组合单侧和双侧数据的适合度检验
摘要
涉及成对器官的临床试验常常产生单侧和双侧数据混合的情况,即每个受试者可能在特定情况下只贡献一个或两个响应。虽然来自不同个体的单侧响应可视为独立,但来自同一受试者的双侧响应可能存在相关性。已developed various statistical methods to account for this intra-subject correlation in the bilateral data, and in practice it is crucial to select an appropriate model for accurate inference. Tang et. al. (2012) discussed model selection issues using a variety of goodness-of-fit test statistics for correlated bilateral data for two groups, and Liu and Ma (2020) extended these methods to settings with groups. In this work, we investigate the goodness-of-fit statistics for the combined unilateral and bilateral data under different statistical models that address the intra-subject correlation, including the Clayton copula model, in addition to those considered in prior studies. Simulation results indicate that the performance of the goodness-of-fit tests is model-dependent, especially when the sample size is small and/or the intra-subject correlation is high, which is consistent with the findings by Liu and Ma (2020) for purely bilateral data. Applications to real data from otolaryngologic and ophthalmologic studies are included.
引用
@article{arxiv.2506.22605,
title = {Goodness-of-fit Tests for Combined Unilateral and Bilateral Data},
author = {Jia Zhou and Chang-Xing Ma},
journal= {arXiv preprint arXiv:2506.22605},
year = {2025}
}
备注
28 pages