MONDAY, April 28, 2025 -- For U.S. patients with severe hemophilia A, prophylaxis with efanesoctocog alfa is neither conventionally nor distributionally cost-effective, according to a study published online April 22 in the

2025年4月28日，星期一——根据4月22日在线发表在《美国医学会杂志》上的一项研究，对于美国重度A型血友病患者，使用efanesoctocog alfa进行预防治疗既不符合传统的成本效益标准，也不符合分布式的成本效益标准。

Annals of Internal Medicine

内科医学年鉴

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Satoko Ito, M.D., Ph.D., from the Yale School of Medicine in New Haven, Connecticut, and colleagues examined the conventional and distributional cost-effectiveness of prophylaxis with efanesoctocog alfa versus standard-care factor VIII (standard and extended half-life) in the United States using a Markov model..

来自康涅狄格州纽黑文耶鲁医学院的伊藤聪医学博士及其同事使用马尔可夫模型，评估了在美国使用efanesoctocog alfa预防与标准护理因子VIII（标准和延长半衰期）的传统和分布成本效益。

The researchers found that efanesoctocog alfa and standard-care factor VIII accrued 19.7 and 14.6 discounted lifetime quality-adjusted life years (QALYs) at costs of $22.1 and $11.2 million, respectively; the incremental cost-effectiveness ratio per QALY was $2.13 million. The distributional cost-effectiveness analysis-derived threshold equity weight was 6.9, which exceeded U.S.

研究人员发现，efanesoctocog alfa 和标准护理因子 VIII 在其生命周期内分别累计获得了 19.7 和 14.6 个质量调整生命年（QALYs），对应的成本分别为 2210 万美元和 1120 万美元；每 QALY 的增量成本效益比为 213 万美元。分布成本效益分析得出的阈值公平权重为 6.9，超过了美国的标准。

equity standards that are commonly used. Across all scenario analyses, the results were consistent. In a sensitivity analysis, the conclusion could only be changed by the price of efanesoctocog alfa. For conventional and distributional cost-effectiveness, efanesoctocog alfa needs to be priced at less than 53 and less than 59 percent of its current price, respectively.

常用的标准。在所有情景分析中，结果都是一致的。在敏感性分析中，只有efanesoctocog alfa的价格能够改变结论。对于常规和分布成本效益而言，efanesoctocog alfa的价格分别需要低于其当前价格的53%和59%。

In 100 percent of 10,000 probabilistic iterations, standard-care factor VIII was favored..

在10000次概率迭代中，有100%的情况下，标准护理的第八因子更受青睐。

'Alternative pricing mechanisms, longer-term clinical follow-up data, and studies across additional jurisdictions where efanesoctocog alfa is approved will be critical to understanding the value of this therapy in the context of existing treatment options in the hemophilia A arena,' the authors write..

“替代性定价机制、更长期的临床随访数据，以及在efanesoctocog alfa获批的其他司法管辖区开展的研究，对于理解该疗法在A型血友病现有治疗选择中的价值至关重要，”作者写道。

Several authors disclosed ties to relevant organizations.

几位作者披露了与相关组织的联系。

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