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一项离散事件模拟,用于模拟在英国快速进展的重度复发缓解型多发性硬化症中,芬戈莫德和那他珠单抗的成本效益。

A discrete event simulation to model the cost-utility of fingolimod and natalizumab in rapidly evolving severe relapsing-remitting multiple sclerosis in the UK.

作者信息

Montgomery Stephen M, Maruszczak Maciej J, Slater David, Kusel Jeanette, Nicholas Richard, Adlard Nicholas

机构信息

a Costello Medical Consulting Ltd , Cambridge , UK.

b Imperial College Healthcare NHS Trust, Charing Cross Hospital , London , UK.

出版信息

J Med Econ. 2017 May;20(5):474-482. doi: 10.1080/13696998.2016.1276070. Epub 2017 Jan 23.

Abstract

OBJECTIVE

Two disease-modifying therapies are licensed in the EU for use in rapidly-evolving severe (RES) relapsing-remitting multiple sclerosis (RRMS), fingolimod and natalizumab. Here a discrete event simulation (DES) model to analyze the cost-effectiveness of natalizumab and fingolimod in the RES population, from the perspective of the National Health Service (NHS) in the UK, is reported.

METHODS

A DES model was developed to track individual RES patients, based on Expanded Disability Status Scale scores. Individual patient characteristics were taken from the RES sub-groups of the pivotal trials for fingolimod. Utility data were in line with previous models. Published costs were inflated to NHS cost year 2015. Owing to the confidential patient access scheme (PAS) discount applied to fingolimod in the UK, a range of discount levels were applied to the fingolimod list price, to capture the likelihood of natalizumab being cost-effective in a real-world setting.

RESULTS

At the lower National Institute of Health and Care Excellence (NICE) threshold of £20,000/quality-adjusted life year (QALY), fingolimod only required a discount greater than 0.8% of list price to be cost-effective. At the upper threshold of £30,000/QALY employed by the NICE, fingolimod was cost-effective if the confidential discount is greater than 2.5%. Sensitivity analyses conducted using fingolimod list-price showed the model to be most sensitive to changes in the cost of each drug, particularly fingolimod.

CONCLUSIONS

The DES model shows that only a modest discount to the UK fingolimod list-price is required to make fingolimod a more cost-effective option than natalizumab in RES RRMS.

摘要

目的

在欧盟,有两种疾病改善疗法被批准用于快速进展型严重(RES)复发缓解型多发性硬化症(RRMS),即芬戈莫德和那他珠单抗。本文报告了一个离散事件模拟(DES)模型,该模型从英国国家医疗服务体系(NHS)的角度分析了那他珠单抗和芬戈莫德在RES人群中的成本效益。

方法

基于扩展残疾状态量表评分,开发了一个DES模型来跟踪个体RES患者。个体患者特征取自芬戈莫德关键试验的RES亚组。效用数据与先前模型一致。已公布的成本被上调至2015年NHS成本年度。由于英国对芬戈莫德实施了保密患者准入计划(PAS)折扣,因此对芬戈莫德标价应用了一系列折扣水平,以了解那他珠单抗在现实环境中具有成本效益的可能性。

结果

在英国国家卫生与临床优化研究所(NICE)较低的阈值20,000英镑/质量调整生命年(QALY)下,芬戈莫德只需折扣大于标价的0.8%就具有成本效益。在NICE采用的较高阈值30,000英镑/QALY下,如果保密折扣大于2.5%,芬戈莫德则具有成本效益。使用芬戈莫德标价进行的敏感性分析表明,该模型对每种药物成本的变化最为敏感,尤其是芬戈莫德。

结论

DES模型表明,只需对英国芬戈莫德标价进行适度折扣,就能使芬戈莫德在RES RRMS中成为比那他珠单抗更具成本效益的选择。

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