Bayesian statistics and the efficiency and ethics of clinical trials
Bayesian statistics and the efficiency and ethics of clinical trials
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DOI：
10.1214/088342304000000044
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发表时间：
2004-02-01
影响因子：
5.7
通讯作者：
Berry, DA
中科院分区：
文献类型：
--
作者：
Berry, DA
贝叶斯方法在医学研究中得到越来越多的应用。贝叶斯方法的灵活性允许构建具有任何期望类型的良好特性的临床试验设计。例子包括在试验中最大限度地有效治疗患者，最大限度地获得有关剂量-反应曲线斜率的信息，最大限度地降低成本，最大限度地减少治疗患者的数量，最大限度地减少试验的长度以及这些所需的组合。当这些是重要的考虑因素时，它们还包括标准频率工作特性。后验概率在积累数据的基础上，通过贝叶斯定理进行更新。这些用于影响试验过程的修改，包括停止应计项目，将应计项目延长到原计划之外，减少治疗组，增加治疗组等。我提倡的方法的一个重要方面是模拟试验的主要终点和患者表现的早期指征之间的关系辅助终点。这有几个非常理想的结果。其一，它提高了适应性试验的效率，因为信息比其他方法更容易获得。
The Bayesian approach is being used increasingly in medical research. The flexibility of the Bayesian approach allows for building designs of clinical trials that have good properties of any desired sort. Examples include maximizing effective treatment of patients in the trial, maximizing information about the slope of a dose-response curve, minimizing costs, minimizing the number of patients treated, minimizing the length of the trial and combinations of these desiderata. They also include standard frequentist operating characteristics when these are important considerations. Posterior probabilities are updated via Bayes' theorem on the basis of accumulating data. These are used to effect modifications of the trial's course, including stopping accrual, extending accrual beyond that originally planned, dropping treatment arms, adding arms, etc. An important aspect of the approach I advocate is modeling the relationship between a trial's primary endpoint and early indications of patient performance-auxiliary endpoints. This has several highly desirable consequences. One is that it improves the efficiency of adaptive trials because information is available sooner than otherwise.