Optimizing Aggregated N-Of-1 Trial Designs for Predictive Biomarker Validation: Statistical Methods and Theoretical Findings.

ABSTRACT: Background and Significance: Parallel-group randomized controlled trials (PG-RCTs) are the gold standard for detecting differences in mean improvement across treatment conditions. However, PG-RCTs provide limited information about individuals, making them poorly optimized for quantifying the relationship of a biomarker measured at baseline with treatment response. In N-of-1 trials, an individual subject moves between treatment conditions to determine their specific response to each treatment. Aggregated N-of-1 trials analyze a cohort of such participants, and can be designed to optimize both statistical power and clinical or logistical constraints, such as allowing all participants to begin with an open-label stabilization phase to facilitate the enrollment of more acutely symptomat

SUBMITTER: Hendrickson RC 

PROVIDER: S-EPMC8521797 | biostudies-literature | 2020

REPOSITORIES: biostudies-literature