Centre for Health Systems and Policy Research, JC School of Public Health and Primary Care, Faculty of Medicine, The Chinese University of Hong Kong, Prince of Wales Hospital, Sha Tin, N.T., Hong Kong, China.
Appl Health Econ Health Policy. 2024 Nov;22(6):849-860. doi: 10.1007/s40258-024-00912-1. Epub 2024 Sep 10.
Patients may get more treatment options with off-label use of drugs while exposed to unknown risks of adverse events. Little is known about the public or demand-side perspective on off-label drug use, which is important to understand how to use off-label treatment and devise financial assistance. This study aimed to quantify public preference for off-label cancer treatment outcomes, process, and costs, and perceived importance of associated adverse events.
A discrete choice experiment and a best-worst scaling were conducted in Hong Kong in December 2022. Quota sampling was used to randomly select the study sample from a territory-wide panel of working-age adults. Preferences and willingness to pay (WTP) for treatment effectiveness, risk of adverse events, mode of drug administration, and availability of off-label treatment guidelines were estimated using a random parameter logit model and latent class model. The relative importance of different adverse events was elicited using Case 1 best-worst scaling.
A total of 435 respondents provided valid responses. In the discrete choice experiment, the respondents indicated that extra overall survival as treatment effectiveness (WTP: HK$448,000/US$57,400 for 12-month vs 3-month extra survival) was the most important attribute for off-label drugs, followed by the risk of adverse events (WTP: HK$318,000/US$40,800 for 10% chance to have adverse event vs 55%), mode of drug administration (WTP: HK$42,000/US$5300 for oral intake vs injection), and availability of guidelines (WTP: HK$31,000/US$4000 for available versus not available). Four groups with distinct preferences were identified, including effectiveness oriented, off-label use refusal, oral intake oriented, and adverse event risk aversion. In the best-worse scaling, hypothyroidism, nausea/vomiting, and arthralgia/joint pain were the three most important adverse events based on the perceptions of respondents. Risk-averse respondents, who were identified from the discrete choice experiment, had different perceived importance of the adverse events compared with those with other preferences.
Knowing the preference and WTP for cancer treatment-related characteristics from a societal perspective facilitates doctors' communications with patients on decision making and treatment goal-setting for off-label treatment, and enables devising financial assistance for related treatments. This study also provides important insight to inform evaluations of public acceptance and information dissemination in drug development as well as future economic evaluations.
药物的标签外使用可为患者提供更多的治疗选择，但也会带来未知的不良事件风险。关于药物标签外使用的公众或需求方观点鲜为人知，这对于了解如何使用标签外治疗和制定财务援助措施非常重要。本研究旨在量化公众对癌症标签外治疗结果、过程和成本的偏好，以及对相关不良事件的重视程度。
2022 年 12 月，在香港进行了一项离散选择实验和最佳最差分级法。通过配额抽样，从全港范围内的成年劳动力小组中随机选择研究样本。使用随机参数逻辑模型和潜在类别模型，估计治疗效果、不良事件风险、药物管理方式和标签外治疗指南可用性的偏好和支付意愿（WTP）。使用案例 1 最佳最差分级法得出不同不良事件的相对重要性。
共有 435 名受访者提供了有效回复。在离散选择实验中，受访者表示，额外的总生存期作为治疗效果（WTP：448,000 港元/57,400 美元，额外 12 个月 vs 3 个月生存期）是标签外药物最重要的属性，其次是不良事件风险（WTP：318,000 港元/40,800 美元，出现不良事件的概率为 10% vs 55%）、药物管理方式（WTP：42,000 港元/5300 美元，口服 vs 注射）和指南可用性（WTP：31,000 港元/4000 美元，有 vs 无）。确定了四个具有不同偏好的组，包括以效果为导向、拒绝标签外使用、以口服为导向和对不良事件风险的厌恶。在最佳最差分级法中，基于受访者的认知，甲状腺功能减退症、恶心/呕吐和关节痛/关节痛是三个最重要的不良事件。从离散选择实验中识别出的风险厌恶型受访者对不良事件的感知重要性与其他偏好的受访者不同。
从社会角度了解癌症治疗相关特征的偏好和 WTP，有助于医生与患者就标签外治疗的决策和治疗目标进行沟通，并为相关治疗制定财务援助措施。本研究还为药物开发中公众接受度和信息传播的评估以及未来的经济评估提供了重要的见解。
