Department of Pharmacy, Huangpu Branch, Shanghai Ninth People's Hospital, School of Medicine, Shanghai Jiao Tong University, Shanghai, China.
Health Services Management Department, Guizhou Medical University, Gui'an, China.
Eur J Health Econ. 2024 Aug;25(6):915-924. doi: 10.1007/s10198-023-01635-1. Epub 2023 Oct 14.
To assess the feasibility of estimating an EQ-5D-5L value set using a small study design in cancer patients and to compare the EQ-5D-5L values based on the preferences of cancer patients with those of the general public.
Patients with clinically diagnosed cancers were recruited from two hospitals in Shanghai, China. In face-to-face interviews using the EQ-PVT survey, health states were valued by cancer patients using both cTTO and DCE methods. cTTO data was modelled alone or jointly with DCE data. Forty-eight models using different model specifications (cross-attribute level effect [CALE] and additive models), random/fixed effects model assumptions, data heteroscedasticity and censoring were estimated. The best performed model was identified in terms of monotonicity of estimated model coefficients and out-of-sample prediction accuracy.
Data collected from 221 cancer patients who participated in the study were included. The hybrid CALE model using both TTO and DCE data performed best in terms of prediction accuracy (Lin's concordance coefficient = 0.989; root mean squared error = 0.058) and suggested that pain/discomfort and anxiety/depression were the most undesirable health problems. Compared to values based on general Chinese public's health preferences, the values based on cancer patients' preferences were much higher and lower for health states characterized by extreme mobility problems and severe/extreme pain or discomfort, respectively.
This study demonstrated the feasibility of using a small design to develop EQ-5D-5L value sets based on cancer patients' health preferences. Since there were signs of differences between preferences of patients and general population, it may be valuable to develop patient-specific value sets and use them in clinical decision making and economic evaluations.
评估在癌症患者中使用小型研究设计估算 EQ-5D-5L 值集的可行性，并比较癌症患者基于偏好的 EQ-5D-5L 值与一般公众的偏好。
从中国上海的两家医院招募患有临床诊断癌症的患者。在使用 EQ-PVT 调查的面对面访谈中，癌症患者使用 TTO 和 DCE 方法对健康状况进行估值。分别对 TTO 数据和 TTO 与 DCE 数据联合建模。使用不同模型规范（交叉属性水平效应 [CALE] 和加性模型）、随机/固定效应模型假设、数据异方差和截尾对 48 个模型进行了估计。根据估计模型系数的单调性和样本外预测准确性来确定最佳模型。
纳入了 221 名参与研究的癌症患者的数据。基于 TTO 和 DCE 数据的混合 CALE 模型在预测准确性方面表现最佳（Lin 一致性系数=0.989；均方根误差=0.058），并表明疼痛/不适和焦虑/抑郁是最不理想的健康问题。与基于中国一般公众健康偏好的价值相比，基于癌症患者偏好的价值在健康状况具有极端移动问题和严重/极度疼痛或不适时分别更高和更低。
本研究证明了使用小型设计基于癌症患者的健康偏好制定 EQ-5D-5L 值集的可行性。由于患者和一般人群之间存在偏好差异的迹象，开发特定于患者的价值集并在临床决策和经济评估中使用它们可能具有价值。
