Janssen Research & Development, Titusville, NJ, USA.
Rheumatology Research Group, Institute of Inflammation and Ageing, University of Birmingham, Birmingham, UK.
Med Decis Making. 2024 Feb;44(2):189-202. doi: 10.1177/0272989X231218265. Epub 2024 Jan 19.
When selecting samples for patient preference studies, it may be difficult or impractical to recruit participants who are eligible for a particular treatment decision. However, a general public sample may not be an appropriate proxy.
This study compares preferences for rheumatoid arthritis (RA) preventive treatments between members of the general public and first-degree relatives (FDRs) of confirmed RA patients to assess whether a sample of the general public can be used as a proxy for FDRs.
Participants were asked to imagine they were experiencing arthralgia and had screening tests indicating a 60% chance of developing RA within 2 yrs. Using a discrete choice experiment, participants were offered a series of choices between no treatment and 2 unlabeled hypothetical treatments to reduce the risk of RA. To assess data quality, time to complete survey sections and comprehension questions were assessed. A random parameter logit model was used to obtain attribute-level estimates, which were used to calculate relative importance, maximum acceptable risk (MAR), and market shares of hypothetical preventive treatments.
The FDR sample ( = 298) spent more time completing the survey and performed better on comprehension questions compared with the general public sample ( = 982). The relative importance ranking was similar between the general public and FDR participant samples; however, other relative preference measures involving weights including MARs and market share differed between groups, with FDRs having numerically higher MARs.
In the context of RA prevention, the general public (average risk) may be a reasonable proxy for a more at-risk sample (FDRs) for overall relative importance ranking but not weights. The rationale for a proxy sample should be clearly justified.
Participants from the general public were compared to first-degree relatives on their preferences for rheumatoid arthritis (RA) preventive treatments using a discrete choice experiment.Preferences were similar between groups in terms of the most important and least important attributes of preventive treatments, with effectiveness being the most important attribute. However, relative weights differed.Attention to the survey and predicted market shares of hypothetical RA preventive treatments differed between the general public and first-degree relatives.The general public may be a reasonable proxy for an at-risk group for patient preferences ranks but not weights in the disease prevention context; however, care should be taken in sample selection for patient preference studies when choosing nonpatients.
在选择参与患者偏好研究的样本时，招募符合特定治疗决策条件的参与者可能较为困难或不切实际。然而，一般公众样本可能并不合适。
本研究通过比较一般公众和确诊类风湿关节炎（RA）患者一级亲属（FDR）对 RA 预防治疗的偏好，评估一般公众样本是否可作为 FDR 的替代样本。
参与者被要求想象自己正在经历关节痛，并接受了筛查测试，结果显示其在 2 年内发生 RA 的可能性为 60%。使用离散选择实验，参与者可在不治疗和两种未标记的假设治疗方案之间进行一系列选择，以降低患 RA 的风险。为了评估数据质量，评估了完成调查部分和理解问题的时间。使用随机参数对数模型获得属性水平估计值，用于计算假设预防治疗的相对重要性、最大可接受风险（MAR）和市场份额。
与一般公众样本（982 人）相比，FDR 样本（298 人）完成调查的时间更长，对理解问题的回答更好。一般公众和 FDR 参与者样本的相对重要性排序相似；然而，MAR 和市场份额等涉及权重的其他相对偏好衡量标准在组间存在差异，FDR 的 MAR 数值更高。
在 RA 预防的背景下，一般公众（平均风险）可能是更具风险的样本（FDR）的合理替代样本，用于总体相对重要性排序，但不适用于权重。对于代理样本的理由应明确说明。
使用离散选择实验，比较了一般公众与一级亲属对 RA 预防治疗的偏好。
一般公众和 FDR 组在预防治疗的最重要和最不重要属性方面的偏好相似，有效性是最重要的属性。然而，相对权重存在差异。
一般公众和 FDR 对调查的关注和预测的假设 RA 预防治疗的市场份额不同。
在疾病预防背景下，一般公众可能是患者偏好排名的风险群体的合理替代样本，但在选择非患者进行患者偏好研究时，应谨慎选择样本。
