Can the Concept Be Proven?

Ying-Ying Zhang () and Naitee Ting ()
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Ying-Ying Zhang: Chongqing University
Naitee Ting: Boehringer Ingelheim Pharmaceuticals, Inc.

Statistics in Biosciences, 2021, vol. 13, issue 1, No 9, 160-177

Abstract: Abstract During clinical development of new medicinal products, phase II is one of the most important stages. The first phase II trial is typically a Proof of Concept (PoC) study with limited sample size. To reduce the bias, one needs to discount the phase II estimate of the treatment effect. Under some mild conditions, the estimated assurances are increasing functions of the per group number of patients and the scaled observed treatment effect of the phase II trial for the three cases (no, additive, and multiplicative bias adjustment); and for multiplicative bias adjustment, the estimated assurance is an increasing function of the retention factor. The theoretical assurances are increasing functions of the per group number of patients of the phase II trial and the scaled true treatment effect of the phase III trial. The numerical simulations illustrate the above theoretical results. After obtaining the results of the phase II trial, it is still difficult for the project team (and the company) to make the Go/No Go decision. Finally, in addition to the investment and the statistical points of view, the Go/No Go decision is also affected by many other factors.

Keywords: Phase II/III clinical trials; Proof of Concept; Launch criterion; Assurance; Go/No Go decision (search for similar items in EconPapers)
Date: 2021
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Citations: View citations in EconPapers (1)

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DOI: 10.1007/s12561-020-09290-3

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