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在英国,对于活跃的继发进展型多发性硬化症的治疗,西尼莫德与继续使用现有疾病修正疗法相比,哪种更具成本效益?

Stick or twist? Cost-effectiveness of siponimod compared with continuing existing disease-modifying therapies in the treatment of active secondary progressive multiple sclerosis in the UK.

机构信息

Costello Medical Consulting Ltd, Cambridge, UK.

Novartis Ireland Limited, Dublin, Ireland.

出版信息

J Med Econ. 2022 Jan-Dec;25(1):669-678. doi: 10.1080/13696998.2022.2078103.

Abstract

OBJECTIVE

Identification of the phenotypic transition from relapsing-remitting multiple sclerosis (RRMS) to secondary progressive multiple sclerosis (SPMS) is often delayed due to disease complexity and an unwillingness to withdraw RRMS disease-modifying therapies (DMTs), driven by limited SPMS treatment options. Despite the paucity of clinical evidence for efficacy in patients with SPMS, DMTs licensed for RRMS are frequently continued into the early stages of SPMS. The cost-effectiveness of oral siponimod, an active SPMS DMT, versus continued oral or infused RRMS DMTs for patients with active SPMS, was evaluated.

METHODS

A cohort Markov model based on disease progression through Expanded Disability Status Scale health states, with annual cycles and lifetime horizon, was employed to determine the cost-effectiveness of siponimod from a UK National Health Service (NHS) perspective for patients with active SPMS. Baseline characteristics, health state utility values, hazard ratios for time to 6-month confirmed disability progression, annualized relapse rate ratios and adverse events for siponimod were obtained from the phase 3 EXPAND clinical trial, supplemented by published literature. Published costs, resource use data and comparator efficacy data were obtained from the literature and, in the absence of data, reasonable assumptions were made.

RESULTS

Quality-adjusted life years (QALYs) were greater for siponimod versus all comparators (3.45 versus 2.69-2.83). Incremental cost-effectiveness ratios (ICERs), calculated as cost per QALY, for siponimod versus natalizumab (dominant), ocrelizumab (£4,760), fingolimod (£10,033) and dimethyl fumarate (£15,837) indicated that siponimod was cost-effective at the commonly accepted willingness-to-pay threshold of £30,000/QALY.

CONCLUSIONS

Recognition of active SPMS and treatment of this phenotype with siponimod offers a cost-effective and clinically beneficial treatment approach compared with the continuation of oral or infused RRMS DMTs.

摘要

目的

由于疾病的复杂性和不愿停止缓解复发型多发性硬化症(RRMS)的疾病修正疗法(DMT),导致继发进展型多发性硬化症(SPMS)的识别常常被延迟,这是因为 SPMS 的治疗选择有限。尽管 SPMS 患者的临床疗效证据有限,但在 SPMS 的早期阶段,仍经常继续使用 RRMS 获批的 DMT。评估了活性 SPMS DMT 西尼莫德口服制剂与继续使用 RRMS DMT 口服或输注治疗活动性 SPMS 患者的成本效益。

方法

采用基于疾病进展通过扩展残疾状况量表健康状态的队列马尔可夫模型,具有年度周期和终生视野,从英国国家医疗服务体系(NHS)的角度确定西尼莫德对活动性 SPMS 患者的成本效益。从 3 期 EXPAND 临床试验中获得西尼莫德的基线特征、健康状态效用值、6 个月确认残疾进展的时间风险比、年化复发率比和不良事件,并用已发表的文献进行补充。从文献中获得已发表的成本、资源使用数据和比较剂疗效数据,在没有数据的情况下,做出了合理的假设。

结果

与所有对照相比,西尼莫德的质量调整生命年(QALY)更高(3.45 与 2.69-2.83)。西尼莫德与那他珠单抗(占优)、奥瑞珠单抗 (£4760)、芬戈莫德 (£10033)和富马酸二甲酯 (£15837)相比,增量成本效益比(ICER),即每 QALY 的成本,表明西尼莫德在普遍接受的 30000 英镑/QALY 的支付意愿阈值内具有成本效益。

结论

与继续使用 RRMS 的 DMT 口服或输注相比,识别活动性 SPMS 并使用西尼莫德治疗这种表型提供了一种具有成本效益和临床获益的治疗方法。

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