Ocrelizumab治疗新西兰原发性进行性多发性硬化症的成本效益的社会与医疗视角

IF 4.4 3区 医学 Q1 ECONOMICS
Richard J Milne, Carsten Schousboe, Julie A Campbell, John Mottershead
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引用次数: 0

摘要

目的:多发性硬化症(MS)是一种进行性、退行性、自身免疫性神经元疾病。本研究比较了社会和医疗保健角度对新西兰ocrelizumab (OCR)与最佳支持治疗(BSC)治疗原发性进展性MS (PPMS)的成本效益的影响。方法:采用基于十种扩展残疾状态量表(EDSS)状态加上死亡的终身队列马尔可夫模型进行分析。它有两个结构相同的组,治疗组的前向转移概率是通过将对照组的前向转移概率(转换为比率,再转换为概率)乘以临床试验ORATORIO中的12周残疾进展风险比得到的。MS的直接和间接成本是根据2017年澳大利亚的一项调查估算的,并使用购买力平价和新西兰消费者价格指数转换为2024年的新西兰元。未来成本和收益按每年3.5%折现。该模型被校准为PPMS的新西兰死亡率,当达到轮椅依赖(EDSS7)时,治疗结束。结果:对于一个40岁的模型队列,从EDSS的ORATORIO分布开始,按目录价格的50%(即每位患者每年37,384美元的50%回扣),OCR与BSC的增量成本效益比(ICER)从社会角度来看,每个QALY为114,427美元(64,651美元),从医疗保健角度来看,为146,711美元(82,892美元)。从社会角度来看,按照财政部120,200新西兰元的意愿支付(WTP)标准,收购成本高达标价(20,935新西兰元;44%的回扣)是合理的。从医疗保健支付方的角度来看,按照财政部43,313新西兰元的隐含WTP计算,收购成本最高可达标价的10%(3738新西兰元;90%的回扣)是合理的。基于这一指标,从社会角度来看,与医疗保健角度相比,OCR投资高出5.6倍是合理的。或者,如果将人均GDP(83,011新西兰元)作为社会资助门槛,则收购成本最高可达标价的37.9%(即62.1%的回扣或14,169新西兰元)是合理的。从社会角度来看,这些结果对平衡计分卡的成本敏感,但从医疗保健角度来看则不然。从这两个角度来看,成本效益对OCR的获得成本和疗效、疗效的潜在减弱、治疗开始时的年龄和EDSS状态、生物仿制药的成本和进入时间以及贴现率都很敏感。结论:从社会的角度来看,用OCR治疗PPMS比从医疗保健的角度来看更具成本效益,因此,针对MS和其他资源密集型慢性疾病的新药的公共资金优先次序将关键取决于研究的角度。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Societal Versus Healthcare Perspectives on the Cost Effectiveness of Ocrelizumab for Treatment of Primary Progressive Multiple Sclerosis in Aotearoa New Zealand.

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.

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来源期刊
PharmacoEconomics
PharmacoEconomics 医学-药学
CiteScore
8.10
自引率
9.10%
发文量
85
审稿时长
6-12 weeks
期刊介绍: PharmacoEconomics is the benchmark journal for peer-reviewed, authoritative and practical articles on the application of pharmacoeconomics and quality-of-life assessment to optimum drug therapy and health outcomes. An invaluable source of applied pharmacoeconomic original research and educational material for the healthcare decision maker. PharmacoEconomics is dedicated to the clear communication of complex pharmacoeconomic issues related to patient care and drug utilization. PharmacoEconomics offers a range of additional features designed to increase the visibility, readership and educational value of the journal’s content. Each article is accompanied by a Key Points summary, giving a time-efficient overview of the content to a wide readership. Articles may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand the scientific content and overall implications of the article.
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