Use of historical control data to augment a small internal control arm in a randomized control trial (RCT) can lead to significant improvement of the efficiency of the trial. It introduces the risk of potential bias, since the historical control population is often rather different from the RCT. Power prior approaches have been introduced to discount the historical data to mitigate the impact of the population difference. However, even with a Bayesian dynamic borrowing which can discount the historical data based on the outcome similarity of the two populations, a considerable population difference may still lead to a moderate bias. Hence, a robust adjustment for the population difference using approaches such as the inverse probability weighting or matching, can make the borrowing more efficient and robust. In this paper, we propose a novel approach integrating propensity score for the covariate adjustment and Bayesian dynamic borrowing using power prior. The proposed approach uses Bayesian bootstrap in combination with the empirical Bayes method utilizing quasi-likelihood for determining the power prior. The performance of our approach is examined by a simulation study. We apply the approach to two Acute Myeloid Leukemia (AML) studies for illustration.
翻译:使用历史控制数据来增加随机控制试验(RCT)中的小型内部控制工具,可以大大提高审判的效率,从而带来潜在偏差的风险,因为历史控制人口往往与RCT有很大不同。已经采用了以往的权力方法,对历史数据进行贴现,以减轻人口差异的影响。然而,即使采用巴耶斯动态借款,根据两种人口类似的结果对历史数据进行贴现,人口差异仍可能导致适度的偏差。因此,采用反概率加权或匹配等方法对人口差异进行有力的调整,可以提高借款的效率和力度。在本文件中,我们提议采用新颖的办法,将共变调整的偏差分和先前使用权力的巴耶斯动态借款结合起来。拟议的办法与经验性海湾方法相结合,利用准相似的海带来确定先前的功率。我们的方法通过模拟研究加以审查。我们采用两种急性脊髓灰血病(AML)研究的方法进行说明。