单臂试验药物医保准入评估的方法学思考

冯依曼; 丁锦希; 李伟; 方刚

doi:10.19546/j.issn.1674-3830.2022.8.002

中国医疗保险 ›› 2022, Vol. 0 ›› Issue (8) : 10-16. DOI: 10.19546/j.issn.1674-3830.2022.8.002

深度聚焦

单臂试验药物医保准入评估的方法学思考

冯依曼¹, 丁锦希^1,2, 李伟^1,2, 方刚¹

作者信息 +

Methodological Considerations on Medical Insurance Access Assessment of Drugs Approved Based on Single Arm Trial

Author information +

文章历史 +

摘要

近年来,越来越多创新药物基于单臂试验获批上市。但单臂试验药物的四个特征,即无参照药物、无直接比对数据、测量指标为替代终点以及证据不稳定,给医保准入评估带来较大的难度和不确定性。本文结合域外实践经验与我国药品目录调整情况,为优化单臂试验药物医保准入评估方法提供建议,推进医保准入管理精细化发展。

Abstract

In recent years, more and more innovative drugs have been approved based on single-arm trials. However, the four characteristics of single arm trial drugs, i.e. lack of compared drugs, lack of direct comparison data, surrogate endpoints and unstable evidence, bring great difficulty and uncertainty to the assessment of medical insurance reimbursement decision. In this paper, based on overseas experience and the adjustment of China’s National Reimbursement Drug List, suggestions were provided for optimizing the evaluation method for single arm trial drugs, in order to promote the refined development of reimbursement access management.

导出引用

冯依曼, 丁锦希, 李伟, 方刚. 单臂试验药物医保准入评估的方法学思考[J]. 中国医疗保险. 2022, 0(8): 10-16 https://doi.org/10.19546/j.issn.1674-3830.2022.8.002

Methodological Considerations on Medical Insurance Access Assessment of Drugs Approved Based on Single Arm Trial[J]. China Health Insurance. 2022, 0(8): 10-16 https://doi.org/10.19546/j.issn.1674-3830.2022.8.002

中图分类号： F840.684 C913.7

参考文献

[1] PATEL D, GRIMSON F, MIHAYLOVA E, et al.Use of External Comparators for Health Technology Assessment Submissions Based on Single-Arm Trials[J]. Value Health, 2021, 24(8): 1118-1125.
[2] 杨宏,李皓静,罗丹阳,等.肿瘤免疫治疗超进展的研究和展望[J].中南医学科学杂志, 2022, 50(02): 297-301.
[3] 冯依曼,丁锦希,李佳明,等.嵌合抗原受体T细胞产品卫生技术评估新思路[J].世界临床药物, 2022, 43(04): 377-385.
[4] 中华医学会肿瘤学分会,中华医学会杂志社.中华医学会胃癌临床诊疗指南(2021版)[J].中华医学杂志, 2022, 102(16): 1169-1189.
[5] 丁锦希,李伟. “守正创新,为人民健康谋福祉”谈2021年国家医保目录调整工作的三大亮点[J].世界临床药物, 2022, 43(01): 1-4.
[6] NICE. Eculizumab for treating atypical haemolytic uraemic syndrome[EB/OL].(2015-01-28)[2022-06-22].https://www.nice.org.uk/guidance/hst1/chapter/4-Evidence-submissions
[7] HAS. Progrès thérapeutique important dans la prise en charge du syndrome hémolytique et urémique atypique [EB/OL].(2013-04-03)[2022-06-22].https://www.has-sante.fr/jcms/c_1319245/fr/soliris-eculizumab
[8] 卢利新,朱慧莉,谢斌.国外新药医疗保险准入机制研究与启示[J].中国医院, 2021, 25(12): 43-45.
[9] JANSEN J P, FLEURENCE R, DEVINE B, et al.Interpreting indirect treatment comparisons and network meta-analysis for health-care decision making: report of the ISPOR Task Force on Indirect Treatment Comparisons Good Research Practices: part 1[J]. Value Health, 2011, 14(4): 417-428.
[10] SIGNOROVITCH J E, SIKIRICA V, ERDER M H, et al.Matching-adjusted indirect comparisons: a new tool for timely comparative effectiveness research[J]. Value Health, 2012, 15(6): 940-947.
[11] NICE. Decision Support Unit. Population-adjusted indirect comparisons (MAIC and STC)[EB/OL].(2016-12-07)[2022-04-19]http://nicedsu.org.uk/technical-support-documents/population-adjusted-indirect-comparisons-maic-and-stc.
[12] ISPOR. Indirect comparisons for single-arm trials or trials without common comparator arms[EB/OL].(2015-05-18)[2022-05-13].https://www.ispor.org/docs/default-source/presentations/233.pdf?sfvrsn=750a5ee8_1.
[13] DEL PAGGIO J C, BERRY J S, HOPMAN W M, et al. Evolution of the Randomized Clinical Trial in the Era of Precision Oncology[J]. JAMA Oncology, 2021, 7(5): 728-734.
[14] HASLAM A, HEY S P, GILL J, et al.A systematic review of trial-level meta-analyses measuring the strength of association between surrogate end-points and overall survival in oncology[J]. Eur J Cancer, 2019, 106: 196-211.
[15] BULLEMENT A, LATIMER N R, BELL GORROD H.Survival Extrapolation in Cancer Immunotherapy: A Validation-Based Case Study[J]. Value Health, 2019, 22(3): 276-283.
[16] VITALE C, STRATI P.CAR T-Cell Therapy for B-Cell non-Hodgkin Lymphoma and Chronic Lymphocytic Leukemia: Clinical Trials and Real-World Experiences[J]. Front Oncol, 2020, 10: 849.
[17] 徐赫,马爱霞.药物经济学中肿瘤免疫疗法生存曲线模拟方法探索[J].中国卫生经济, 2020, 39(10): 58-61.
[18] GRIFFITHS E A, MACAULAY R, VADLAMUDI N K, et al.The Role of Noncomparative Evidence in Health Technology Assessment Decisions[J]. Value Health, 2017, 20(10): 1245-1251.
[19] EVANS S R.Clinical trial structures[J]. J Exp Stroke Transl Med, 2010, 3(1): 8-18.
[20] TANG H, FOSTER N R, GROTHEY A, et al.Comparison of error rates in single-arm versus randomized phase II cancer clinical trials[J]. J Clin Oncol, 2010, 28(11): 1936-1941.
[21] 杨莉,韦彦伊,吴迪,等.美国和欧盟基于替代终点的新药上市审批程序研究及启示[J].中国新药杂志, 2022, 31(02): 132-141.
[22] 谭重庆,彭六保,曾小慧,等.药物经济学评价中敏感度分析的参数分布[J].中国新药与临床杂志, 2015, 34(12): 961-964.
[23] MCCULLAGH L, SCHMITZ S, BARRY M, et al.Examining the Feasibility and Utility of Estimating Partial Expected Value of Perfect Information (via a Nonparametric Approach) as Part of the Reimbursement Decision-Making Process in Ireland: Application to Drugs for Cancer[J]. Pharmacoeconomics, 2017, 35(11): 1177-1185.
[24] Ellis K.Cost-effectiveness of Chimeric Antigen Receptor T-cell Therapy for Treating Large B-cell Lymphoma Patients in Canada[D]. University of Waterloo, 2020.