Assessing the heterogeneity of treatment effects via potential outcomes of individual patients

Zhiwei Zhang, Chenguang Wang, Lei Nie, Guoxing Soon

Research output: Contribution to journalArticlepeer-review

20 Scopus citations


Summary: There is growing interest in understanding the heterogeneity of treatment effects (HTE), which has important implications in treatment evaluation and selection. The standard approach to assessing HTE (i.e. subgroup analyses based on known effect modifiers) is informative about the heterogeneity between subpopulations but not within. It is arguably more informative to assess HTE in terms of individual treatment effects, which can be defined by using potential outcomes. However, estimation of HTE based on potential outcomes is challenged by the lack of complete identifiability. The paper proposes methods to deal with the identifiability problem by using relevant information in baseline covariates and repeated measurements. If a set of covariates is sufficient for explaining the dependence between potential outcomes, the joint distribution of potential outcomes and hence all measures of HTE will then be identified under a conditional independence assumption. Possible violations of this assumption can be addressed by including a random effect to account for residual dependence or by specifying the conditional dependence structure directly. The methods proposed are shown to reduce effectively the uncertainty about HTE in a trial of human immunodeficiency virus.

Original languageEnglish (US)
Pages (from-to)687-704
Number of pages18
JournalJournal of the Royal Statistical Society. Series C: Applied Statistics
Issue number5
StatePublished - Nov 2013
Externally publishedYes


  • Causal inference
  • Conditional independence
  • Copula
  • Counterfactual
  • Random effect
  • Sensitivity analysis

ASJC Scopus subject areas

  • Statistics and Probability
  • Statistics, Probability and Uncertainty


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