卷调查(包括 Neuro-QoL 简短形式)。Neuro-QoL 效用评分系统(NQU)是基于多属性效用理论,使用一般人群样本中的数据得出的。使用具有线性插值的等渗回归估计 6 个 Neuro-QoL 领域的单一属性负效用函数,然后将其与乘法模型相结合。使用多发性硬化症患者数据评估 NQU 的有效性。
共完成了 203 名一般人群参与者(50.2%为女性;平均年龄=45.0 岁)和 62 名多发性硬化症患者(62.9%为女性;平均年龄=46.1 岁)的访谈。一般人群和多发性硬化症患者样本的平均(SD)NQU 评分为 0.94(0.06)和 0.82(0.13)。NQU 通过基于残疾程度的分组区分不同亚组,显示出明确的组间有效性。NQU 通过与通用指标的相关性显示出收敛有效性(与 EQ-5D-5L 和 Health Utilities Index Mark 3 的相关性分别为 0.66 和 0.63;均<0.001)。
使用 NQU,可以从完成 6 个 Neuro-QoL 领域项目的任何多发性硬化症治疗组、亚组或患者样本中得出效用值。由于 Neuro-QoL 常用于多发性硬化症患者,因此 NQU 极大地扩展了在资源分配多发性硬化症治疗成本效益分析中量化结果的选择。