Cheng Bin, Chow Shein-Chung
Department of Biostatistics, Columbia University, New York, New York, USA.
J Biopharm Stat. 2010 Nov;20(6):1171-7. doi: 10.1080/10543406.2010.514460.
In recent years, the use of adaptive design methods in clinical trials has attracted much attention due to its flexibility in identifying the best clinical benefit of the test treatment under investigation. The flexibility, however, comes at the price of decreasing the accuracy and reliability of the statistical inference drawn. In addition, it is susceptible to abuse. The Food and Drug Administration (FDA) draft guidance justifiably distinguishes between well-understood and less well-understood adaptive designs and suggests the use of the latter with caution. In this discussion paper, we further classify the less well-understood adaptive designs into the categories of flexible and wildly flexible ones and recommend the latter not be used. In addition, we suggest a set of performance characteristics as criteria for choosing a good design from a pool of flexible adaptive designs and group sequential designs.
近年来，由于自适应设计方法在确定所研究的试验治疗的最佳临床益处方面具有灵活性，其在临床试验中的应用备受关注。然而，这种灵活性是以降低所做统计推断的准确性和可靠性为代价的。此外，它容易被滥用。美国食品药品监督管理局（FDA）的指导草案合理地区分了充分理解和理解较少的自适应设计，并建议谨慎使用后者。在这篇讨论文件中，我们进一步将理解较少的自适应设计分为灵活型和极其灵活型两类，并建议不要使用后者。此外，我们提出了一组性能特征，作为从一系列灵活的自适应设计和成组序贯设计中选择良好设计的标准。
