A robust likelihood approach to inference for paired multiple binary endpoints data

Tsung-Shan Tsou and Wei-Cheng Hsiao

Journal of Applied Statistics, 2024, vol. 51, issue 14, 2851-2865

Abstract: We introduce a robust likelihood approach to inference for paired multiple binary endpoints data. One can easily implement the methodology without dealing with the model that incorporates a large number of joint probabilities of no direct relevance to the inference of interest. We present the robust score test statistic for testing the equality of two treatment effects to exemplify the utility and simplicity of the method. Our novel technique is applicable when patients have different numbers of endpoints and for unpaired endpoints. The extension of our robust approach to multiple endpoints data with more categories is straightforward. We use simulations and real data analysis to highlight the efficacy of our robust procedure.

Date: 2024
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DOI: 10.1080/02664763.2024.2321904

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