Hobbs Brian P, Carlin Bradley P, Mandrekar Sumithra J, Sargent Daniel J
Department of Biostatistics, M.D. Anderson Cancer Center, Houston, Texas 77030, USA.
Biometrics. 2011 Sep;67(3):1047-56. doi: 10.1111/j.1541-0420.2011.01564.x. Epub 2011 Mar 1.
Bayesian clinical trial designs offer the possibility of a substantially reduced sample size, increased statistical power, and reductions in cost and ethical hazard. However when prior and current information conflict, Bayesian methods can lead to higher than expected type I error, as well as the possibility of a costlier and lengthier trial. This motivates an investigation of the feasibility of hierarchical Bayesian methods for incorporating historical data that are adaptively robust to prior information that reveals itself to be inconsistent with the accumulating experimental data. In this article, we present several models that allow for the commensurability of the information in the historical and current data to determine how much historical information is used. A primary tool is elaborating the traditional power prior approach based upon a measure of commensurability for Gaussian data. We compare the frequentist performance of several methods using simulations, and close with an example of a colon cancer trial that illustrates a linear models extension of our adaptive borrowing approach. Our proposed methods produce more precise estimates of the model parameters, in particular, conferring statistical significance to the observed reduction in tumor size for the experimental regimen as compared to the control regimen.
贝叶斯临床试验设计提供了大幅减少样本量、提高统计功效以及降低成本和伦理风险的可能性。然而,当先验信息与当前信息冲突时,贝叶斯方法可能导致高于预期的I型错误,以及试验成本更高、时间更长的可能性。这促使人们研究分层贝叶斯方法的可行性,该方法用于纳入对先验信息具有自适应稳健性的历史数据,而这些先验信息被证明与累积的实验数据不一致。在本文中,我们提出了几个模型,这些模型允许历史数据和当前数据中的信息具有可比性,以确定使用了多少历史信息。一个主要工具是基于高斯数据的可比性度量来阐述传统的功效先验方法。我们通过模拟比较了几种方法的频率主义性能,并以一个结肠癌试验的例子作为结尾,该例子说明了我们的自适应借用方法的线性模型扩展。我们提出的方法对模型参数产生了更精确的估计,特别是与对照方案相比,赋予了实验方案中观察到的肿瘤大小减少以统计学显著性。