Multi-Arm, Multi-Stage Designs for Small Patient Populations
Multi-Arm, Multi-Stage Designs for Small Patient Populations Optimizing Rare Disease Trials with Multi-Arm, Multi-Stage Designs Introduction: The Need for Innovative Designs in Rare Disease Research Rare disease clinical trials face persistent challenges—limited patient populations, ethical constraints around control arms, and high uncertainty in treatment effects. In such scenarios, traditional parallel-group designs can be inefficient, slow,…
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