Accelerating Mental Health Precision Trial : An Effective Visualization-Driven Tool for Power and Sample Size Estimation in Biomarker-based Study Designs
Chen, D. Z.; Xie, A.; Ma, C.
Show abstract
Precision medicine has given rise to a spectrum of biomarker-guided trial designs, from simple enrichment and strategy designs to more complex adaptive frameworks. To address the need for user-friendly tools that span this spectrum, we developed a unified R Shiny platform that first implements three standard designs: the randomize-all design, the enrichment design, and the biomarker-strategy design, allowing researchers to perform power and sample size calculations under each framework with intuitive inputs and visual outputs. Building on this foundation, the platform further extends to support two-stage general randomized basket trial designs with interim analysis, which can be viewed as a generalization of the standard designs to multiple biomarker-defined subgroups. The tool was rigorously validated by comparison with established R pipelines and published formulas, and user testing confirmed its intuitive interface. By providing seamless integration from standard to advanced designs under a common input-output framework, our platform enables researchers to directly compare power and sample size requirements across different design choices using the same underlying assumptions. The result is a freely accessible tool offering effective visualizations for the full spectrum of biomarker-guided trial designs, available at https://ampt.obicloud.ca/. Future improvements may further expand the tools capabilities to accommodate the increasing complexity of trial designs needed by the research community.
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