Objectives

There is increasing interest in expanding the elements of value to be considered when making health policy decisions. To help inform value frameworks, this study quantified preferences for disease attributes in a general public sample and examined which combination of attributes (disease profiles) are considered most important for research and treatment.

Methods

A discrete choice experiment (DCE) was conducted in a US general population sample, recruited through online consumer panels. Respondents were asked to select one of a set of health conditions they believed to be most important, characterized by attributes defined by a previous qualitative study: onset age; cause of disease; life expectancy; caregiver requirement; symptom burden (characterized by the Health Utilities Index with varying levels of ambulation independence, dexterity limitations, and degree of pain and discomfort); and disease prevalence. A fractional factorial DCE design was implemented using R, and 60 choice sets were generated (separated into blocks of 10 per participant). Data were analyzed using a mixed-logit regression model, and results used to assess the likelihood of preferring disease profiles. Based on individual attribute preferences, overall preferences for disease profiles, including a profile aligned with Duchenne muscular dystrophy (DMD), were compared.

Results

Fifty-two percent of respondents (n = 537) were female, and 70.6% were aged 18–54 years. Attributes considered most important were those related to life expectancy (odds ratio [OR], 95% confidence interval [CI] 1.88 [1.56–2.27] for a 50% reduction in remaining life expectancy vs no impact), and symptom burden (OR [95% CI] 1.84 [1.47–2.31] for severe vs mild burden). Greater importance was also found for pediatric onset, caregiver requirement, and diseases affecting more people. As an example of disease profile preferences, a DMD-like pediatric inherited disease with 50% reduction in life expectancy, extensive caregiver requirement, severe symptom burden, and 1:5000 prevalence had 2.37-fold higher odds of being selected as important versus an equivalent disease with adult onset and no life expectancy reduction.

Conclusions

Of disease attributes included in this DCE, respondents valued higher prevalence of disease, life expectancy and symptom burden as most important for prioritizing research and treatment. Based on expressed attribute preferences, a case study of an inherited pediatric disease involving substantial reductions to length and quality of life and requiring caregiver support has relatively high odds of being identified as important compared to diseases reflecting differing attribute profiles. These findings can help inform expansions of value frameworks by identifying important attributes from the societal perspective.

中文翻译：

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比较疾病概况的偏好：美国社会视角的离散选择实验

目标

人们越来越有兴趣扩大制定卫生政策决策时要考虑的价值要素。为了帮助了解价值框架，本研究量化了公众样本中对疾病属性的偏好，并检查了哪些属性组合（疾病概况）被认为对研究和治疗最重要。

方法

一项离散选择实验（DCE）是在通过在线消费者小组招募的美国普通人群样本中进行的。受访者被要求选择他们认为最重要的一组健康状况中的一个，其特征是先前定性研究定义的属性：发病年龄；疾病原因；预期寿命;照顾者要求；症状负担（以健康公用事业指数为特征，具有不同程度的步行独立性、灵活性限制以及疼痛和不适程度）；和疾病流行情况。使用 R 实现了部分阶乘 DCE 设计，并生成了 60 个选择集（每个参与者分为 10 个选项集）。使用混合对数回归模型分析数据，并将结果用于评估偏好疾病特征的可能性。根据个人属性偏好，对疾病概况的总体偏好进行比较，包括与杜氏肌营养不良症 (DMD) 一致的概况。

结果

52% 的受访者 ( n = 537) 是女性，70.6% 的年龄在 18-54 岁之间。最重要的属性是与预期寿命相关的属性（比值比 [OR]，95% 置信区间 [CI] 1.88 [1.56–2.27]，剩余预期寿命减少 50% 与无影响）和症状负担（OR [ 95% CI] 1.84 [1.47–2.31]（严重与轻度负担）。还发现儿科发病、护理人员需求和影响更多人的疾病也更重要。作为疾病概况偏好的一个例子，一种预期寿命缩短 50%、需要大量护理人员、症状负担严重且患病率为 1:5000 的 DMD 类儿科遗传性疾病，其被选择为重要疾病的几率比同等疾病高 2.37 倍成人发病且预期寿命不减少的疾病。

结论

在本 DCE 中包含的疾病属性中，受访者认为较高的疾病患病率、预期寿命和症状负担对于优先研究和治疗最为重要。根据表达的属性偏好，与反映不同属性特征的疾病相比，涉及生命长度和质量大幅缩短并需要护理人员支持的遗传性儿科疾病的案例研究被确定为重要的可能性相对较高。这些发现可以通过从社会角度识别重要属性来帮助扩展价值框架。