Walley Rosalind J, Smith Claire L, Gale Jeremy D, Woodward Phil
UCB Pharma, 208 Bath Road, Slough, SL1 3WE, UK.
Eli Lilly and Company Limited, Erl Wood Manor, Windlesham, GU20 6PH, UK.
Pharm Stat. 2015 May-Jun;14(3):205-15. doi: 10.1002/pst.1675. Epub 2015 Apr 10.
This paper illustrates how the design and statistical analysis of the primary endpoint of a proof-of-concept study can be formulated within a Bayesian framework and is motivated by and illustrated with a Pfizer case study in chronic kidney disease. It is shown how decision criteria for success can be formulated, and how the study design can be assessed in relation to these, both using the traditional approach of probability of success conditional on the true treatment difference and also using Bayesian assurance and pre-posterior probabilities. The case study illustrates how an informative prior on placebo response can have a dramatic effect in reducing sample size, saving time and resource, and we argue that in some cases, it can be considered unethical not to include relevant literature data in this way.
本文阐述了如何在贝叶斯框架内制定概念验证研究主要终点的设计和统计分析，并以辉瑞公司在慢性肾病方面的案例研究为例进行说明。文中展示了如何制定成功的决策标准，以及如何根据这些标准评估研究设计，既使用基于真实治疗差异的成功概率的传统方法，也使用贝叶斯保证和先验-后验概率。该案例研究表明，关于安慰剂反应的信息性先验如何能在减少样本量、节省时间和资源方面产生显著效果，并且我们认为在某些情况下，不以这种方式纳入相关文献数据可能被视为不道德。
