Statistical advances in clinical trials and clinical research.
Statistical advances in clinical trials and clinical research.
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DOI：
10.1016/j.trci.2018.04.006
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发表时间：
2018
期刊：
影响因子：
--
通讯作者：
Cummings JL
中科院分区：
文献类型：
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作者：
Shan G;Banks S;Miller JB;Ritter A;Bernick C;Lombardo J;Cummings JL
神经退行性疾病迫切需要新的治疗方法，而临床试验方法是新药开发的重要组成部分。虽然神经系统疾病临床试验的平行组研究设计通常用于测试与安慰剂相比的新治疗方法的有效性，但它不能有效地利用正在进行的研究的信息来提高试验的成功率，或者在新治疗确实无效时提前停止试验。我们回顾了临床试验设计的一些最新进展，包括无效设计和适应性设计。无效设计和非劣效性设计分别用于检验一种新疗法的非优性和非劣效性。我们提供了一些使用这两种设计和正确分析这些研究数据的指导。自适应设计越来越多地用于临床试验，以提高试验的灵活性和效率，并有可能减少资源、时间和成本。我们回顾了一些典型的自适应设计和新的统计方法，以应对自适应设计带来的统计挑战。临床试验设计的统计进展可能有助于缩短研究时间，并在发现神经系统疾病的新疗法期间使更多的患者获得更好的治疗。推进神经科学研究的统计基础是生物医学研究卓越奖支持的核心活动的关键方面，该奖项支持神经变性和转化神经科学中心。
New treatments for neurodegenerative disease are urgently needed, and clinical trial methods are an essential component of new drug development. Although a parallel-group study design for neurological disorder clinical trials is commonly used to test the effectiveness of a new treatment as compared to placebo, it does not efficiently use information from the on-going study to increase the success rate of a trial or to stop a trial earlier when the new treatment is indeed ineffective. We review some recent advances in designs for clinical trials, including futility designs and adaptive designs. Futility designs and noninferiority designs are used to test the nonsuperiority and the noninferiority of a new treatment, respectively. We provide some guidance on using these two designs and analyzing data from these studies properly. Adaptive designs are increasingly used in clinical trials to improve the flexibility and efficiency of trials with the potential to reduce resources, time, and costs. We review some typical adaptive designs and new statistical methods to handle the statistical challenges from adaptive designs. Statistical advances in clinical trial designs may be helpful to shorten study length and benefit more patients being treated with a better treatment during the discovery of new therapies for neurological disorders. Advancing statistical underpinnings of neuroscience research is a critical aspect of the core activities supported by the Center of Biomedical Research Excellence award supporting the Center for Neurodegeneration and Translational Neuroscience.