Analysis of treatment response data from eligibility designs

Abstract

In this paper, we develop and compare two alternative approaches for calculating the effect of the actual intake when treatments are randomized, but compliance with the assignment in the treatment arm is less than perfect for reasons that are correlated with the outcome. The approaches are based on different identification assumptions about these unobserved confounders. In the first approach, which stems from [Sommer, A., Zeger, S., 1991. On estimating efficacy in clinical trials. Statistics in Medicine 10, 45-52], the unobserved confounders are modeled by a discrete indicator variable that represents subject-type, defined in terms of the potential intake in the face of each possible assignm..