中国新药杂志

帕博利珠单抗一线治疗局部晚期/转移性非小细胞肺癌的快速卫生技术评估

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刘一 ¹ , 黄琳 ¹ , 门鹏 ²^,³ , 杨毅恒 ²^,³^,⁴ , 封宇飞 ¹ , 樊榕榕 ¹ , 刘彦国 ¹

中国新药杂志 | 药物安全与合理应用 2021,30(12): 1137-1146

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中国新药杂志 |药物安全与合理应用 2021 , 30 (12) : 1137 -1146

帕博利珠单抗一线治疗局部晚期/转移性非小细胞肺癌的快速卫生技术评估

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刘一¹, 黄琳¹, 门鹏²^,³, 杨毅恒²^,³^,⁴, 封宇飞¹, 樊榕榕¹, 刘彦国¹

作者信息

¹北京大学人民医院，北京　100044

²北京大学第三医院药剂科，北京　100191

³北京大学医学部药物评价中心，北京　100191

⁴北京市药学质量控制和改进中心，北京　100191

通讯作者:

封宇飞，男，主任药师，硕士生导师，主要从事药物经济学及医院药学研究。联系电话：（010）88325751，E-mail：fengyufei@126.com。

作者简介:

刘一，女，副主任药师，主要从事临床药理学及药物分析学研究。联系电话：（010）88325749，E-mail：lyi1267@126.com。

A rapid health technology assessment of pembrolizumab in first-line therapy for locally advanced/metastatic non-small cell lung cancer

Yi LIU¹, Lin HUANG¹, Peng MEN²^,³, Yi-heng YANG²^,³^,⁴, Yu-fei FENG¹, Rong-rong FAN¹, Yan-guo LIU¹

Affiliations

¹Peking University People's Hospital, Beijing 100044, China

²Department of Pharmacy, Peking University Third Hospital, Beijing 100191, China

³Institute for Drug Evaluation, Peking University Health Science Center, Beijing 100191, China

⁴Beijing Pharmacy Center for Quality Control and Improvement, Beijing 100191, China

出版时间: 2021-06-30

文章导航

摘要

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目的：

评价帕博利珠单抗（pembrolizumab, PEM）一线治疗局部晚期/转移性非小细胞肺癌（non-small cell lung cancer, NSCLC）的有效性、安全性和经济性。

方法：

系统检索中英文数据库和卫生技术评估（HTA）相关的网站及数据库。由2位评价者根据入选与排除标准独立筛选文献、提取资料和评价质量后，对有效性、安全性和经济性进行综合分析。

结果：

最终纳入HTA报告3篇，系统评价/Meta分析6篇，药物经济学研究14篇。有效性方面，与化学治疗（chemotherapy，CT）相比，PEM单药治疗可提高（指有统计学意义，下同）程序性死亡因子配体-1（PD-L1）≥50%的突变阴性的局部晚期/转移性NSCLC患者的总生存期（OS），但未提高PD-L1 1%~49%患者的OS；PEM+CT可提高突变阴性的局部晚期/转移性NSCLC患者（无论PD-L1表达水平）的OS及无进展生存期（PFS）、突变阴性的转移性非鳞状NSCLC患者的OS及PFS、转移性鳞状NSLCL患者的PFS。与纳武利尤单抗（nivolumab，NIV）相比，PEM+CT可提高PD-L1≥50%的突变阴性的局部晚期/转移性NSCLC患者的OS及PFS、突变阴性的转移性非鳞状NSCLC患者的OS及PFS，但未提高转移性鳞状NSLCL患者的OS和PFS。与NIV+CT相比，PEM+CT可提高突变阴性的转移性非鳞状NSCLC患者的OS。与贝伐珠单抗（bevacizumab，BEV）相比，PEM+CT可提高突变阴性的局部晚期/转移性NSCLC患者的OS及突变阴性的转移性非鳞状NSCLC患者的OS。安全性方面，对于突变阴性的局部晚期/转移性NSCLC患者，与CT相比，PEM可降低1~5级治疗相关不良反应、3~5级治疗相关不良反应及严重不良反应；PEM+CT在1~5级治疗相关不良反应、3~5级治疗相关不良反应及严重不良反应的发生情况相当；与NIV相比，PEM+CT可增加严重不良反应；与NIV+CT相比，PEM+CT严重不良反应的发生情况相当。经济性方面，与CT相比，对于PD-L1≥1%的突变阴性的局部晚期/转移性NSCLC患者，PEM单药在中国不具有经济性；对突变阴性的转移性非鳞状NSCLC患者，PEM+CT在中国也不具有经济性。

结论：

PEM一线治疗NSCLC具有良好的有效性和安全性，但以研究中的价格分析，在我国的成本效果尚不佳，进一步降低其价格可有助于提升其经济性。

关键词

帕博利珠单抗 / 非小细胞肺癌 / 免疫检查点抑制剂 / 卫生技术评估

Abstract

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Objective:

To evaluate the effectiveness, safety and economy of pembrolizumab (PEM) for locally advanced/metastatic NSCLC.

