多中心临床试验疗效一致性评价方法

中国临床药理学与治疗学 ›› 2011, Vol. 16 ›› Issue (10): 1126-1131.

多中心临床试验疗效一致性评价方法

王菲¹, 谢海棠², 江波¹, 言方荣^1,3

¹中国药科大学基础部数学教研室,南京 210009,江苏;
²皖南医学院弋矶山医院安徽省药物临床评价中心,芜湖 241001,安徽;
³中国药科大学天然药物活性物质与功能国家重点实验室,南京 210009,江苏

收稿日期:2011-06-14 修回日期:2011-09-06 出版日期:2011-10-26 发布日期:2011-11-02
通讯作者: 言方荣,男,博士,讲师,研究方向:生物统计与群体药物代谢动力学。Tel: 13805170446, E-mail: f.r.yan@163.com
作者简介:王菲,女,硕士,讲师,研究方向:生物统计。Tel: 13347806876, E-mail: blue6923@163.com
基金资助:
国家自然科学基金项目(11171065)

Consistency efficacy assessment of multi-regional clinical trials

WANG Fei¹, XIE Hai-tang², JIANG Bo¹, YAN Fang-rong^1,3

¹Department of Mathematics, China Pharmaceutical University, Nanjing 210009, Jiangsu,China;
²Anhui Provincial Centre for Drug Clinical Evaluation, Affiliated Yijishan Hospital of Wannan Medical College, Wuhu 241001, Anhui,China;
³State Key Laboratory of Natural Medicines, China Pharmaceutical University, Nanjing 210009, Jiangsu,China

Received:2011-06-14 Revised:2011-09-06 Online:2011-10-26 Published:2011-11-02

摘要/Abstract

摘要： 随着临床试验全球化的增长趋势,多中心临床试验已经受到了广泛的关注。其中各个中心治疗效果的一致性评价是多中心临床试验中的一个关键问题。本文系统综述了关于多中心临床试验在评价整体一致性中的常用方法,对一致性评价方法的选择、检验的功效和假阳性的概率以及样本容量与方法之间的关系给出了一些简单的讨论,旨在为从事多中心临床工作者提供借鉴,希望有更多的国内学者关注这一领域的研究,推动多中心临床试验在我国的发展,为新药临床试验提供理论依据。

关键词: 多中心临床试验, 一致性, 异质性, 定量, 定性

Abstract: The multi-regional clinical trials (MRCTs) have received wide attention with an increasing trend of globalization of clinical trials. A key issue in MRCTs is how to assess the consistency of treatment effect across regions. In this paper, we provide a systematic review of methods that be commonly used for assessing the consistency in MRCTs. We also give some simple discussion about the choice of methods of assessing the consistency of treatment effect across regions, such as the power of the test, the false-positive rate and the relationship between the type I error and the sample size. The purpose of this paper is to provide some reference for the members in designing and conducting MRCTs and offer some theory evidences of clinical trails for new drug development. We hope that more researchers can concern this study in this field, promoting the development of MRCTs in our country.

Key words: Multi-regional clinical trials (MRCTs), Consistency, Heterogeneity, Quantitative, Qualitative

中图分类号:

R969.4

王菲, 谢海棠, 江波, 言方荣. 多中心临床试验疗效一致性评价方法[J]. 中国临床药理学与治疗学, 2011, 16(10): 1126-1131.

WANG Fei, XIE Hai-tang, JIANG Bo, YAN Fang-rong. Consistency efficacy assessment of multi-regional clinical trials[J]. Chinese Journal of Clinical Pharmacology and Therapeutics, 2011, 16(10): 1126-1131.

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