Bullement Ash, McMordie Samuel Thomas, Hatswell Anthony James, Li Nanxin, Wilson Koo
Delta Hat, Nottingham, UK.
University College London, London, UK.
Pharmacoecon Open. 2020 Mar;4(1):133-142. doi: 10.1007/s41669-019-0158-8.
Patients with severe hemophilia A (SHA) in Italy are routinely treated with standard half-life recombinant factor VIII (rFVIII) products. rFVIII Fc-fusion protein (rFVIIIFc) is an extended half-life rFVIII product that enables less frequent administration than rFVIII, which may support improved adherence. Available data indicate low breakthrough bleed rates and potentially improved long-term joint health for patients treated with rFVIIIFc prophylaxis.
This study assessed the cost effectiveness of rFVIIIFc versus rFVIII from an Italian healthcare perspective.
A Semi-Markov model was constructed to assess the lifetime costs and benefits of rFVIII and rFVIIIFc prophylaxis. rFVIII product acquisition costs from a published Italian database were included for both prophylaxis and the resolution of breakthrough bleeding. Clinical outcomes within the model were determined based on published annualized bleeding rates and literature regarding the development of target joints (TJs) as the incidence of bleeds and TJs is associated with impaired health-related quality of life. Cost effectiveness was assessed using cost per quality-adjusted life-year (QALY) gained.
Compared with rFVIII, rFVIIIFc was associated with a per-patient cost saving of approximately €1.3 million and QALY gains of 0.39 over a lifetime horizon. Sensitivity analyses considering alternative efficacy, dosing, and structural assumptions each showed that rFVIIIFc dominated rFVIII (i.e., provided more QALYs at a reduced cost).
This cost-effectiveness analysis demonstrated that rFVIIIFc may offer a cost-effective treatment option for patients with SHA in Italy.
在意大利，重度A型血友病（SHA）患者通常接受标准半衰期重组凝血因子VIII（rFVIII）产品治疗。rFVIII Fc融合蛋白（rFVIIIFc）是一种延长半衰期的rFVIII产品，与rFVIII相比，给药频率更低，这可能有助于提高依从性。现有数据表明，接受rFVIIIFc预防治疗的患者突破性出血率较低，长期关节健康状况可能得到改善。
本研究从意大利医疗保健的角度评估了rFVIIIFc与rFVIII的成本效益。
构建了一个半马尔可夫模型，以评估rFVIII和rFVIIIFc预防治疗的终身成本和效益。预防治疗和突破性出血的解决均纳入了来自意大利已发表数据库的rFVIII产品采购成本。模型中的临床结局根据已发表的年化出血率以及关于靶关节（TJ）发展的文献确定，因为出血和TJ的发生率与健康相关生活质量受损有关。使用每获得一个质量调整生命年（QALY）的成本来评估成本效益。
与rFVIII相比，rFVIIIFc在患者终身期间每位患者可节省成本约130万欧元，QALY增加0.39。考虑替代疗效、给药方案和结构假设的敏感性分析均表明，rFVIIIFc优于rFVIII（即，以更低的成本提供更多的QALY）。
这项成本效益分析表明，rFVIIIFc可能为意大利的SHA患者提供一种具有成本效益的治疗选择。
