Hirniak Sam, Edginton Andrea N, Iorio Alfonso, Wong William W L
School of Pharmacy, Faculty of Science, University of Waterloo, 10A Victoria St S, Kitchener, ON, N2G 1C5, Canada.
Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, ON, Canada.
Pharmacoeconomics. 2025 Jul;43(7):765-778. doi: 10.1007/s40273-025-01484-1. Epub 2025 Apr 1.
Hemophilia A is a costly, lifelong illness with multiple prophylaxis options. Previously, these options were assessed using a Peterson score-based model to simulate joint damage over time. This study built a model for the economic evaluation of hemophilia A with less socioeconomic selection bias utilizing the hemophilia joint health score (HJHS).
A mechanistically defined HJHS-based state-transition microsimulation model was implemented for the cost-utility analysis conducted over a lifetime horizon from a Canadian provincial Ministry of Health perspective, with a 1.5% discount rate on (costs and outcomes), to compare the following interventions: standard half-life (SHL), extended half-life (EHL), emicizumab, and efanesocotog alfa (EA). The health states are HJHS levels, waiting for surgery, postoperative time, and death. Individuals experience bleeds, joint bleeds (increasing the HJHS), and surgery in each health state. Disutilities include injections and postoperative time. Model validation included face validity, internal validity, comparison analysis, external validity, and predictive validity. Probabilistic analysis, pricing threshold analysis, and one-way scenario analyses were completed.
EA showed lower levels of hospitalizations and surgeries and an improved joint damage experience in the simulation. However, EA was not cost-effective against emicizumab, which continued to be the most cost-effective intervention. Pricing threshold analysis indicated that a price decrease would be required for EA to dominate SHL (50% decrement) and emicizumab (55% decrement).
This is the first cost-effectiveness model incorporating HJHS to apply sequential joint damage to hemophilia A. While EA offers clinical benefits, our analysis suggests it will not be cost-effective from a Canadian provincial Ministry of Health perspective without a significant price decrease.
甲型血友病是一种需要长期治疗且花费高昂的疾病，有多种预防方案。此前，这些方案是使用基于彼得森评分的模型来模拟随时间推移的关节损伤情况。本研究利用血友病关节健康评分（HJHS）建立了一个经济评估模型，以减少甲型血友病经济评估中的社会经济选择偏差。
从加拿大省级卫生部的角度出发，采用基于HJHS的机制定义的状态转换微观模拟模型，对一生的成本效用进行分析，成本和结果的贴现率为1.5%，以比较以下干预措施：标准半衰期（SHL）、延长半衰期（EHL）、emicizumab和efanesocotog alfa（EA）。健康状态包括HJHS水平、等待手术、术后时间和死亡。个体在每个健康状态下会发生出血、关节出血（增加HJHS）和手术。负效用包括注射和术后时间。模型验证包括表面效度、内部效度、比较分析、外部效度和预测效度。完成了概率分析、定价阈值分析和单向情景分析。
在模拟中，EA显示出较低的住院和手术水平，以及改善的关节损伤情况。然而，与emicizumab相比，EA不具有成本效益，emicizumab仍然是最具成本效益的干预措施。定价阈值分析表明，EA要优于SHL（降价50%）和emicizumab（降价55%），需要降低价格。
这是第一个纳入HJHS以对甲型血友病应用序贯关节损伤的成本效益模型。虽然EA具有临床益处，但我们的分析表明，从加拿大省级卫生部的角度来看，在没有大幅降价的情况下，它不具有成本效益。
