Testing Risk Difference of Two Proportions for Combined Unilateral and Bilateral Data

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Hauptverfasser: Zhou, Jia, Ma, Chang-Xing
Format: Preprint
Veröffentlicht: 2025
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author Zhou, Jia
Ma, Chang-Xing
author_facet Zhou, Jia
Ma, Chang-Xing
contents In clinical studies with paired organs, binary outcomes often exhibit intra-subject correlation and may include a mixture of unilateral and bilateral observations. Under Donner's constant correlation model, we develop three likelihood-based test statistics (the likelihood ratio, Wald-type, and score tests) for assessing the risk difference between two proportions. Simulation studies demonstrate good control of type I error and comparable power among the three tests, with the score test showing slightly better stability. Applications to otolaryngologic and ophthalmologic data illustrate the methods. An online calculator is also provided for power analysis and risk difference testing. The score test is recommended for practical use and future studies with combined unilateral and bilateral binary data.
format Preprint
id arxiv_https___arxiv_org_abs_2510_18834
institution arXiv
publishDate 2025
record_format arxiv
spellingShingle Testing Risk Difference of Two Proportions for Combined Unilateral and Bilateral Data
Zhou, Jia
Ma, Chang-Xing
Methodology
62F03
In clinical studies with paired organs, binary outcomes often exhibit intra-subject correlation and may include a mixture of unilateral and bilateral observations. Under Donner's constant correlation model, we develop three likelihood-based test statistics (the likelihood ratio, Wald-type, and score tests) for assessing the risk difference between two proportions. Simulation studies demonstrate good control of type I error and comparable power among the three tests, with the score test showing slightly better stability. Applications to otolaryngologic and ophthalmologic data illustrate the methods. An online calculator is also provided for power analysis and risk difference testing. The score test is recommended for practical use and future studies with combined unilateral and bilateral binary data.
title Testing Risk Difference of Two Proportions for Combined Unilateral and Bilateral Data
topic Methodology
62F03
url https://arxiv.org/abs/2510.18834