探讨药物国际多中心临床试验设计的一种新方法SGDDP  

Statistical considerations on a new design method for multiregional clinical trial: SGDDP

作　　者：黄钦[1] 王骏[1] 曲荣华 陈刚 袁志龙 

机构地区：[1]国家食品药品监督管理总局药品审评中心,北京100038 [2]北京师范大学数学科学学院,北京100875 [3]美国强生制药公司

出　　处：《中国新药杂志》2015年第7期721-724,744,共5页Chinese Journal of New Drugs

摘　　要：在新药上市申请中,常常运用多地区开展的国际多中心临床试验(MRCT)数据和总体结果作为获准注册的主要证据。然而,对考察所涉及某个国家或地区的目标人群的药物效应时,却需要考虑种族因素和当地的医疗实践的影响,因此给评价和决策带来极大挑战。2012年,我们提出一种基于加权Z-检验的思路来设计MRCT的新方法,简称SGDDP。该方法结合来自目标种族人群和非目标种族人群(NTE)的信息,通过降低非目标种族人群在加权检验统计量中的信息权重,并严格控制统计假设的I类错误,从而检验评估在目标种族人群(TE)的有效性。本文对该方法进行简要介绍,并以最常用的连续型变量指标为例对研究所需的目标种族(TE)人群样本量进行了相应估算。Due to the potential impact of ethnic factors on clinical outcomes,the global registration of a new treatment is challenging. In 2012,we proposed a new method called SGDDP using weighted Z-tests to design the trial and estimate sample size for simultaneous global drug development. In the proposed method,a weighted Ztest that combines the information collected from both the targeted ethnic（ TE） group and the nontargeted ethnic（ NTE） group is used to test the efficacy of a new treatment for the TE group. The influence of ethnic factors and local medical practice on the treatment effect is accounted for by down-weighting the information collected from the NTE group in the combined test statistic. This design controls rigorously the overall false positive rate at a given level. This article summarized the essential points of the new method and as a example calculated the sample size needed for the TE group in the trial program for most commonly used endpoints of continuous variables.

关 键 词：国际多中心临床试验 药物研发 种族群体 药品监管 加权Z-检验 

分 类 号：R95[医药卫生—药学]