New Developments in Medical Clinical Trials
New Developments in Medical Clinical Trials
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医学临床试验新进展
DOI：
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发表时间：
2004
期刊：
影响因子：
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通讯作者：
J. Massaro
中科院分区：
文献类型：
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作者：
R. D'Agostino;J. Massaro
本文回顾了几个新的发展和长期的良好做法进行临床试验。讨论从明确研究目标的必要性开始，继续澄清目标和样本人群，并详细说明主要变量与次要变量，以及在存在多个结局的情况下需要进行α调整。在这里，我们还审查了替代终点的问题。接下来是研究设计问题，包括盲法、随机化和多中心研究。然后，我们讨论了目前的趋势，安慰剂对照试验的活性对照非劣效性试验，越来越多地使用独立的数据监测委员会，意向治疗样本的分析的突出性，以及缺失数据的插补的重要性。最后，我们简要讨论了分析单位，新的统计分析方法的作用，安全性问题，子集分析，最重要的是，临床意义。
This paper reviews several new developments and long-standing good practices for conducting clinical trials. Discussion starts with the need for clear statements of study objectives, proceeds to clarify target and sample population, and elaborates on primary vs. secondary variables with the need for alpha adjustment in the presence of multiple outcomes. Here we also review the issue of surrogate endpoints. Study design issues—including blinding, randomization, and multicenter studies—come next. Then we discuss the current trend of the replacement of placebo-controlled trials by active controlled non-inferiority trials, the increasing use of Independent Data Monitoring Committees, the prominence of analysis on Intention-to-Treat samples, and the importance of imputation of missing data. We close with a brief discussion of the unit of analysis, the role of newer statistical analysis methods, safety issues, subset analysis, and, most importantly, clinical significance.