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

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

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    Article provided by Royal Statistical Society in its journal Journal of the Royal Statistical Society: Series B (Statistical Methodology).

    Volume (Year): 69 (2007)
    Issue (Month): 3 ()
    Pages: 463-482

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    Handle: RePEc:bla:jorssb:v:69:y:2007:i:3:p:463-482
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