Decision‐making in early clinical drug development. (17th March 2016)

• Record Type: Journal Article
• Authors: Frewer, Paul; Mitchell, Pat; Watkins, Claire; Matcham, James
• Is Part Of: Pharmaceutical statistics. Volume 15:Number 3(2016)
• Page Start: 255
• Page End: 263
• Publication Date: 2016-03-17
• Subjects: decision criteria -- operating characteristics -- interim analysis -- three‐outcome designs; Pharmacy -- Statistical methods -- Periodicals; Pharmacy -- Statistics -- Periodicals; 615.10727
• DOI: 10.1002/pst.1746
• Languages: English
• ISSNs: 1539-1604

Abstract

Abstract : 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.