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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。
In new drug listing applications, multi-region international multicentre clinical trial (MRCT) data and overall results are often used as the main evidence for registration approval. However, when examining the drug effects of target populations in a country or region, the impact of racial factors and local medical practices needs to be considered, posing a great challenge to evaluation and decision-making. In 2012, we proposed a new method to design MRCT based on the weighted Z-test, referred to as SGDDP for short. The method combines information from target ethnic groups and non-target ethnic groups (NTEs) with the goal of reducing the information weight of non-target ethnic groups in weighted test statistics and strictly controlling Type I errors in statistical assumptions, The effectiveness of the crowd (TE). This article gives a brief introduction of this method, and uses the most commonly used continuous variable indicator as an example to estimate the sample size of target population (TE) needed by the research.