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

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 © 2016 John Wiley & Sons, Ltd.

Source: Pharmaceutical Statistics - August 31, 2016 Category: Statistics Authors: Simon Kirby, Christy Chuang ‐Stein Tags: Main Paper Source Type: research