Decision-making in early clinical drug development.
This paper illustrates an approach to setting the decision framework for a study in early clinical drug development. It shows how the criteria for a go and a stop decision are calculated based on pre-specified target and lower reference values. The framework can lead to a three-outcome approach by including a consider zone; this could enable smaller studies to be performed in early development, with other information either external to or within the study used to reach a go or stop decision. In this way, Phase I/II trials can be geared towards providing actionable decision-making rather than the traditional focus on statistical significance. The example provided illustrates how the decision criteria were calculated for a Phase II study, including an interim analysis, and how the operating characteristics were assessed to ensure the decision criteria were robust. Copyright © 2016 John Wiley & Sons, Ltd.
['Frewer|Paul|P|', 'Mitchell|Pat|P|', 'Watkins|Claire|C|', 'Matcham|James|J|']
D017321:Clinical Trials, Phase I as Topic D017322:Clinical Trials, Phase II as Topic D003627:Data Interpretation, Statistical D003657:Decision Making D015195:Drug Design D006801:Humans D012107:Research Design
decision criteria interim analysis operating characteristics three-outcome designs