FRBx: A Threshold-Based Measure of the Fraction Who Benefit for Ordinal Clinical Outcomes

Abstract

Randomized trial analyses typically focus on the average treatment effect. While highly useful, this metric cannot distinguish whether treatment benefits the majority or a small subgroup. Thus, researchers proposed the metric of fraction who benefit (FRB), which measures the proportion of patients who would have a better outcome under treatment than control. Statistical methodology has been developed for the FRB. However, the FRB framework does not account for the magnitude of benefit - how small or large are the benefits that individual patients receive?

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Non-identifiable parameter, Ordinal outcome, Randomized trial, Treatment effect heterogeneity

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Wake Forest University