An Approach for Estimating Causal Effects in Randomized Trials with Noncompliance

Abstract

We developed methods for estimating the causal risk difference and causal risk ratio in randomized trials with noncompliance. The developed estimator is unbiased under the assumption that biases due to noncompliance are identical between both treatment arms. The biases are defined as the difference or ratio between the expectations of potential outcomes for a group that received the test treatment and that for the control group in each randomly assigned group. Although the instrumental variable estimator yields an unbiased estimate under a sharp null hypothesis but may yield a biased estimate under a non-null hypothesis, the bias of the developed estimator does not depend on whether this hypothesis holds. Then the estimate of the causal effect from the developed estimator may have a smaller bias than that from the instrumental variable estimator when the treatment effect exists. There is not yet a standard method for coping with noncompliance, and thus it is important to evaluate estimates under different assumptions. The developed estimator can serve this purpose. Its application to a field trial for coronary heart disease is provided.

Keywords:

• Causal inference
• Instrumental variable
• Potential outcome

Mathematics Subject Classification:

• 62P10
• 92B15

Acknowledgment

The author thanks the reviewer for comments and suggestions.