A Cautionary Note When a Dose-Ranging Study is Used for Proving the Concept

Qiqi Deng, Kun Wang, Xiaofei Bai and Naitee Ting ()
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Qiqi Deng: Boehringer-Ingelheim Pharmaceuticals, Inc.
Kun Wang: Shenzhen Middle School
Xiaofei Bai: Boehringer-Ingelheim Pharmaceuticals, Inc.
Naitee Ting: Boehringer-Ingelheim Pharmaceuticals, Inc.

Statistics in Biosciences, 2019, vol. 11, issue 1, No 6, 127-140

Abstract: Abstract The objective of a Proof-of-Concept (PoC) clinical trial is to formulate a “Go/NoGo” decision based on study results. If such a decision cannot be made from outcomes of a PoC trial, then this creates a situation of inconclusiveness. Inconclusiveness could lead to many undesirable consequences. Recently, many project teams are combining the PoC with dose-ranging studies. When this is the case, there are likely more potential of causing inconclusiveness. This paper points out some of these risks, and hopes to caution project team members to consider these risks while designing a combined PoC and dose-ranging clinical trial. When studying the problem of inconclusiveness, the concept of minimally statistically significant difference is extended from a two-sample PoC setting to a combined PoC and dose-ranging trial where multiple dose groups are involved.

Keywords: Clinical trial; PoC; Dose–ranging; MSSD; Go/NoGo; Inconclusiveness (search for similar items in EconPapers)
Date: 2019
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DOI: 10.1007/s12561-018-9224-5

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