Cost-effectiveness of caplacizumab in immune thrombotic thrombocytopenic purpura in the United States.

IF 2.3 4区 医学 Q2 HEALTH CARE SCIENCES & SERVICES
Sean D Sullivan, Shruti Chaturvedi, Preety Gautam, Alix Arnaud
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Abstract

Background: Immune thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening thrombotic microangiopathy. Caplacizumab is the only treatment approved by the European Medicines Agency and the US Food and Drug Administration for iTTP, to be given in combination with plasma exchange therapy (PEX) and immunosuppression (IS). The National Institute for Health and Care Excellence's independent appraisal committee assessed the cost-effectiveness of caplacizumab and concluded that the addition of caplacizumab to PEX+IS is cost-effective under a patient access scheme in the United Kingdom.

Objective: To assess the cost-effectiveness of caplacizumab in iTTP from the US payer perspective.

Methods: The National Institute for Health and Care Excellence's model was adapted to the US setting using US costs and discount rates. In contrast to previous cost-effectiveness analyses that accounted only for acute outcomes, our model consisted of a 3-month decision tree for an acute iTTP episode, followed by a Markov model to project long-term costs and outcomes (time horizon: up to 55 years; 3-monthly cycles).

Results: Patients taking caplacizumab with PEX+IS experienced an incremental gain of 2.96 life years (LYs) and 1.75 quality-adjusted LYs relative to PEX+IS alone, at an increased lifetime cost of $256,000. The incremental cost-effectiveness ratio was $86,400 per LY and $146,300 per quality-adjusted LY gained.

Conclusions: Considering willingness-to-pay thresholds of $150,000 to $200,000, the addition of caplacizumab to PEX+IS may be cost-effective compared with PEX+IS alone for the treatment of iTTP in a US setting.

美国卡普拉珠单抗治疗免疫性血栓性血小板减少性紫癜的成本效益
背景:免疫性血栓性血小板减少性紫癜(iTTP)是一种罕见的危及生命的血栓性微血管疾病。Caplacizumab是欧洲药品管理局和美国食品和药物管理局批准的唯一治疗iTTP的药物,可与血浆交换疗法(PEX)和免疫抑制(is)联合使用。国家健康与护理卓越研究所的独立评估委员会评估了caplacizumab的成本效益,并得出结论,在英国的患者准入计划下,在PEX+IS中添加caplacizumab具有成本效益。目的:从美国付款人的角度评估卡帕单抗在iTTP治疗中的成本-效果。方法:采用美国成本和贴现率,对国家卫生与保健卓越研究所的模型进行了调整,以适应美国的情况。与以往只考虑急性结局的成本效益分析不同,我们的模型包括急性iTTP发作的3个月决策树,然后是预测长期成本和结果的马尔可夫模型(时间范围:长达55年;三个月周期)。结果:与单独使用PEX+IS相比,服用caplacizumab联合PEX+IS的患者获得了2.96个生命年(LYs)和1.75个质量调整生命年,增加的生命周期成本为25.6万美元。增加的成本效益比为每项LY $86 400,每项质量调整LY $146 300。结论:考虑到支付意愿阈值为15万至20万美元,在美国,与单独使用PEX+IS相比,在PEX+IS中添加caplacizumab治疗iTTP可能更具成本效益。
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来源期刊
Journal of managed care & specialty pharmacy
Journal of managed care & specialty pharmacy Health Professions-Pharmacy
CiteScore
3.50
自引率
4.80%
发文量
131
期刊介绍: JMCP welcomes research studies conducted outside of the United States that are relevant to our readership. Our audience is primarily concerned with designing policies of formulary coverage, health benefit design, and pharmaceutical programs that are based on evidence from large populations of people. Studies of pharmacist interventions conducted outside the United States that have already been extensively studied within the United States and studies of small sample sizes in non-managed care environments outside of the United States (e.g., hospitals or community pharmacies) are generally of low interest to our readership. However, studies of health outcomes and costs assessed in large populations that provide evidence for formulary coverage, health benefit design, and pharmaceutical programs are of high interest to JMCP’s readership.
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