对用于冠心病二级预防的降低脂蛋白(a)的基因沉默疗法进行早期卫生技术评估。

IF 3.6 3区 医学 Q2 PHARMACOLOGY & PHARMACY
Angela Burvill, Gerald F Watts, Richard Norman, Zanfina Ademi
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引用次数: 0

摘要

背景:奥帕西兰和佩拉卡森是能降低脂蛋白(a)的基因沉默疗法。心血管结果试验正在进行中。孟德尔随机研究估计了降低脂蛋白(a)的临床疗效:我们的研究估算了在澳大利亚医疗保健系统中,除标准治疗外,奥帕西然和皮拉卡森在降低冠心病(CHD)复发风险方面的成本效益价格:我们开发了决策树和终身马尔可夫模型。对于奥帕西兰,参与者在基线和三个月注射时均有冠心病和脂蛋白(a)260 nmol/L,并在OCEAN(a)结果试验(NCT05581303)中进行了分析。基线冠心病风险、成本和效用均来自公开资料。临床试验数据用于推导治疗对脂蛋白(a)的降低作用。孟德尔随机化研究数据用于估算下游临床效益。年贴现率为 5%。对于pelacarsen,参试者有CHD,基线时脂蛋白(a)为226 nmol/L,每月注射一次,根据脂蛋白(a)HORIZON(NCT04023552)试验进行分析:结果:除标准治疗外,Olpasiran 可为每人节省 0.87 个质量调整生命年(QALYs)。按每年 1867 澳元(每剂 467 澳元)的价格计算,奥帕西兰作为标准治疗的补充具有成本效益,每 QALY 的临界值为 28,000 澳元。Pelacarsen的成本效益为每年984澳元(每剂82澳元)。在每 QALY 的 ICER 临界值为 50,000 澳元时,奥帕西然和培拉伐森的成本效益分别为每年 4207 澳元和 2464 澳元:这一早期健康技术评估模型采用了临床试验的纳入标准。从澳大利亚医疗保健的角度来看,如果年度治疗价格分别为 1867 澳元和 984 澳元,奥帕西然和培拉伐森将具有成本效益。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Early health technology assessment of gene silencing therapies for lowering lipoprotein(a) in the secondary prevention of coronary heart disease.

Background: Olpasiran and pelacarsen are gene-silencing therapies that lower lipoprotein(a). Cardiovascular outcome trials are ongoing. Mendelian randomisation studies estimated clinical benefits from lipoprotein(a) lowering.

Objective: Our study estimated prices at which olpasiran and pelacarsen, in addition to standard-of-care, would be deemed cost-effective in reducing risk of recurrent coronary heart disease (CHD) events in the Australian healthcare system.

Methods: We developed a decision tree and lifetime Markov model. For olpasiran, participants had CHD and lipoprotein(a) 260 nmol/L at baseline and three-monthly injections, profiled on OCEAN(a) Outcomes trial (NCT05581303). Baseline risks of CHD, costs and utilities were obtained from published sources. Clinical trial data were used to derive reductions in lipoprotein(a) from treatment. Mendelian randomisation study data were used to estimate downstream clinical benefits. Annual discounting was 5 %. For pelacarsen, participants had CHD and lipoprotein(a) 226 nmol/L at baseline and one- monthly injections, profiled on Lp(a) HORIZON (NCT04023552) trial.

Results: Olpasiran in addition to standard-of-care saved 0.87 discounted quality-adjusted life years (QALYs) per person. Olpasiran in addition to standard-of-care would be cost- effective at annual prices of AU$1867 (AU$467 per dose) at threshold AU$28,000 per QALY. Pelacarsen would be cost-effective at annual prices of AU$984 (AU$82 per dose). For ICER threshold AU$50,000 per QALY, olpasiran and pelacarsen would be cost-effective at annual prices AU$4207 and AU$2464 respectively.

Conclusion: This early health technology assessment model used inclusion criteria from clinical trials. Olpasiran and pelacarsen would be cost-effective if annual treatment prices were AU$1867 and AU$984 respectively, from the Australian healthcare perspective.

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来源期刊
CiteScore
7.00
自引率
6.80%
发文量
209
审稿时长
49 days
期刊介绍: Because the scope of clinical lipidology is broad, the topics addressed by the Journal are equally diverse. Typical articles explore lipidology as it is practiced in the treatment setting, recent developments in pharmacological research, reports of treatment and trials, case studies, the impact of lifestyle modification, and similar academic material of interest to the practitioner. While preference is given to material of immediate practical concern, the science that underpins lipidology is forwarded by expert contributors so that evidence-based approaches to reducing cardiovascular and coronary heart disease can be made immediately available to our readers. Sections of the Journal will address pioneering studies and the clinicians who conduct them, case studies, ethical standards and conduct, professional guidance such as ATP and NCEP, editorial commentary, letters from readers, National Lipid Association (NLA) news and upcoming event information, as well as abstracts from the NLA annual scientific sessions and the scientific forums held by its chapters, when appropriate.
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