A critical evaluation of longitudinal proportional effect models

Fuente: arXiv
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Hauptverfasser: Donohue, Michael C., Insel, Philip S., Langford, Oliver
Format: Preprint
Veröffentlicht: 2025
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author Donohue, Michael C.
Insel, Philip S.
Langford, Oliver
author_facet Donohue, Michael C.
Insel, Philip S.
Langford, Oliver
contents Nonlinear longitudinal proportional effect models have been proposed to improve power and provide direct estimates of the proportional treatment effect in randomized clinical trials. These models assume a fixed proportional treatment effect over time, which can lead to bias and Type I error inflation when the assumption is violated. Even when the proportional effect assumption holds, these models are biased, and their inference is sensitive to the labeling of treatment groups. Typically, this bias favors the active group, inflates Type I error, and can result in one-sided testing. Conversely, the bias can make it more difficult to detect treatment harm, creating a safety concern.
format Preprint
id arxiv_https___arxiv_org_abs_2502_00214
institution arXiv
publishDate 2025
record_format arxiv
spellingShingle A critical evaluation of longitudinal proportional effect models
Donohue, Michael C.
Insel, Philip S.
Langford, Oliver
Methodology
Nonlinear longitudinal proportional effect models have been proposed to improve power and provide direct estimates of the proportional treatment effect in randomized clinical trials. These models assume a fixed proportional treatment effect over time, which can lead to bias and Type I error inflation when the assumption is violated. Even when the proportional effect assumption holds, these models are biased, and their inference is sensitive to the labeling of treatment groups. Typically, this bias favors the active group, inflates Type I error, and can result in one-sided testing. Conversely, the bias can make it more difficult to detect treatment harm, creating a safety concern.
title A critical evaluation of longitudinal proportional effect models
topic Methodology
url https://arxiv.org/abs/2502.00214