Estimating quantile treatment effect on the original scale of the outcome variable: a case study of common cold treatments

TL;DR

This paper introduces a method to estimate and visualize quantile treatment effects on the original measurement scale, providing more detailed insights into treatment effects across the entire population.

Contribution

The authors developed a back-transformed QTE (BQTE) method that presents effects on the original outcome scale, enhancing interpretability over traditional quantile-based presentation.

Findings

Relative scale summarizes BQTE distribution better than mean difference.

The BQTE approach effectively describes variability in treatment effects.

Application to cold treatment data demonstrates practical utility.

Abstract

The effects of treatments on continuous outcomes can be estimated by the mean difference (i.e. by measurement units) and the relative effect scales (i.e. by percentages), both of which provide only a single effect size estimate over the study population. Quantile treatment effect (QTE) analysis is more informative as it describes the effect of the treatment across the whole population. A drawback of QTE has been that it is usually presented over the quantiles of the control group distribution, whereas presentation over the measurement units is often more informative. We developed a method to estimate back-transformed QTE (BQTE), that presents QTE as a function of the outcome value in the control group, using piecewise linear interpolation and bootstrapping. We further applied the BQTE function to provide informative bounds on the treatment effect at the upper and lower tails of the population. To illustrate the approach, we used 3 data sets of treatment for the common cold: zinc gluconate lozenges, zinc acetate lozenges, and nasal carrageenan. In all data sets, the relative scale provided a better summary of the BQTE distribution than the mean difference. The BQTE approach is particularly useful for describing the variability of effects on the duration of illnesses, length of hospital stay and other continuous outcomes that can vary greatly in the population. Using this method, it is possible to present the QTE by the measurement units, which provides an informative addition to the standard presentation by quantiles.