Department of Health Policy and Management, The Johns Hopkins Bloomberg School of Public Health, 615 N. Wolfe St, Baltimore, MD, 21205, USA.
Department of Health, Behavior, and Society, The Johns Hopkins Bloomberg School of Public Health, 615 N. Wolfe St, Baltimore, MD, 21205, USA.
Patient. 2019 Apr;12(2):247-257. doi: 10.1007/s40271-018-0330-8.
Best-worst scaling methods have been used in several Duchenne and Becker muscular dystrophy (DBMD) studies to quantify patient and caregiver priorities and preferences and promote patient-focused drug development (PFDD). We sought to assess the extent to which different members of the DBMD community would accept a discrete-choice experiment (DCE) that incorporates uncertainty regarding individual-level benefit.
A community advisory board encouraged the development and testing of a DCE to further examine treatment preferences in DBMD and to facilitate the inclusion of a policy-relevant uncertainty attribute. The DCE assessed preferences across a primary outcome (muscle strength) and several risks (uncertainty regarding treatment benefit, kidney damage risk, and fracture risk). The single instrument was tested among adult patients, caregivers, and professionals at the national Parent Project Muscular Dystrophy annual meeting. The DCE was analyzed using conditional logit. Instrument acceptability was evaluated using a previously developed set of questions assessing ease of understanding and answering, and if answers reflected the respondents' real preferences. We proposed a 75% agreement rate as a threshold of acceptability, and used a Z score to assess if this was met, exceeded, or rejected.
A total of 161 people completed the survey including 9 patients, 87 caregivers, and 65 professionals. Patients reported high acceptability across all evaluation items (p values > 0.21). Caregivers and professionals exceeded the benchmark of acceptability on understanding and reflecting real preferences (p < 0.001). Professionals met the benchmark (p = 0.08) for ease of answering, but caregivers did not (p < 0.01). DCE results demonstrated that all groups made meaningful trade-offs, with patients being less tolerant of risks than either caregivers or professionals (p < 0.001), and with no significant difference between caregivers and professionals (p = 0.46).
This study demonstrates the acceptable application of a single instrument across a multi-stakeholder population that used a complex preference method and included a policy-relevant uncertainty variable. Ease of answering was lowest among caregivers, but a post-hoc analysis revealed that it was most difficult for those with children under the age of 10, while those with older children met the threshold. The success of this study has laid the foundation for a global study of DBMD preferences using this method.
最佳最差标度法已在几项杜氏肌营养不良症（Duchenne and Becker muscular dystrophy，DBMD）研究中使用，以量化患者和照护者的优先事项和偏好，并促进以患者为中心的药物开发（patient-focused drug development，PFDD）。我们旨在评估 DBMD 社区的不同成员接受包含个体获益不确定性的离散选择实验（discrete-choice experiment，DCE）的程度。
一个社区顾问委员会鼓励开发和测试 DCE，以进一步研究 DBMD 中的治疗偏好，并促进纳入一个与政策相关的不确定性属性。DCE 评估了主要结局（肌肉力量）和几个风险（治疗获益不确定性、肾脏损伤风险和骨折风险）的偏好。该单一工具在全国肌营养不良症父母项目年度会议上的成年患者、照护者和专业人员中进行了测试。使用条件逻辑回归分析 DCE。使用一组先前开发的问题评估工具的可接受性，这些问题评估了理解和回答的难易程度，以及答案是否反映了受访者的真实偏好。我们提出了 75%的一致性率作为可接受性的阈值，并使用 Z 分数来评估是否达到、超过或拒绝该阈值。
共有 161 人完成了调查，包括 9 名患者、87 名照护者和 65 名专业人员。患者对所有评估项目的可接受性均较高（p 值均>0.21）。照护者和专业人员在理解和反映真实偏好方面达到了可接受性的基准（p<0.001）。专业人员在回答的难易程度方面达到了基准（p=0.08），但照护者没有（p<0.01）。DCE 结果表明，所有组都进行了有意义的权衡，患者比照护者或专业人员更不能容忍风险（p<0.001），而照护者和专业人员之间没有显著差异（p=0.46）。
这项研究证明了一种单一工具在多利益相关者人群中的可接受应用，该工具使用了复杂的偏好方法并纳入了一个与政策相关的不确定性变量。照护者的回答难度最低，但事后分析显示，对于有 10 岁以下儿童的照护者来说，难度最大，而对于有较大儿童的照护者来说，则达到了这一标准。这项研究的成功为使用这种方法进行全球 DBMD 偏好研究奠定了基础。
