Renfro Lindsay A, Mandrekar Sumithra J
a Division of Biomedical Statistics and Informatics , Mayo Clinic , Rochester , MN , USA.
J Biopharm Stat. 2018;28(2):217-228. doi: 10.1080/10543406.2017.1372778. Epub 2017 Oct 9.
Within the field of cancer research, discovery of biomarkers and genetic mutations that are potentially predictive of treatment benefit is motivating a paradigm shift in how cancer clinical trials are conducted. In this review, we provide an overview of the class of trials known as "master protocols," including basket trials, umbrella trials, and platform trials. For each, we describe standardized terminology, provide a motivating example with modeling details and decision rules, and discuss statistical advantages and limitations. We conclude with a discussion of general statistical considerations and challenges encountered across these types of trials.
在癌症研究领域，发现可能预测治疗获益的生物标志物和基因突变正在推动癌症临床试验开展方式的范式转变。在本综述中，我们概述了被称为“主方案”的试验类别，包括篮子试验、伞式试验和平台试验。对于每一种试验，我们描述了标准化术语，提供了一个带有建模细节和决策规则的激励性示例，并讨论了统计优势和局限性。我们最后讨论了这些类型试验中遇到的一般统计考虑因素和挑战。
