Sambucini Valeria
a Dipartimento di Scienze Statistiche , Sapienza Università di Roma , Roma , Italia.
J Biopharm Stat. 2015;25(3):474-89. doi: 10.1080/10543406.2014.920856.
Single-arm studies are typically used in phase II of clinical trials, whose main objective is to determine whether a new treatment warrants further testing in a randomized phase III trial. The introduction of randomization in phase II, to avoid the limits of studies based on historical controls, is a critical issue widely debated in the recent literature. We use a Bayesian approach to compare single-arm and randomized studies, based on a binary response variable, in terms of their abilities of reaching the correct decision about the new treatment, both when it performs better than the standard one and when it is less effective. We evaluate how the historical control rate, the total sample size, and the elicitation of the prior distributions affect the decision about which study performs better.
单臂研究通常用于临床试验的II期，其主要目的是确定一种新疗法是否值得在随机III期试验中进一步测试。在II期引入随机化以避免基于历史对照的研究的局限性，是近期文献中广泛讨论的一个关键问题。我们采用贝叶斯方法，基于二元反应变量，比较单臂研究和随机研究在新疗法比标准疗法效果更好以及效果更差时做出关于新疗法正确决策的能力。我们评估历史对照率、总样本量和先验分布的确定如何影响关于哪种研究表现更好的决策。