Methods:

Chinese and English databases and HTA organization websites were systematically searched. Two reviewers independently identified studies, extracted data and assessed the quality of included studies. Qualitative analysis was performed from the perspectives of efficacy, safety and economy.

Results:

Three HTA reports, six systematic reviews and 14 pharmacoeconomic studies were included. In terms of effectiveness, compared with chemotherapy (CT), PEM alone could improve the overall survival (OS) of patients of locally advanced or metastatic NSCLC with PD-L1 50%, but not that of patients with PD-L1 1%~49%. PEM+CT could improve (with statistical significance, the same below) the OS and progressive-free survival (PFS) of locally advanced or metastatic NSCLC patients (regardless of PD-L1 expression level) and metastatic non squamous NSCLC patients; it could improve the PFS of metastatic squamous NSCLC patients. Compared with NIV, PEM+CT could improve the OS and PFS of PD-L1 50% of advanced or metastatic NSCLC patients and metastatic non squamous NSCLC patients, but it did not improve the OS and PFS of metastatic squamous NSCLC patients. Compared with NIV+CT, PEM+CT could improve the OS of patients with mutation negative metastatic NSCLC. Compared with BEV, PEM+CT can improve the OS of advanced or metastatic NSCLC patients with negative mutation and nonsquamous NSCLC patients with negative mutation. In terms of safety, compared with CT, PEM could reduce grade 1~5 treatment-related side effects, grade 3~5 treatment-related side effects and serious side effects in patients with advanced or metastatic NSCLC with negative mutations. Compared with CT, the incidence of grade 1~5 treatment-related side effects, grade 3~5 treatment-related side effects and serious side effects were comparable. Compared with NIV, PEM+CT increased severe side effects. The severe side effects of PEM+CT were comparable to those of NIV+CT. In terms of economy, compared with CT, for mutation-negative locally advanced or metastatic NSCLC patients with PD-L1 1%, PEM alone is not cost-effective in China. For mutation-negative metastatic non-squamous NSCLC patients, PEM+CT is also not cost-effective in China.

Conclusion:

PEM has favorable efficacy and safety for the first-line treatment of NSCLC, However, according to its price analyzed in the study, it is not cost-effective in China currently and further price reducing may help improve its cost-effectiveness.

Key words

pembrolizumab / non-small cell lung cancer / immune checkpoint inhibitors / health technology assessment

引用本文

刘一, 黄琳, 门鹏, 杨毅恒, 封宇飞, 樊榕榕, 刘彦国. 帕博利珠单抗一线治疗局部晚期/转移性非小细胞肺癌的快速卫生技术评估. 中国新药杂志, 2021 , 30 (12) : 1137 -1146 .

Yi LIU, Lin HUANG, Peng MEN, Yi-heng YANG, Yu-fei FENG, Rong-rong FAN, Yan-guo LIU. A rapid health technology assessment of pembrolizumab in first-line therapy for locally advanced/metastatic non-small cell lung cancer[J]. Chinese Journal of New Drugs, 2021 , 30 (12) : 1137 -1146 .

基金

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北京市卫健委药械处快速卫生技术评估与医院新药遴选专项：白求恩·求索—药学科研能力建设项目(B-19-H-20200622)

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2021年第30卷第12期

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首发时间：2026-10-28
出版时间：2021-06-30

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录用日期：2020-09-14

基金

北京市卫健委药械处快速卫生技术评估与医院新药遴选专项：白求恩·求索—药学科研能力建设项目(B-19-H-20200622)

作者信息

¹北京大学人民医院，北京　100044

²北京大学第三医院药剂科，北京　100191

³北京大学医学部药物评价中心，北京　100191

⁴北京市药学质量控制和改进中心，北京　100191

通讯作者:

封宇飞，男，主任药师，硕士生导师，主要从事药物经济学及医院药学研究。联系电话：（010）88325751，E-mail：fengyufei@126.com。

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2种不同金属材料的力学参数

科 Family	属数 Number of genus	种数 Number of species	占总种数比例 Percentage of total species (%)	属 Genus	种数 Number of species	占总种数比例 Percentage of total species (%)
鹅膏菌科Amanitaceae	2	11	5.26	鹅膏菌属 Amanita	10	4.78
小菇科 Mycenaceae	2	12	5.74	丝盖伞属 Inocybe	5	2.39
多孔菌科 Polyporaceae	8	14	6.70	蜡蘑属 Laccaria	5	2.39
红菇科 Russulaceae	3	23	11.00	小皮伞属 Marasmius	6	2.87
				小菇属 Mycena	11	5.26
				光柄菇属 Pluteus	5	2.39
				红菇属 Russula	17	8.13
				栓菌属 Trametes	5	2.39

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