Treatment Benefit and Treatment Harm Rate to Characterize Heterogeneity in Treatment Effect

Changyu Shen, Jaesik Jeong, Xiaochun Li, Peng Sheng Chen, Alfred Buxton

Research output: Contribution to journalArticle

7 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)724-731
Number of pages8
JournalBiometrics
Volume69
Issue number3
DOIs
StatePublished - Sep 2013

Keywords

  • Causal inference
  • Heterogeneity in treatment effect
  • Potential outcomes
  • Sub-group analysis

ASJC Scopus subject areas

  • Applied Mathematics
  • Statistics and Probability
  • Agricultural and Biological Sciences(all)
  • Biochemistry, Genetics and Molecular Biology(all)
  • Immunology and Microbiology(all)
  • Medicine(all)

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