Bornkamp, Bjoern, Zhou, Jie, Xi, Dong and Cao, Weihua (2025) Futility Analyses for the MCP-Mod Methodology Based on Longitudinal Models. Statistics in medicine, 44 (20-22). e70274. ISSN 1097-0258

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.

Item Type:	Article
Keywords:	Humans Longitudinal Studies Asthma Models, Statistical Computer Simulation Medical Futility
Date Deposited:	08 Oct 2025 00:45
Last Modified:	08 Oct 2025 00:45
URI:	https://oak.novartis.com/id/eprint/54434