Kengkla Kirati, Wilairat Preyanate, Natesirinilkul Rungrote, Sosothikul Darintr, Phisalprapa Pochamana, Saokaew Surasak
Division of Clinical Pharmacy, Department of Pharmaceutical Care, School of Pharmaceutical Sciences, University of Phayao, Mueang Phayao, Phayao, Thailand.
Center of Health Outcomes Research and Therapeutic Safety (Cohorts), School of Pharmaceutical Sciences, University of Phayao, Mueang Phayao, Phayao, Thailand.
Haemophilia. 2024 Nov;30(6):1288-1297. doi: 10.1111/hae.15105. Epub 2024 Oct 5.
In Thailand, an upper-middle-income country, managing haemophilia A (HA) with inhibitors poses significant challenges, often necessitating bypassing agents (BPAs) for bleeding control. This study evaluates the cost-effectiveness and budget impact of emicizumab, a novel prophylactic agent, as an alternative to both episodic and prophylactic BPA treatments from a societal perspective.
A Markov model was employed to estimate the lifetime societal costs and outcomes of emicizumab prophylaxis for HA patients with inhibitors. Treatment efficacy, cost, and epidemiological data were obtained through a comprehensive literature review and incorporated into the model. A 5-year budget impact analysis complemented the cost-utility analysis, with a 3% annual discount rate applied to future costs and outcomes.
In the base-case scenario, emicizumab prophylaxis in HA patients aged 2 years and above demonstrated superior cost-effectiveness, yielding 18.1 quality-adjusted life years (QALYs) per patient over a lifetime and resulting in cost savings of 138 million Thai Baht (THB) compared to BPA prophylaxis. Compared to episodic BPA treatment, emicizumab yielded 30.5 QALYs and saved 25 million THB per patient. The 5-year budget impact was projected at 1775 million THB.
Emicizumab offers a cost-saving approach for HA treatment with inhibitors in Thailand, promising significant health benefits and budgetary savings. This supports its potential inclusion in Thailand's National List of Essential Medicines to enhance haemophilia care access.
Managing haemophilia A (HA) with inhibitors in Thailand, an upper-middle-income country, faces challenges due to limited access to effective treatments or newer drugs for bleeding management. Emicizumab prophylaxis found to as a cost-effective and viable alternative to traditional treatments, effectively preventing bleeding in Thai HA patients over 2 years old with inhibitors. Demonstrating improved clinical outcomes and reduced costs, emicizumab prophylaxis outperforms episodic BPA treatments, positioning it as a superior treatment option for HA patients with inhibitors in Thailand.
在泰国这个中高收入国家，对患有抑制物的甲型血友病（HA）患者进行管理面临重大挑战，控制出血通常需要使用旁路制剂（BPAs）。本研究从社会角度评估了新型预防药物艾美赛珠单抗作为间歇性和预防性BPA治疗替代方案的成本效益和预算影响。
采用马尔可夫模型估计患有抑制物的HA患者接受艾美赛珠单抗预防治疗的终身社会成本和结局。通过全面的文献综述获取治疗效果、成本和流行病学数据，并将其纳入模型。一项为期5年的预算影响分析对成本效用分析进行了补充，未来成本和结局采用3%的年贴现率。
在基础病例情景中，2岁及以上HA患者接受艾美赛珠单抗预防治疗显示出更高的成本效益，每位患者终身可获得18.1个质量调整生命年（QALYs），与BPA预防治疗相比可节省1.38亿泰铢。与间歇性BPA治疗相比，艾美赛珠单抗每位患者可产生30.5个QALYs并节省2500万泰铢。预计5年预算影响为17.75亿泰铢。
在泰国，艾美赛珠单抗为患有抑制物的HA患者提供了一种节省成本的治疗方法，有望带来显著的健康益处和预算节省。这支持了将其纳入泰国基本药物国家清单以改善血友病治疗可及性的可能性。
在泰国这个中高收入国家，由于有效治疗或用于出血管理的新药获取有限，对患有抑制物的甲型血友病（HA）患者进行管理面临挑战。发现艾美赛珠单抗预防治疗是传统治疗的一种具有成本效益且可行的替代方案，可有效预防泰国2岁以上患有抑制物的HA患者出血。艾美赛珠单抗预防治疗显示出改善的临床结局和降低的成本，优于间歇性BPA治疗，使其成为泰国患有抑制物的HA患者的更优治疗选择。
