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Treatment Benefit and Treatment Harm Rate to Characterize Heterogeneity in Treatment Effect
Author(s) -
Shen Changyu,
Jeong Jaesik,
Li Xiaochun,
Chen PengSheng,
Buxton Alfred
Publication year - 2013
Publication title -
biometrics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.298
H-Index - 130
eISSN - 1541-0420
pISSN - 0006-341X
DOI - 10.1111/biom.12038
Subject(s) - harm , treatment effect , sensitivity (control systems) , econometrics , population , randomized controlled trial , computer science , medicine , statistics , intensive care medicine , mathematics , psychology , engineering , environmental health , social psychology , electronic engineering , traditional medicine
Summary 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.