Principal quantile treatment effect estimation using principal scores.

Abstract

Intercurrent events and estimands play a key role in defining the treatment effects of interest precisely. Sometimes the median or other quantiles of outcomes in a principal stratum according to potential occurrence of intercurrent events are of interest in randomized clinical trials. Naïve analyses such as those based on the observed occurrence of the intercurrent events lead to biased results. Therefore, we propose principal quantile treatment effect estimators that can nonparametrically estimate the distribution of potential outcomes by principal score weighting without relying on the exclusion restriction assumption. Our simulation studies show that the proposed method works in situations where the median or quantiles may be regarded as the preferred population-level summary over the mean. We illustrate our proposed method by using data from a randomized controlled trial conducted on patients with nonerosive reflux disease.