A Longitudinal Model and Graphic for Benefit-risk Analysis,with Case Study

Abstract

A novel method for simultaneously visualizing benefit and risk over time is presented. The underlying model represents a subject's benefit-risk state at a given time as one of five discrete clinical states, one being premature study withdrawal. The new graphic uses colors to represent each subject's changing state over the course of the clinical trial. The user can grasp how a treatment affects subjects in aggregate, then further examine how individuals are affected. It is possible to tell whether the beneficial and harmful outcomes are correlated. The method is particularly appropriate for treatments that provide only symptomatic relief. An approved drug for chronic pain is presented as a worked example.