Utility-based optimization of Fujikawa's basket trial design -- Pre-specified protocol of a comparison study
Abstract
Basket trial designs are a type of master protocol in which the same therapy is tested in several strata of the patient cohort. Many basket trial designs implement borrowing mechanisms. These allow sharing information between similar strata with the goal of increasing power in responsive strata while at the same time constraining type-I error inflation to a bearable threshold. These borrowing mechanisms can be tuned using numerical tuning parameters. The optimal choice of these tuning parameters is subject to research. In a comparison study using simulations and numerical calculations, we are planning to investigate the use of utility functions for quantifying the compromise between power and type-I error inflation and the use of numerical optimization algorithms for optimizing these functions. The present document is the protocol of this comparison study, defining each step of the study in accordance with the ADEMP scheme for pre-specification of simulation studies.
Keywords
Cite
@article{arxiv.2403.02058,
title = {Utility-based optimization of Fujikawa's basket trial design -- Pre-specified protocol of a comparison study},
author = {Lukas D Sauer and Alexander Ritz and Meinhard Kieser},
journal= {arXiv preprint arXiv:2403.02058},
year = {2024}
}
Comments
26 pages, 1 figure; updated content in reaction to anonymous review: new section "Methodology of utility functions in basket trial designs", discussion of literature and four new scenario sets in section "Outcome scenarios", two new algorithms and detailed explanation in section "Optimization algorithms", new section "Discussion", further minor changes