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荷兰一种用于治疗晚期中肠神经内分泌肿瘤的孤儿药镥[177Lu]奥曲肽对比奥曲肽长效释放制剂的经济性评价。

Economic evaluation of orphan drug Lutetium-Octreotate vs. Octreotide long-acting release for patients with an advanced midgut neuroendocrine tumour in the Netherlands.

机构信息

Departments of Economics, Econometrics and Finance, Faculty of Economics and Business, University of Groningen, Groningen, The Netherlands.

Coronel Institute of Occupational Health and Research Center for Insurance Medicine, Amsterdam UMC, University of Amsterdam, Amsterdam Public Health Research Institute, Amsterdam, The Netherlands.

出版信息

Eur J Health Econ. 2021 Aug;22(6):991-999. doi: 10.1007/s10198-021-01303-2. Epub 2021 Apr 7.

Abstract

OBJECTIVES

Multiple studies showed positive effects of Lutetium-Octreotate (LO) treatment in neuroendocrine tumours. LO has been used in the Netherlands since the 1980s and recently received the orphan status shortly after the acquisition by Novartis. Since then, the official list price has increased sixfold. From a value-based pricing perspective, we analysed the impact of the increase in price on the incremental cost-effectiveness ratio (ICER) of LO treatment compared to optimal best supportive care, a high dose of Octreotide long-acting release (O-LAR), using the clinical data of the NETTER-1 trial.

METHODS

A Markov model was developed to evaluate the costs per quality-adjusted life-year (QALY) for LO treatment compared to O-LAR from the healthcare perspective. A scenario analysis was conducted to compare the cost-effectiveness with the initial and increased price level of the LO-treatment.

RESULTS

At the increased price level, the cost-effectiveness analysis rendered a deterministic ICER of €53,500 per QALY, while at the initial pricing, the ICER was €19,000 per QALY. The probabilistic sensitivity analysis (PSA) showed that LO had a high probability of being cost-effective at both the increased and initial price level, considering a cost-effectiveness threshold of €80,000.

CONCLUSIONS

Even at the increased price level, LO treatment can still be considered cost-effective using the applicable Dutch willingness-to-pay threshold of 80,000 euro per QALY. Considering the public scrutiny in relation to this price increase, these outcomes raise the question whether traditional cost-effectiveness methods are sufficient in fully capturing the societal acceptance of prices of new medicines.

摘要

目的

多项研究表明镥[177Lu]奥曲肽(LO)治疗神经内分泌肿瘤具有积极作用。LO 自 20 世纪 80 年代在荷兰使用,最近在被诺华收购后不久获得了孤儿药地位。此后,官方定价上涨了六倍。从基于价值的定价角度出发,我们使用 NETTER-1 试验的临床数据,分析价格上涨对 LO 治疗相对于最佳支持治疗(大剂量奥曲肽长效释放[O-LAR])的增量成本效果比(ICER)的影响。

方法

从医疗保健角度出发,我们开发了一个马尔可夫模型,以评估 LO 治疗相对于 O-LAR 的每质量调整生命年(QALY)的成本。进行了情景分析,以比较 LO 治疗的初始和涨价水平的成本效果。

结果

在涨价水平下,成本效果分析得出 LO 治疗的确定性 ICER 为每 QALY 53500 欧元,而在初始定价下,ICER 为每 QALY 19000 欧元。概率敏感性分析(PSA)表明,考虑到 80000 欧元的成本效果阈值,在涨价和初始定价水平下,LO 具有很高的成本效果可能性。

结论

即使在涨价水平下,LO 治疗仍可被认为具有成本效果,因为荷兰适用的每 QALY 80000 欧元的意愿支付阈值。考虑到对这一价格上涨的公众关注,这些结果提出了一个问题,即传统的成本效果方法是否足以充分捕捉新药物价格的社会接受度。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9b84/8275500/90c5d430f726/10198_2021_1303_Fig1_HTML.jpg

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