Abstract:
:In clinical research, investigators are interested in inferring the average causal effect of a treatment. However, the causal parameter that can be used to derive the average causal effect is not well defined for ordinal outcomes. Although some definitions have been proposed, they are limited in that they are not identical to the well-defined causal risk for a binary outcome, which is the simplest ordinal outcome. In this paper, we propose the use of a causal parameter for an ordinal outcome, defined as the proportion that a potential outcome under one treatment condition would not be smaller than that under the other condition. For a binary outcome, this proportion is identical to the causal risk. Unfortunately, the proposed causal parameter cannot be identified, even under randomization. Therefore, we present a numerical method to calculate the sharp nonparametric bounds within a sample, reflecting the impact of confounding. When the assumption of independent potential outcomes is included, the causal parameter can be identified when randomization is in play. Then, we present exact tests and the associated confidence intervals for the relative treatment effect using the randomization-based approach, which are an extension of the existing methods for a binary outcome. Our methodologies are illustrated using data from an emetic prevention clinical trial.
journal_name
Stat Medjournal_title
Statistics in medicineauthors
Chiba Ydoi
10.1002/sim.7400subject
Has Abstractpub_date
2017-11-10 00:00:00pages
3966-3975issue
25eissn
0277-6715issn
1097-0258journal_volume
36pub_type
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