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在美国,芬戈莫德与干扰素β-1a 治疗复发缓解型多发性硬化症的成本效益比较。

Cost-effectiveness of fingolimod versus interferon beta-1a for relapsing remitting multiple sclerosis in the United States.

机构信息

University of Connecticut School of Pharmacy, Storrs, CT, USA.

出版信息

J Med Econ. 2012;15(6):1088-96. doi: 10.3111/13696998.2012.693553. Epub 2012 May 24.

Abstract

OBJECTIVE

Fingolimod has been shown to be more efficacious than interferon (IFN) beta-1a, but at a higher drug acquisition cost. The aim of this study was to assess the cost-effectiveness of fingolimod compared to IFN beta-1a in patients diagnosed with relapsing-remitting multiple sclerosis (RRMS) in the US.

METHODS

A Markov model comparing fingolimod to intramuscular IFN beta-1a using a US societal perspective and a 10-year time horizon was developed. A cohort of 37-year-old patients with RRMS and a Kurtzke Expanded Disability Status Scale score of 0-2.5 were assumed. Data sources included the Trial Assessing Injectable Interferon vs FTY720 Oral in Relapsing-Remitting Multiple Sclerosis (TRANSFORMS) and other published studies of MS. Outcomes included costs in 2011 US dollars, quality-adjusted life years (QALYs), number of relapses avoided, and incremental cost-effectiveness ratios (ICERs).

RESULTS

Compared to IFN beta-1a, fingolimod was associated with fewer relapses (0.41 vs 0.73 per patient per year) and more QALYs gained (6.7663 vs 5.9503), but at a higher cost ($565,598 vs $505,234). This resulted in an ICER of $73,975 per QALY. Results were most sensitive to changes in drug costs and the disutility of receiving IFN beta-1a. Monte Carlo simulation demonstrated fingolimod was cost-effective in 35% and 70% of 10,000 iterations, assuming willingness-to-pay thresholds of $50,000 and $100,000 per QALY, respectively.

LIMITATIONS

Event rates were primarily derived from a single randomized clinical trial with 1-year duration of follow-up and extrapolated to a 10-year time horizon. Comparison was made to only one disease-modifying drug-intramuscular IFN beta-1a.

CONCLUSION

Fingolimod use is not likely to be cost-effective compared to IFN beta-1a unless fingolimod cost falls below $3476 per month or a higher than normal willingness-to-pay threshold is accepted by decision-makers.

摘要

目的

与干扰素(IFN)β-1a 相比,芬戈莫德的疗效更优,但药物获取成本更高。本研究旨在评估芬戈莫德治疗美国复发缓解型多发性硬化症(RRMS)患者的成本效益。

方法

采用美国社会视角和 10 年时间范围,建立了比较芬戈莫德与肌内 IFNβ-1a 的 Markov 模型。假设一个 37 岁的 RRMS 患者队列,其 Kurtzke 扩展残疾状况量表评分为 0-2.5。数据来源包括评估注射用干扰素与 FTY720 口服治疗 RRMS 的试验(TRANSFORMS)和其他 MS 研究。结果包括 2011 年美元成本、质量调整生命年(QALYs)、避免的复发次数和增量成本效益比(ICER)。

结果

与 IFNβ-1a 相比,芬戈莫德的复发率更低(0.41 次/患者/年比 0.73 次/患者/年),QALYs 更高(6.7663 比 5.9503),但成本更高(565598 美元比 505234 美元)。这导致每 QALY 的 ICER 为 73975 美元。结果对药物成本和接受 IFNβ-1a 的不良感受的变化最为敏感。蒙特卡罗模拟表明,假设支付意愿阈值分别为 50000 美元和 100000 美元/ QALY,在 10000 次迭代的 35%和 70%中,芬戈莫德具有成本效益。

局限性

事件发生率主要来自一项为期 1 年的随机临床试验,并外推至 10 年时间范围。仅与一种疾病修饰药物-肌内 IFNβ-1a 进行比较。

结论

除非芬戈莫德的月成本低于 3476 美元,或者决策者接受高于正常的支付意愿阈值,否则与 IFNβ-1a 相比,芬戈莫德的使用不太可能具有成本效益。

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