Optimal planning of adaptive two-stage designs

Adaptive designs are playing an increasingly important role in the planning of clinical trials. While there exists various research on the optimal determination of a two-stage design, non-optimal versions still are frequently applied in clinical research. In this article, we strive to motivate the a...

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Bibliographic Details
Main Authors: Pilz, Maximilian (Author) , Kunzmann, Kevin (Author) , Herrmann, Carolin (Author) , Rauch, Geraldine (Author) , Kieser, Meinhard (Author)
Format: Article (Journal)
Language:English
Published: 15 June 2021
In: Statistics in medicine
Year: 2021, Volume: 40, Issue: 13, Pages: 3196-3213
ISSN:1097-0258
DOI:10.1002/sim.8953
Online Access:Verlag, kostenfrei, Volltext: https://doi.org/10.1002/sim.8953
Verlag, kostenfrei, Volltext: https://onlinelibrary.wiley.com/doi/abs/10.1002/sim.8953
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Author Notes:Maximilian Pilz, Kevin Kunzmann, Carolin Herrmann, Geraldine Rauch, Meinhard Kieser
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Summary:Adaptive designs are playing an increasingly important role in the planning of clinical trials. While there exists various research on the optimal determination of a two-stage design, non-optimal versions still are frequently applied in clinical research. In this article, we strive to motivate the application of optimal adaptive designs and give guidance on how to determine them. It is demonstrated that optimizing a trial design with respect to particular objective criteria can have a substantial benefit over the application of conventional adaptive sample size recalculation rules. Furthermore, we show that in many practical situations, optimal group-sequential designs show an almost negligible performance loss compared to optimal adaptive designs. Finally, we illustrate how optimal designs can be tailored to specific operational requirements by customizing the underlying optimization problem.
Item Description:Gesehen am 17.04.2023
Physical Description:Online Resource
ISSN:1097-0258
DOI:10.1002/sim.8953