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Estimation of treatment effects in randomized trials with non-compliance and a dichotomous outcome

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  • Mark J. van der Laan
  • Alan Hubbard
  • Nicholas P. Jewell

Abstract

We propose a class of estimators of the treatment effect on a dichotomous outcome among the treated subjects within covariate and treatment arm strata in randomized trials with non-compliance. Recent papers by Vansteelandt and Goetghebeur, and Robins and Rotnitzky have presented consistent and asymptotically linear estimators of a causal odds ratio, which rely, beyond correct specification of a model for the causal odds ratio, on a correctly specified model for a potentially high dimensional nuisance parameter. In this paper we propose consistent, asymptotically linear and locally efficient estimators of a causal relative risk and a new parameter-called a switch causal relative risk-which relies only on the correct specification of a model for the parameter of interest. Our estimators are always consistent and asymptotically linear at the null hypothesis of no-treatment effect, thereby providing valid testing procedures. We examine the finite sample properties of these instrumental-variable-based estimators and the associated testing procedures in simulations and a data analysis of decaffeinated coffee consumption and miscarriage. Copyright 2007 Royal Statistical Society.

Suggested Citation

  • Mark J. van der Laan & Alan Hubbard & Nicholas P. Jewell, 2007. "Estimation of treatment effects in randomized trials with non-compliance and a dichotomous outcome," Journal of the Royal Statistical Society Series B, Royal Statistical Society, vol. 69(3), pages 463-482.
  • Handle: RePEc:bla:jorssb:v:69:y:2007:i:3:p:463-482
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    Cited by:

    1. Paul S. Clarke & Tom M. Palmer & Frank Windmeijer, 2011. "Estimating structural mean models with multiple instrumental variables using the generalised method of moments," CeMMAP working papers CWP28/11, Centre for Microdata Methods and Practice, Institute for Fiscal Studies.
    2. Paul S. Clarke & Frank Windmeijer, 2012. "Instrumental Variable Estimators for Binary Outcomes," Journal of the American Statistical Association, Taylor & Francis Journals, vol. 107(500), pages 1638-1652, December.
    3. Paul Clarke & Frank Windmeijer, 2009. "Identification of Causal Effects on Binary Outcomes Using Structural Mean Models," The Centre for Market and Public Organisation 09/217, Department of Economics, University of Bristol, UK.

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