We overview Bayesian estimation, hypothesis testing, and model-averaging and illustrate how they benefit parametric survival analysis. We contrast the Bayesian framework to the currently dominant frequentist approach and highlight advantages, such as seamless incorporation of historical data, continuous monitoring of evidence, and incorporating uncertainty about the true data generating process. We illustrate the application of the Bayesian approaches on an example data set from a colon cancer trial. We compare the Bayesian parametric survival analysis and frequentist models with AIC/BIC model selection in fixed-n and sequential designs with a simulation study. In the example data set, the Bayesian framework provided evidence for the absence of a positive treatment effect on disease-free survival in patients with resected colon cancer. Furthermore, the Bayesian sequential analysis would have terminated the trial 10.3 months earlier than the standard frequentist analysis. In a simulation study with sequential designs, the Bayesian framework on average reached a decision in almost half the time required by the frequentist counterparts, while maintaining the same power, and an appropriate false-positive rate. Under model misspecification, the Bayesian framework resulted in higher false-negative rate compared to the frequentist counterparts, which resulted in a higher proportion of undecided trials. In fixed-n designs, the Bayesian framework showed slightly higher power, slightly elevated error rates, and lower bias and RMSE when estimating treatment effects in small samples. We have made the analytic approach readily available in RoBSA R package. The outlined Bayesian framework provides several benefits when applied to parametric survival analyses. It uses data more efficiently, is capable of greatly shortening the length of clinical trials, and provides a richer set of inferences.
翻译:我们将贝叶斯估计、假设测试和模型稳定化加以审视,并举例说明这些模型如何有利于计量生存分析。我们将贝叶斯框架与目前占主导地位的贝伊斯框架比照目前流行的常流主义方法,并突出其优点,例如无缝地纳入历史数据、不断监测证据和纳入真实数据生成过程的不确定性。我们用结肠癌试验的一组示例数据来说明贝伊斯方法的应用。我们用模拟研究来比较巴伊西亚参数生存分析和常流模式与固定和连续设计中的AIC/BIC模型选择。在举例数据集中,贝伊斯框架提供了证据,表明对重结肠癌患者无病存活率缺乏积极治疗效果的证据。此外,巴伊斯连续分析比标准常流分析早10.3个月结束了试验。在进行测序设计的模拟研究中,巴伊斯框架平均在常流派对应方所需几乎一半的时间里作出决定,同时保持同样的动力,并有适当的更富反应率。在模型分析中,贝伊斯的临床框架提高了对无病的临床效果,在精确度分析中进行了更高的精确率率率分析,从而得出了精确度分析。