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新型心血管疾病二级预防治疗策略:成本效益的系统评价

Novel Treatment Strategies for Secondary Prevention of Cardiovascular Disease: A Systematic Review of Cost-Effectiveness.

机构信息

School of Public Health and Preventive Medicine, Monash University, 553 St Kilda Rd, Melbourne, VIC, 3004, Australia.

Department of Pharmacy, Faculty of Health and Medical Sciences, University of Copenhagen, Copenhagen, Denmark.

出版信息

Pharmacoeconomics. 2020 Oct;38(10):1095-1113. doi: 10.1007/s40273-020-00936-0.

Abstract

BACKGROUND

New pharmacological therapies for the treatment of cardiovascular disease (CVD) have emerged in recent years. The high rates of CVD and the need for long-term treatment to decrease risk factors makes cost-effectiveness crucial for their successful long-term implementation.

OBJECTIVE

This study assessed cost-effectiveness studies of novel pharmacological treatments (ezetimibe, proprotein convertase subtilisin/kexin type 9 [PCSK9] inhibitors, omega-3 polyunsaturated fatty acids [n-3 PUFAs], and the cardiovascular polypill) compared with standard care for the secondary prevention of CVD.

METHODS

We searched seven databases and the reference list of selected literature reviews for eligible cost-effective analyses (CEA) published between January 2009 and January 2020 that evaluated the above novel treatments versus standard care. Two independent reviewers performed the screening and evaluation in accordance with the Consolidated Health Economic Evaluation Reporting Standards statement. Cost results were adapted to 2018 US dollars (US$) to facilitate comparisons between studies. Consideration of cost-effectiveness was based on the original study criteria.

RESULTS

Thirty-two studies were included in this review, most of them adopting a healthcare perspective. Studies evaluating ezetimibe, PCSK9 inhibitors and n-3 PUFAs assessed their addition to standard care compared with standard care alone, while studies analysing the polypill evaluated the replacement of multiple monotherapies for a fixed-dose combination. Ten studies reported on ezetimibe, fifteen evaluated PCSK9 inhibitors, five focused on n-3 PUFAs and seven on the polypill. From a healthcare perspective, ezetimibe was cost effective in 62.5% of the studies (incremental cost-effectiveness ratios [ICERs] ranged from US$27,195 to US$204,140), n-3 PUFAs in 60% (ICERs from US$57,128 to US$139,082) and the cardiovascular polypill in 100% (ICERs from dominant to US$30,731) compared with standard care. Conversely, only 10% of the studies considered PCSK9 inhibitors cost effective compared with standard care from a healthcare perspective (ICERs ranged from US$231,119 to US$1,223,831). Additionally, ezetimibe was cost effective in 50% of the studies, PCSK9 inhibitors in 33% and the polypill in 50% of the studies adopting a societal perspective. The key model-related parameters predicting cost-effectiveness included drug cost, time horizon, and the baseline risk of cardiovascular events.

CONCLUSIONS

Based on current pricing and willingness-to-pay thresholds, most CEA studies considered ezetimibe, n-3 PUFAs and the polypill to be cost effective compared with standard care but not PCSK9 inhibitors for secondary prevention of CVD.

摘要

背景

近年来,出现了一些用于治疗心血管疾病(CVD)的新的药理学疗法。CVD 的高发病率和需要长期治疗以降低风险因素,使得成本效益成为其长期成功实施的关键。

目的

本研究评估了新型药理学治疗(依泽替米贝、前蛋白转化酶枯草溶菌素/糜蛋白酶 9 [PCSK9]抑制剂、ω-3 多不饱和脂肪酸 [n-3 PUFA]和心血管复方药)与 CVD 二级预防的标准治疗相比的成本效益研究。

方法

我们检索了七个数据库和选定文献综述的参考文献列表,以获取 2009 年 1 月至 2020 年 1 月期间发表的评估上述新型治疗方法与标准治疗相比的具有成本效益的分析(CEA)。两名独立的审查员根据合并健康经济评估报告标准声明进行筛选和评估。将成本结果调整为 2018 年美元(US$),以促进研究之间的比较。成本效益的考虑基于原始研究标准。

结果

本综述纳入了 32 项研究,其中大多数采用了医疗保健视角。评估依泽替米贝、PCSK9 抑制剂和 n-3 PUFA 的研究评估了它们与标准治疗相比的添加效果,而分析复方药的研究则评估了用固定剂量组合替代多种单药治疗的效果。有 10 项研究报告了依泽替米贝,15 项研究评估了 PCSK9 抑制剂,5 项研究集中于 n-3 PUFA,7 项研究集中于复方药。从医疗保健的角度来看,依泽替米贝在 62.5%的研究中具有成本效益(增量成本效益比[ICER]范围为 27195 美元至 204140 美元),n-3 PUFA 在 60%的研究中具有成本效益(ICER 范围为 57128 美元至 139082 美元),心血管复方药在 100%的研究中具有成本效益(ICER 范围为占优至 30731 美元),与标准治疗相比。相反,只有 10%的研究认为 PCSK9 抑制剂从医疗保健的角度来看具有成本效益(ICER 范围为 231119 美元至 1223831 美元)。此外,依泽替米贝在 50%的研究、PCSK9 抑制剂在 33%的研究和复方药在 50%的研究中具有成本效益,采用了社会视角。预测成本效益的关键模型相关参数包括药物成本、时间范围和心血管事件的基线风险。

结论

根据当前的定价和支付意愿阈值,与标准治疗相比,大多数 CEA 研究认为依泽替米贝、n-3 PUFA 和复方药具有成本效益,但 PCSK9 抑制剂在 CVD 的二级预防中没有成本效益。

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