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新西兰社会与医疗保健视角下奥瑞珠单抗治疗原发性进行性多发性硬化症的成本效益分析

Societal Versus Healthcare Perspectives on the Cost Effectiveness of Ocrelizumab for Treatment of Primary Progressive Multiple Sclerosis in Aotearoa New Zealand.

作者信息

Milne Richard J, Schousboe Carsten, Campbell Julie A, Mottershead John

机构信息

Health Outcomes Associates Ltd, Christchurch, New Zealand.

Roche Singapore Pte Ltd, Singapore, Singapore.

出版信息

Pharmacoeconomics. 2025 May 7. doi: 10.1007/s40273-025-01486-z.

Abstract

OBJECTIVES

Multiple sclerosis (MS) is a progressive, degenerative, autoimmune neuronal disease. This study compares the impact of a societal versus a healthcare perspective on the cost effectiveness of treatment of primary progressive MS (PPMS) with ocrelizumab (OCR) versus best supportive care (BSC) in New Zealand.

METHODS

The analysis utilises a lifetime cohort Markov model based on ten Expanded Disability Status Scale (EDSS) states, plus death. It has two structurally identical arms, with forward transition probabilities in the treatment arm obtained by multiplying forward transition probabilities (converted to rates, and back to probabilities) in the control arm by the 12-week disability progression hazard ratio in the clinical trial ORATORIO. Direct and indirect costs of MS were estimated from a 2017 Australian survey and converted to 2024 NZ dollars using purchasing power parity and the NZ consumer price index. Future costs and benefits were discounted at 3.5% per annum. The model is calibrated to NZ mortality for PPMS, and therapy ends when wheelchair dependence (EDSS7) is reached.

RESULTS

For a modelled cohort 40 years of age starting at the ORATORIO distribution of EDSS, at 50% of the list price (viz. 50% rebate on $NZ37,384 per patient per annum), the incremental cost-effectiveness ratio (ICER) of OCR versus BSC is $NZ114,427 per QALY ($US64,651) from a societal perspective or $NZ146,711 ($US82,892) from a healthcare perspective. From a societal perspective, at Treasury's willingness to pay (WTP) criterion of $NZ120,200, an acquisition cost up to 56% of list price ($NZ20,935; 44% rebate) is justifiable. From a healthcare payer perspective, at Treasury's implied WTP of $NZ43,313, an acquisition cost up to 10% of list price ($NZ3738; 90% rebate) is justifiable. Based on this metric, a 5.6-fold higher investment in OCR can be justified from a societal perspective compared with a healthcare perspective. Alternatively, an acquisition cost up to 37.9% of list price (viz. 62.1% rebate or $NZ14,169) could be justified if the criterion of one GDP per capita ($NZ83,011) is used as a societal funding threshold. These results are sensitive to the cost of BSC from a societal perspective but not from a healthcare perspective. From both perspectives the cost effectiveness is sensitive to the acquisition cost and efficacy of OCR, potential waning of efficacy, the age and EDSS state when therapy begins, the cost and timing of entry of a biosimilar pharmaceutical and the discount rate.

CONCLUSIONS

Treatment of PPMS with OCR is more cost effective from a societal than a healthcare perspective, therefore prioritisation of public funding of novel pharmaceuticals for MS and other resource intensive chronic health conditions will depend critically upon the study perspective.

摘要

目的

多发性硬化症(MS)是一种进行性、退行性自身免疫性神经疾病。本研究比较了从社会视角与医疗保健视角来看,在新西兰使用奥瑞珠单抗(OCR)与最佳支持治疗(BSC)治疗原发性进行性MS(PPMS)的成本效益影响。

方法

该分析采用基于十个扩展残疾状态量表(EDSS)状态加死亡情况的终身队列马尔可夫模型。它有两个结构相同的分支,治疗分支中的向前转移概率是通过将对照组中的向前转移概率(转换为发生率,再转换回概率)乘以临床试验ORATORIO中的12周残疾进展风险比来获得的。MS的直接和间接成本根据2017年澳大利亚的一项调查进行估算,并使用购买力平价和新西兰消费者价格指数换算为2024年新西兰元。未来成本和效益按每年3.5%进行贴现。该模型根据PPMS的新西兰死亡率进行校准,当达到轮椅依赖(EDSS 7)时治疗结束。

结果

对于一个从EDSS的ORATORIO分布开始的40岁模拟队列,在标价的50%(即每位患者每年37384新西兰元有50%的折扣)时,从社会视角看,OCR与BSC相比的增量成本效益比(ICER)为每质量调整生命年114427新西兰元(64651美元),从医疗保健视角看为146711新西兰元(82892美元)。从社会视角看,按照财政部的支付意愿(WTP)标准(120200新西兰元),高达标价56%的购置成本(20935新西兰元;44%的折扣)是合理的。从医疗保健支付方视角看,按照财政部隐含的WTP(43313新西兰元),高达标价10%的购置成本(3738新西兰元;90%的折扣)是合理的。基于此指标,从社会视角看,对OCR的投资可比医疗保健视角高出5.6倍是合理的。或者,如果将人均国内生产总值(83011新西兰元)的标准用作社会资金阈值,那么高达标价37.9%的购置成本(即62.1%的折扣或14169新西兰元)可能是合理的。这些结果从社会视角看对BSC成本敏感,但从医疗保健视角看不敏感。从两个视角看,成本效益对OCR的购置成本和疗效、疗效可能的减弱、治疗开始时的年龄和EDSS状态、生物类似药进入市场的成本和时间以及贴现率都很敏感。

结论

从社会视角而非医疗保健视角看,用OCR治疗PPMS更具成本效益,因此,对于MS和其他资源密集型慢性健康状况的新型药物,公共资金的优先分配将严重取决于研究视角。

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