德国治疗中重度血友病 B 的 Etranacogene Dezaparvovec 与延长半衰期预防疗法的成本效益分析。

IF 2 Q2 ECONOMICS
PharmacoEconomics Open Pub Date : 2024-05-01 Epub Date: 2024-03-23 DOI:10.1007/s41669-024-00480-z
Niklaus Meier, Hendrik Fuchs, Katya Galactionova, Cedric Hermans, Mark Pletscher, Matthias Schwenkglenks
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引用次数: 0

摘要

背景和目的:血友病 B 是一种罕见的遗传性疾病,由血液中缺乏凝血因子 IX(FIX)引起,会导致体内和体外出血。根据目前的治疗标准,血友病的治疗要么是预防性治疗,要么是通过静脉注射 FIX 按需治疗。由于血友病 B 需要终生治疗,而 FIX 又价格昂贵,因此这些治疗策略给患者和医疗系统带来了沉重负担。Etranacogene dezaparvovec (ED) 是一种治疗 B 型血友病的基因疗法,最近已获得美国食品药品管理局批准,并获得了欧洲药品管理局的有条件上市许可建议。我们的目的是从德国医疗支付方的角度来考察 ED 与延长半衰期 FIX(EHL-FIX)预防中重度 B 型血友病的成本效益:该模型使用 ED 第 3 期临床试验出版物中的数据和其他二手数据源,模拟和比较患者在一生中接受 ED 或 EHL-FIX 预防治疗的情况,当 ED 的疗效减弱时,ED 患者有可能转而接受 EHL-FIX 预防治疗。该分析的主要结果包括贴现总成本、贴现质量调整生命年 (QALY)、增量成本效益和增量净货币效益。成本和效果的年贴现率为 3%。通过概率分析和额外的单变量敏感性分析对不确定性进行了研究:结果:概率分析显示,以每次 ED 治疗花费 150 万欧元计算,接受 ED 而非 EHL-FIX 预防治疗的患者可获得 0.50 QALYs,节省成本 117.9829 万欧元。ED 是最主要的治疗策略。按 50,000 欧元/QALY 的支付意愿计算,增量净货币效益为 1,204,840.00 欧元:讨论:与 EHL-FIX 预防疗法相比,ED 可为血友病患者节约成本并改善健康状况,因此是一种具有潜在成本效益的替代疗法,具体取决于价格。由于缺乏有关 ED 长期有效性的证据,这些结果还不确定。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Cost-Effectiveness Analysis of Etranacogene Dezaparvovec Versus Extended Half-Life Prophylaxis for Moderate-to-Severe Haemophilia B in Germany.

Background and objective: Haemophilia B is a rare genetic disease that is caused by a deficiency of coagulation factor IX (FIX) in the blood and leads to internal and external bleeding. Under the current standard of care, haemophilia is treated either prophylactically or on-demand via intravenous infusions of FIX. These treatment strategies impose a high burden on patients and health care systems as haemophilia B requires lifelong treatment, and FIX is costly. Etranacogene dezaparvovec (ED) is a gene therapy for haemophilia B that has been recently approved by the United States Food and Drug Administration and has received a recommendation for conditional marketing authorization by the European Medicines Agency. We aimed to examine the cost-effectiveness of ED versus extended half-life FIX (EHL-FIX) prophylaxis for moderate-to-severe haemophilia B from a German health care payer perspective.

Methods: A microsimulation model was implemented in R. The model used data from the ED phase 3 clinical trial publication and further secondary data sources to simulate and compare patients receiving ED or EHL-FIX prophylaxis over a lifetime horizon, with the potential for ED patients to switch treatment to EHL-FIX prophylaxis when the effectiveness of ED waned. Primary outcomes of this analysis included discounted total costs, discounted quality-adjusted life years (QALYs), incremental cost-effectiveness, and the incremental net monetary benefit. The annual discount rate for costs and effects was 3%. Uncertainty was examined via probabilistic analysis and additional univariate sensitivity analyses.

Results: Probabilistic analysis indicated that patients treated with ED instead of EHL-FIX prophylaxis gained 0.50 QALYs and experienced cost savings of EUR 1,179,829 at a price of EUR 1,500,000 per ED treatment. ED was the dominant treatment strategy. At a willingness to pay of EUR 50,000/QALY, the incremental net monetary benefit amounted to EUR 1,204,840.

Discussion: Depending on the price, ED can save costs and improve health outcomes of haemophilia patients compared with EHL-FIX prophylaxis, making it a potentially cost-effective alternative. These results are uncertain due to a lack of evidence regarding the long-term effectiveness of ED.

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来源期刊
CiteScore
3.50
自引率
0.00%
发文量
64
审稿时长
8 weeks
期刊介绍: PharmacoEconomics - Open focuses on applied research on the economic implications and health outcomes associated with drugs, devices and other healthcare interventions. The journal includes, but is not limited to, the following research areas:Economic analysis of healthcare interventionsHealth outcomes researchCost-of-illness studiesQuality-of-life studiesAdditional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in PharmacoEconomics -Open may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances.All manuscripts are subject to peer review by international experts. Letters to the Editor are welcomed and will be considered for publication.
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