title: Treatment benefit and treatment harm rate to characterize heterogeneity in treatment effect.
abstract: It is well recognized that the conventional summary of treatment effect by averaging across individual patients has its limitation in ignoring the heterogeneous responses to the treatment in the target population. However, there are few alternative metrics in the literature that are designed to capture such heterogeneity. We propose the concept of treatment benefit rate (TBR) and treatment harm rate (THR) that characterize both the overall treatment effect and the magnitude of heterogeneity. We discuss a method to estimate TBR and THR that easily incorporates a sensitivity analysis scheme, and illustrate the idea through analysis of a randomized trial that evaluates the implantable cardioverter-defibrillator (ICD) in reducing mortality. A simulation study is presented to assess the performance of the proposed method.
authors.affiliation: Department of Biostatistics, Indiana University School of Medicine, Indianapolis, Indiana 46202, U.S.A.
authors.forename: Changyu
authors.initials: C
authors.lastname: Shen
authors.forename: Jaesik
authors.initials: J
authors.lastname: Jeong
authors.forename: Xiaochun
authors.initials: X
authors.lastname: Li
authors.forename: Peng-Sheng
authors.initials: PS
authors.lastname: Chen
authors.forename: Alfred
authors.initials: A
authors.lastname: Buxton
mesh_terms: D001145:Arrhythmias, Cardiac / Q000401:mortality / Q000628:therapy; D001699:Biometry / Q000379:methods*; D003198:Computer Simulation; D017147:Defibrillators, Implantable; D006801:Humans; D016032:Randomized Controlled Trials as Topic / Q000706:statistics & numerical data; D016896:Treatment Outcome
keywords: Causal inference; Heterogeneity in treatment effect; Potential outcomes; Sub-group analysis