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arXiv · 2406.19965

Futility analyses for the MCP-Mod methodology based on longitudinal models

Björn Bornkamp·Jie Zhou·Dong Xi·Weihua Cao
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Abstract

This article discusses futility analyses for the MCP-Mod methodology. Formulas are derived for calculating predictive and conditional power for MCP-Mod, which also cover the case when longitudinal models are used allowing to utilize incomplete data from patients at interim. A simulation study is conducted to evaluate the repeated sampling properties of the proposed decision rules and to assess the benefit of using a longitudinal versus a completer only model for decision making at interim. The results suggest that the proposed methods perform adequately and a longitudinal analysis outperforms a completer only analysis, particularly when the recruitment speed is higher and the correlation over time is larger. The proposed methodology is illustrated using real data from a dose-finding study for severe uncontrolled asthma.

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Björn Bornkamp, Jie Zhou, Dong Xi, Weihua Cao. 2024-06-28. Futility analyses for the MCP-Mod methodology based on longitudinal models. https://arxiv.org/abs/2406.19965

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