A model-informed clinical trial simulation tool with a graphical user interface for Duchenne muscular dystrophy.

ABSTRACT: Quantitative model-based clinical trial simulation tools play a critical role in informing study designs through simulation before actual execution. These tools help drug developers explore various trial scenarios in silico to select a clinical trial design to detect therapeutic effects more efficiently, therefore reducing time, expense, and participants' burden. To increase the usability of the tools, user-friendly and interactive platforms should be developed to navigate various simulation scenarios. However, developing such tools challenges researchers, requiring expertise in modeling and interface development. This tutorial aims to address this gap by guiding developers in creating tailored R Shiny apps, using an example of a model-based clinical trial simulation tool that we developed

SUBMITTER: Kim J 

PROVIDER: S-EPMC12625128 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature