The Research Center of National Drug Policy & Ecosystem, China Pharmaceutical University, No.639 Longmian Avenue, Jiangning District, Nanjing, China.
The 923rd Hospital of the Joint Logistics Support Force of the People's Liberation Army, Nanning, China.
BMC Health Serv Res. 2024 Oct 10;24(1):1215. doi: 10.1186/s12913-024-11547-2.
Transfusion-dependent β-thalassemia (TDT) is one of the global public health concerns highlighted by the World Health Organization. Patients with TDT require regular blood transfusion to survive. However, the availability of blood resources is extremely limited. The purpose of this study was to investigate transfusion burden and willingness to pay (WTP) for temporary remission of anemia status among patients with TDT and to explore the associated factors.
Adult patients with TDT were recruited through cluster sampling across several high-incidence provinces in China. Consenting patients completed online questionnaires on demographic information, transfusion burden and WTP with real-time WeChat communication assistance from researchers. The guiding techniques of double-bounded dichotomous choices and open-ended questions in the contingent valuation method (CVM) were used to obtain participants' WTP for 1 unit of leukocyte-depleted red blood cells. WTP calculations were performed using maximum likelihood estimation, with further insights gained through subgroup analysis based on gender, family monthly income level and convenience of blood transfusion.
The analysis included 149 TDT patients from five high-incidence provinces, with an average monthly income of $198.5. Patients received an average of 3.7 units per transfusion, 15.4 times annually, with an average WTP of $70.4 per unit (95% CI [62.0, 78.9]). Estimated WTP for temporary anemia alleviation per transfusion totaled $260.6, exceeding monthly income by 1.32 times. Higher WTP was observed among males, higher-income households, and those with at least junior education. Lower WTP was noted among patients with lower transfusion volumes and those needing to travel for transfusion or during hospitalization for blood transfusion.
High WTP indicated a strong desire for temporary anemia relief. Most TDT patients faced significant economic and transfusion burden. The evident gap in meeting clinical needed underscores the urgent demand for innovative treatments to reduce transfusion dependency, potentially transforming TDT care and improving socioeconomic well-being and clinical outcomes. These findings supported evidence-based decision-making for TDT pharmacoeconomics and efficient healthcare resource allocation in China.
输血依赖型β-地中海贫血症(TDT)是世界卫生组织(WHO)重点关注的全球公共卫生问题之一。TDT 患者需要定期输血才能存活。然而,血液资源的供应极其有限。本研究旨在调查 TDT 患者的输血负担和缓解贫血状态的支付意愿(WTP),并探讨相关因素。
采用整群抽样方法,在我国多个高发省份招募成年 TDT 患者。知情同意的患者在研究者实时微信通讯协助下完成在线问卷调查,内容包括人口统计学信息、输血负担和 WTP。采用双边界二分选择和条件价值评估方法(CVM)中的开放式问题指导技术,获得参与者对 1 单位去白细胞红细胞的 WTP。使用最大似然估计法进行 WTP 计算,并进一步根据性别、家庭月收入水平和输血便利性进行亚组分析。
本研究共纳入来自五个高发省份的 149 名 TDT 患者,平均月收入为 198.5 美元。患者每次输血平均接受 3.7 个单位,每年 15.4 次,平均 WTP 为每个单位 70.4 美元(95%CI [62.0,78.9])。每次输血的临时贫血缓解 WTP 估计为 260.6 美元,是月收入的 1.32 倍。男性、高收入家庭和至少受过初中教育的患者 WTP 较高。输血量较低和需要异地输血或住院输血的患者 WTP 较低。
较高的 WTP 表明患者对临时贫血缓解有强烈的愿望。大多数 TDT 患者面临着沉重的经济和输血负担。临床需求与实际情况之间的明显差距突显了对创新治疗方法的迫切需求,以减少对输血的依赖,可能会改变 TDT 的治疗方式,提高患者的社会经济福利和临床结局。这些发现为 TDT 药物经济学和中国医疗资源的有效配置提供了基于证据的决策依据。