在英国，对于无抑制剂的血友病 A 成人和青少年患者，重组凝血因子 VIII Fc 与emicizumab 预防治疗的成本效益比较。
Cost-effectiveness of recombinant factor VIII Fc versus emicizumab for prophylaxis in adults and adolescents with haemophilia A without inhibitors in the UK.
机构信息
Global Health Economics and Outcomes Research, Swedish Orphan Biovitrum AB (Sobi™), Stockholm, Sweden.
Health Economics and Outcomes Research Department, Putnam PHMR, Krakow, Poland.
出版信息
Eur J Haematol. 2023 Mar;110(3):262-270. doi: 10.1111/ejh.13901. Epub 2022 Dec 11.
INTRODUCTION
The economic and clinical burden of haemophilia A is high. Primary prophylaxis with factor VIII replacement therapy is the recognised standard of care, but the emergence of non-factor therapies, such as emicizumab, is extending treatment options for people with haemophilia A.
AIM
There are currently no direct comparisons of efficacy or cost between recombinant factor FVIII Fc-fusion protein efmoroctocog alfa (a recombinant factor FVIII Fc-fusion protein referred to herein as rFVIIIFc) and emicizumab; therefore, a cost-effectiveness model was developed to compare prophylactic treatment with rFVIIIFc versus emicizumab in patients with haemophilia A without inhibitors in the UK.
METHODS
The cost-effectiveness model was based on a matching-adjusted indirect comparison and included male patients, aged ≥12 years, with haemophilia A without inhibitors. The model was designed as a Markov process with a flexible lifelong time horizon, and cost-effectiveness was presented as an incremental cost-effectiveness ratio. Base-case analysis and sensitivity analyses (including scenario analyses, one-way deterministic sensitivity analysis [DSA] and probability sensitivity analysis [PSA]) were performed using the following treatment strategies: individualised prophylaxis with rFVIIIFc and prophylaxis with emicizumab administered once weekly (scenario analyses used regimens of once every 2 weeks or once every 4 weeks).
RESULTS
Base-case analysis, DSA and PSA indicated that, compared with emicizumab administered once weekly, rFVIIIFc individualised prophylaxis was the dominant treatment strategy, with lower costs, a greater number of quality-adjusted life years, and a lower number of bleeds.
CONCLUSIONS
rFVIIIFc has proven efficacy and is cost-effective compared with emicizumab, providing clinicians with a viable treatment option to improve the health outcomes for adults and adolescents with haemophilia A in the UK.
简介
甲型血友病的经济和临床负担很高。使用凝血因子 VIII 替代疗法进行初级预防是公认的标准治疗方法，但非因子疗法（如emicizumab）的出现为甲型血友病患者提供了更多的治疗选择。
目的
目前尚无 rFVIIIFc（一种重组凝血因子 VIII Fc 融合蛋白）与 emicizumab 在疗效和成本方面的直接比较；因此，本研究开发了一个成本效益模型，以比较英国无抑制剂的甲型血友病患者使用 rFVIIIFc 与 emicizumab 进行预防性治疗的效果。
方法
该成本效益模型基于匹配调整间接比较，包括年龄≥12 岁、无抑制剂的甲型血友病男性患者。模型设计为具有灵活的终生时间范围的马尔可夫过程，成本效益以增量成本效益比表示。使用以下治疗策略进行基础案例分析和敏感性分析（包括方案分析、单因素确定性敏感性分析 [DSA] 和概率敏感性分析 [PSA]）：rFVIIIFc 的个体化预防和每周一次的 emicizumab 预防（方案分析使用每 2 周或每 4 周一次的方案）。
结果
基础案例分析、DSA 和 PSA 表明，与每周一次的 emicizumab 相比，rFVIIIFc 的个体化预防是一种具有成本效益的治疗策略，其成本更低，质量调整生命年更多，出血更少。
结论
rFVIIIFc 与 emicizumab 相比具有疗效优势，且具有成本效益，为英国的成年和青少年甲型血友病患者提供了一种可行的治疗选择，可改善其健康结局。
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