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

J Appl Stat. 2024 Feb 27;51(14):2851-2865. doi: 10.1080/02664763.2024.2321904. eCollection 2024.

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.

Keywords: Paired data; fisher information; multiple endpoints; robust likelihood; score test.

Grants and funding

This work is supported by grant MOST110-2118-M-008-002-MY3 (Tsou) and MOST 111-2118-M-031-002 -(Hsiao) of National Science and Technology Council, Taiwan, R.O.C.