Statistical Considerations in Proof-of-Concept Studies

Laurence Colin () and Brian Smith
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Laurence Colin: Novartis Institutes for Biomedical Research
Brian Smith: Novartis Institutes for Biomedical Research

A chapter in Statistical Methods in Biomarker and Early Clinical Development, 2019, pp 221-245 from Springer

Abstract: Abstract The proof-of-concept study is typically the earliest study investigating whether the compound has any efficacy in a patient population. A proof-of-concept study is defined as small-scale trial in well-defined diseases or targeted patient populations, which allows testing of a preclinical hypothesis about a mechanism of action and a quick demonstration of potential therapeutic benefit to patients. In addition, there is growing emphasis on the need to show as early as possible that the compound hit its intended target, which we call proof-of-mechanism. Demonstrating proof-of-mechanism is especially important, as we will discuss in later sections, when clinical activity results are negative.

Date: 2019
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DOI: 10.1007/978-3-030-31503-0_11

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