A comparison of five approaches to decision-making for a first clinical trial of efficacy

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• 作者：Simon Kirby and Christy Chuang-Stein
• 刊名：Pharmaceutical Statistics
• 出版年：2017
• 出版时间：January/February 2017
• 年：2017
• 卷：16
• 期：1
• 页码：37-44
• 全文大小：817K
• ISSN：1539-1612

文摘

The first trial of clinical efficacy is an important step in the development of a compound. Such a trial gives the first indication of whether a compound is likely to have the efficacy needed to be successful. Good decisions dictate that good compounds have a large probability of being progressed and poor compounds have a large probability of being stopped. In this paper, we consider and contrast five approaches to decision-making that have been used. To illustrate the use of the five approaches, we conduct a comparison for two plausible scenarios with associated assumptions for sample sizing. The comparison shows some large differences in performance characteristics of the different procedures. Which decision-making procedures and associated performance characteristics are preferred will depend on the focus of interest and the decision maker's attitude to risk. Copyright