Reproducibility probability in clinical trials
Reproducibility probability in clinical trials
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DOI：
10.1002/sim.1177
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发表时间：
2002-06-30
影响因子：
2
通讯作者：
Chow, SC
中科院分区：
文献类型：
--
作者：
Shao, J;Chow, SC
对于新药产品的上市批准，美国食品和药物管理局(FDA)要求通过进行至少两项充分和良好控制的临床试验来提供该药物产品有效性的实质性证据。进行第二次临床试验的目的是研究第一次试验的临床结果在相同研究方案的第二次试验中是否可重现。在某些情况下，1997年的FDA现代化法案包括一项条款，允许来自一项充分和受控良好的临床试验调查和确证证据的数据，以确定对待批准的药物和生物候选进行风险/益处评估的有效性。在本文中，我们引入了给定临床试验的可重复性概率的概念，它有助于为监管机构决定单个临床试验是否足够以及制药公司在未来的临床试验中调整样本量提供重要信息。在临床试验中常用的几种研究设计下，研究了三种方法，即估计功率法、置信限方法和贝叶斯方法来评估重复性概率。版权所有(C)2002 John Wiley Sons，Ltd.
For marketing approval of a new drug product, the United States Food and Drug Administration (FDA) requires that substantial evidence of the effectiveness of the drug product be provided through the conduct of at least two adequate and well-controlled clinical trials. The purpose of conducting the second clinical trial is to study whether the clinical result from the first trial is reproducible in the second trial with the same study protocol. Under certain circumstance, the FDA Modernization Act of 1997 includes a provision to allow data from one adequate and well-controlled clinical trial investigation and confirmatory evidence to establish effectiveness for risk/benefit assessment of drug and biological candidates for approval. In this paper, we introduce the concept of reproducibility probability for a given clinical trial, which is useful in providing important information for regulatory agencies in deciding whether a single clinical trial is sufficient and for pharmaceutical companies in adjusting the sample size in a future clinical trial. Three approaches, the estimated power approach, the method of confidence bounds and the Bayesian approach, are studied in evaluating reproducibility probabilities under several study designs commonly used in clinical trials. Copyright (C) 2002 John Wiley Sons, Ltd.