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日本HER2阳性转移性乳腺癌患者一线帕妥珠单抗治疗的经济学评估

Economic Evaluation of First-Line Pertuzumab Therapy in Patients with HER2-Positive Metastatic Breast Cancer in Japan.

作者信息

Moriwaki Kensuke, Uechi Saki, Fujiwara Takaaki, Hagino Yu, Shimozuma Kojiro

机构信息

Comprehensive Unit for Health Economic Evidence Review and Decision Support (CHEERS), Research Organization of Science and Technology, Ritsumeikan University, #209, Research Park Bid. No. 2, 134, Minami-machi, Chudoji, Simogyo-ku, Kyoto, 600-8813, Japan.

Laboratory of Medical Statistics, Kobe Pharmaceutical University, 4-19-1, Motoyamakita-machi, Higashinada-ku, Hyogo, 658-8558, Japan.

出版信息

Pharmacoecon Open. 2021 Sep;5(3):437-447. doi: 10.1007/s41669-020-00254-3. Epub 2021 Jan 23.

Abstract

OBJECTIVE

The purpose of this analysis was to evaluate the cost effectiveness of the combination of pertuzumab, trastuzumab, and docetaxel (PTD) for the treatment of patients with human epidermal growth factor receptor-2 (HER2)-positive breast cancer in Japan.

METHODS

A partitioned survival analysis model was developed to predict costs and quality-adjusted life-years (QALYs) in a PTD arm and a trastuzumab plus docetaxel (TD) arm. Direct medical costs were considered from the perspective of the Japanese healthcare system. The time horizon of the model was set to 20 years. Data on overall survival and progression-free survival were derived from the CLEOPATRA trial. Cost parameters were estimated using a real-world claims database. Utilities were derived from published sources outside Japan. The incremental cost-effectiveness ratio (ICER) of PTD therapy compared with TD therapy was estimated. Sensitivity analysis was conducted to assess the uncertainty in parameter settings.

RESULTS

Compared with TD therapy, PTD therapy incurred an additional cost of $US174,479 and conferred an additional 0.949 QALYs. This resulted in an ICER of $US183,901 per QALY gained. Utility weights for progression-free survival and progressed disease had a relatively large impact on the base-case result, but the ICERs remained higher than $US75,000 per QALY over the full range of model parameters. Based on a probabilistic sensitivity analysis, the probability that PTD is cost effective was estimated to be 3.3%.

CONCLUSIONS

Applying a willingness-to-pay threshold of $US75,000 per QALY, PTD therapy as first-line therapy would not be cost effective. Further research is required on utilities and clinical benefits for Japanese patients with breast cancer.

摘要

目的

本分析旨在评估帕妥珠单抗、曲妥珠单抗和多西他赛联合用药(PTD)治疗日本人类表皮生长因子受体2(HER2)阳性乳腺癌患者的成本效益。

方法

建立了一个分区生存分析模型,以预测PTD组和曲妥珠单抗联合多西他赛(TD)组的成本和质量调整生命年(QALY)。从日本医疗保健系统的角度考虑直接医疗成本。模型的时间范围设定为20年。总生存和无进展生存数据来自CLEOPATRA试验。成本参数使用真实世界的索赔数据库进行估计。效用值来自日本以外的已发表资料。估计了PTD治疗与TD治疗相比的增量成本效益比(ICER)。进行敏感性分析以评估参数设置的不确定性。

结果

与TD治疗相比,PTD治疗额外产生了174,479美元的成本,并带来了额外的0.949个QALY。这导致每获得一个QALY的ICER为183,901美元。无进展生存和疾病进展的效用权重对基础病例结果有相对较大的影响,但在整个模型参数范围内,ICER仍高于每QALY 75,000美元。基于概率敏感性分析,估计PTD具有成本效益的概率为3.3%。

结论

采用每QALY 75,000美元的支付意愿阈值,PTD治疗作为一线治疗不具有成本效益。需要对日本乳腺癌患者的效用值和临床益处进行进一步研究。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/24ef/8333148/af01f443fcd8/41669_2020_254_Fig1_HTML.jpg

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