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肿瘤不可知疗法的经济学评价:拉罗替尼治疗 TRK 融合阳性癌症的荷兰经济价值。

Economic Evaluation of a Tumour-Agnostic Therapy: Dutch Economic Value of Larotrectinib in TRK Fusion-Positive Cancers.

机构信息

IQVIA, Real World Solutions, Herikerbergweg 314, 1101 CT, Amsterdam, The Netherlands.

HEOR Value Hub, Med-I-Mart BVBA/SPRL, Helshovenstraat 23, 3840, Hoepertingen, Belgium.

出版信息

Appl Health Econ Health Policy. 2022 Sep;20(5):717-729. doi: 10.1007/s40258-022-00740-1. Epub 2022 Jul 18.

Abstract

BACKGROUND

Larotrectinib is the first tumour-agnostic therapy that has been approved by the European Medicines Agency. Tumour-agnostic therapies are indicated for a multitude of tumour types. The economic models supporting reimbursement submissions of tumour-agnostic therapies are complex because of the multitude of indications per model.

OBJECTIVE

The objective of this paper was to evaluate the cost effectiveness of larotrectinib compared with standard of care in patients with cancer with tropomyosin receptor kinase fusion-positive tumour types in the Netherlands.

METHODS

A previously constructed cost-effectiveness model with a partitioned survival approach was adapted to the Dutch setting, simulating costs and effects of treatment in patients with tropomyosin receptor kinase fusion-positive cancer. The cost-effectiveness model conducts a naïve comparison of larotrectinib to a weighted comparator standard-of-care arm. Dutch specific resource use and costs were implemented and inflated to reflect 2019 euros. The analysis includes a lifetime horizon and a societal perspective.

RESULTS

Larotrectinib versus Dutch standard of care resulted in 5.61 incremental (QALYs) and €232,260 incremental costs, leading to an incremental cost-effectivenes ratio of €41,424/QALY. The probabilistic sensitivity analysis reveals a 88% chance of larotrectinib being cost effective compared with the pooled comparator standard-of-care arm at the applicable €80,000/QALY willingness-to-pay threshold in the Netherlands.

CONCLUSIONS

The incremental cost-effectivenes ratio was well below the applicable threshold for diseases with a high burden of disease in the Netherlands (€80,000). At this threshold, larotrectinib was estimated to be a cost-effective treatment for patients with tropomyosin receptor kinase fusion-positive cancer compared with current standard of care in the Netherlands.

摘要

背景

拉罗替尼是第一种获得欧洲药品管理局批准的肿瘤不可知论治疗药物。肿瘤不可知论疗法适用于多种肿瘤类型。由于每个模型的适应症繁多,支持肿瘤不可知论疗法报销申请的经济模型非常复杂。

目的

本文旨在评估拉罗替尼在荷兰具有原肌球蛋白受体激酶融合阳性肿瘤类型的癌症患者中的成本效益,与标准护理相比。

方法

采用已构建的分割生存方法成本效益模型,适用于荷兰的情况,模拟原肌球蛋白受体激酶融合阳性癌症患者的治疗成本和效果。成本效益模型对拉罗替尼与加权对照标准护理臂进行了简单比较。实施了荷兰特定的资源利用和成本,并将其膨胀以反映 2019 年欧元。分析包括终生和社会视角。

结果

拉罗替尼与荷兰标准护理相比,增加了 5.61 个增量(QALYs)和 232260 欧元增量成本,导致增量成本效益比为 41424 欧元/QALY。概率敏感性分析表明,与适用的 80000 欧元/QALY 荷兰意愿支付阈值相比,拉罗替尼与 pooled comparator standard-of-care 臂相比具有 88%的成本效益可能性。

结论

增量成本效益比远低于荷兰高疾病负担疾病的适用阈值(80000 欧元)。在这个阈值下,与荷兰目前的标准护理相比,拉罗替尼估计对具有原肌球蛋白受体激酶融合阳性癌症的患者是一种具有成本效益的治疗方法。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1921/9385762/69e74e84c627/40258_2022_740_Fig1_HTML.jpg

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