注射用司妥昔单抗治疗中国特发性多中心Castleman病的成本效用分析

doi:10.5246/jcps.2023.10.068

中国药学(英文版) ›› 2023, Vol. 32 ›› Issue (10): 842-851.DOI: 10.5246/jcps.2023.10.068

• 【药事管理与临床药学专栏】 • 上一篇下一篇

注射用司妥昔单抗治疗中国特发性多中心Castleman病的成本效用分析

马俊¹, 倪良玉², 朱奇云³, 杨照⁴^,^*(), 江滨⁵^,^*()

1. 北京大学第三医院, 北京 100191
2. 北京大学医学部, 北京 100191
3. 加州大学圣地亚哥分校, 美国圣地亚哥 92093
4. 北京大学第一医院, 北京 100034
5. 北京大学医学部药学院药事管理与临床药学系, 北京 100191

收稿日期:2023-05-12 修回日期:2023-06-01 接受日期:2023-06-14 出版日期:2023-11-04 发布日期:2023-11-04
通讯作者: 杨照, 江滨
作者简介:
+ Tel.: +86-13601100121, E-mail: binjiang@bjmu.edu.cn
+ Tel.: +86-15210596561, E-mail: 15210596561@163.com
基金资助:
Capital’s Funds for Health Improvement and Research (Grant No. 2022-2-4079).

Cost-utility of siltuximab injection for idiopathic multicentric Castleman disease (iMCD) in China

Jun Ma¹, Liangyu Ni², Qiyun Zhu³, Zhao Yang⁴^,^*(), Bin Jiang⁵^,^*()

1 Peking University Third Hospital, Beijing 100191, China
2 Peking University Health Science Center, Beijing 100191, China
3 University of California San Diego, San Diego 92093, USA
4 Peking University First Hospital, Beijing 100034, China
5 Department of Administrative and Clinical Pharmacy, Peking University School of Pharmaceutical Sciences, Beijing 100191, China

Received:2023-05-12 Revised:2023-06-01 Accepted:2023-06-14 Online:2023-11-04 Published:2023-11-04
Contact: Zhao Yang, Bin Jiang

摘要/Abstract

摘要：

司妥昔单抗是治疗罕见病特发性多中心Castleman病的创新药物, 临床试验证明其具有确切的疗效和良好的安全性, 但目前对于司妥昔单抗的经济学评价研究有限。本研究旨在基于现有临床资料以及公开发表数据, 采用分区生存模型的分析方法, 从医疗卫生体系的角度出发, 评估司妥昔单抗组和安慰剂组终生治疗中国iMCD患者的平均治疗成本和健康产出, 并进行价格阈值分析、增量分析结果与敏感性分析。研究发现司妥昔单抗组相比安慰剂组可增加6.31 LYs和5.09 QALYs。当WTP为24 2928元/QALY时, 司妥昔单抗组相比安慰剂组具有经济性的最大价格阈值为8921.47元/瓶(400 mg)。因此, 司妥昔单抗作为iMCD的一线治疗方式, 相比安慰剂组, 健康产出获益更高。

关键词: 司妥昔单抗, 特发性多中心Castleman病, 成本效用, 分区生存模型

Abstract:

Siltuximab is an innovative drug used to treat idiopathic multicentric Castleman disease (iMCD), a rare condition. Clinical trials have demonstrated its efficacy and safety. However, there is limited research on the economic evaluation of siltuximab. Therefore, using a partition survival model, we aimed to evaluate the average treatment cost and health outcomes for Chinese patients with iMCD in the siltuximab group compared to the placebo group based on existing clinical and published data. We also conducted price threshold analysis, incremental cost-utility analysis, and sensitivity analysis. The results revealed that compared to the placebo group, the siltuximab group showed an increase of 6.31 life-years (LYs) and 5.09 quality-adjusted life-years (QALYs). At a willingness-to-pay (WTP) threshold of 242 928 yuan/QALY, the maximum price threshold for siltuximab compared to the placebo group was 8921.47 yuan per bottle (400 mg). Therefore, as a first-line treatment for iMCD, siltuximab demonstrated superior health outcomes compared to placebo.

Key words: Siltuximab, Idiopathic multicentric Castleman disease, Cost-utility, Partitioned survival model

Supporting:

马俊, 倪良玉, 朱奇云, 杨照, 江滨. 注射用司妥昔单抗治疗中国特发性多中心Castleman病的成本效用分析[J]. 中国药学(英文版), 2023, 32(10): 842-851.

Jun Ma, Liangyu Ni, Qiyun Zhu, Zhao Yang, Bin Jiang. Cost-utility of siltuximab injection for idiopathic multicentric Castleman disease (iMCD) in China[J]. Journal of Chinese Pharmaceutical Sciences, 2023, 32(10): 842-851.

图/表 10

Figure 1. Schematic presentation of partition survival model.

Table 1. AIC and BIC of standard parameter model fitting K-M curve.

Table 2. Health status utility.

Figure 2. Simulation results of survival analysis of siltuximab and placebo groups.

Table 3. Health outcome analysis.

Table 4. Health outcome analysis.

Table 5. Incremental cost-utility analysis results.

Figure 3. Univariate deterministic sensitivity analysis.

Figure 4. Cost-effectiveness scatterplot.

Figure 5. Incremental cost-effectiveness scatterplot.

参考文献 17

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注射用司妥昔单抗治疗中国特发性多中心Castleman病的成本效用分析

Cost-utility of siltuximab injection for idiopathic multicentric Castleman disease (iMCD) in China

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