Pharmaceutical Statistics· 2026Q1
Testing Risk Difference of Two Proportions for Combined Unilateral and Bilateral Data
- 0citations
- Q1SCImago
- 2026year
Short summary
Three new likelihood-based tests (likelihood ratio, Wald-type, score) accurately assess risk differences between two proportions in clinical studies with paired organs, even with mixed unilateral and bilateral data.
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Key points
- Developed three likelihood-based tests (likelihood ratio, Wald-type, score) for risk difference between two proportions.
- Methods are applicable to paired organ data with intra-subject correlation and mixed unilateral/bilateral observations.
- Simulation studies confirm good type I error control and comparable power for all three tests.
- The score test demonstrated slightly superior performance, especially with moderate to large sample sizes.
AI-generated from the title and abstract; the full text is not read.
Abstract
ABSTRACT 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 more favorable performance, particularly for moderate to large sample sizes, supporting its use in practical applications and future studies involving combined unilateral and bilateral data. Applications to otolaryngologic and ophthalmologic data illustrate the methods. An online calculator is also provided for power analysis and risk difference testing.
The authors' abstract, as published at the source. Pharmaceutical Statistics, 2026 · DOI ↗
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Statistics, Probability and UncertaintyDecision Sciences