Majda Thomas, Lee Janet S, Curtis Randall, Kowal Stacey L
Genentech, South San Francisco, California, USA
Genentech, South San Francisco, California, USA.
BMJ Open. 2025 Sep 9;15(9):e103112. doi: 10.1136/bmjopen-2025-103112.
Emicizumab is the first bispecific antibody approved for prophylaxis in people with haemophilia A with or without factor VIII inhibitors. Aggregate distributional cost-effectiveness analysis assesses health equity impacts by evaluating how health effects and costs from funding an intervention are distributed among population subgroups. The objective was to evaluate how funding emicizumab for people with severe haemophilia A (PwSHA) impacts population health and health disparities in the USA.
Population-level model of PwSHA from the perspective of the US healthcare system, using published sources and considering a lifetime time horizon.
Emicizumab versus other haemophilia A prophylaxis treatments.
Quality-adjusted life-years (QALYs) gained and change in Atkinson index of inequality in quality-adjusted life expectancy.
When an estimated 6512 PwSHA in the USA were treated with emicizumab, the US healthcare system would save US$160 billion over those individuals' lifetimes. If these cost savings fund additional healthcare interventions in the overall population, funding emicizumab would improve overall US population health (1 068 903 QALYs gained, using a threshold of US$150 000/QALY) and reduce existing overall US inequities (-0.01% on the Atkinson index).In all scenarios tested for sensitivity, increased emicizumab and prophylaxis utilisation led to further reductions in health disparities and greater increases in population health. Results were robust to deterministic variations in the allocation of cost savings due to emicizumab use.
Funding emicizumab treatments for PwSHA improves overall population health and reduces overall health inequities in the USA. Cost savings from the use of emicizumab free up important resources that can be leveraged to support other healthcare interventions, but decisions on how these funds are used have large consequences for equity.
艾美赛珠单抗是首个被批准用于预防有或没有凝血因子VIII抑制剂的A型血友病患者的双特异性抗体。总体分布成本效益分析通过评估资助一项干预措施所产生的健康影响和成本在人群亚组中的分配情况，来评估健康公平性影响。目的是评估为重度A型血友病患者（PwSHA）提供艾美赛珠单抗资助对美国人群健康和健康差异的影响。
从美国医疗保健系统的角度建立PwSHA的人群水平模型，使用已发表的资料并考虑终身时间范围。
艾美赛珠单抗与其他A型血友病预防治疗方法对比。
获得的质量调整生命年（QALY）以及质量调整预期寿命的阿特金森不平等指数的变化。
在美国，当估计有6512名PwSHA接受艾美赛珠单抗治疗时，美国医疗保健系统在这些个体的一生中将节省1600亿美元。如果这些成本节省用于为总体人群提供额外的医疗保健干预措施，资助艾美赛珠单抗将改善美国总体人群健康（获得1068903个QALY，使用阈值为150000美元/QALY）并减少美国现有的总体不平等（阿特金森指数下降0.01%）。在所有敏感性测试情景中，增加艾美赛珠单抗和预防治疗的使用导致健康差异进一步减少，人群健康增加更多。结果对于因使用艾美赛珠单抗而节省成本的分配中的确定性变化具有稳健性。
为PwSHA提供艾美赛珠单抗治疗资金可改善美国总体人群健康并减少总体健康不平等。使用艾美赛珠单抗节省的成本释放了重要资源，可用于支持其他医疗保健干预措施，但关于如何使用这些资金的决策对公平性有重大影响。
