Statistical inference on qualitative differences in the magnitude of an effect

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Main Authors: Aaron Hudson, Ali Shojaie
Format: Artículo Open Access
Published: Wiley 2024
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author Aaron Hudson
Ali Shojaie
author_facet Aaron Hudson
Ali Shojaie
Aaron Hudson
Ali Shojaie
collection Wiley Open Access
contents Statistical inference on qualitative differences in the magnitude of an effect Aaron Hudson Ali Shojaie Statistics in Medicine Qualitative interactions occur when a treatment effect or measure of association varies in sign by sub‐population. Of particular interest in many biomedical settings are absence/presence qualitative interactions, which occur when an effect is present in one sub‐population but absent in another. Absence/presence interactions arise in emerging applications in precision medicine, where the objective is to identify a set of predictive biomarkers that have prognostic value for clinical outcomes in some sub‐population but not others. They also arise naturally in gene regulatory network inference, where the goal is to identify differences in networks corresponding to diseased and healthy individuals, or to different subtypes of disease; such differences lead to identification of network‐based biomarkers for diseases. In this paper, we argue that while the absence/presence hypothesis is important, developing a statistical test for this hypothesis is an intractable problem. To overcome this challenge, we approximate the problem in a novel inference framework. In particular, we propose to make inferences about absence/presence interactions by quantifying the relative difference in effect size, reasoning that when the relative difference is large, an absence/presence interaction occurs. The proposed methodology is illustrated through a simulation study as well as an analysis of breast cancer data from the Cancer Genome Atlas. 10.1002/sim.10025 http://onlinelibrary.wiley.com/termsAndConditions#am
doi_str_mv 10.1002/sim.10025
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spellingShingle Statistical inference on qualitative differences in the magnitude of an effect
Aaron Hudson
Ali Shojaie
Statistics in Medicine
Statistical inference on qualitative differences in the magnitude of an effect Aaron Hudson Ali Shojaie Statistics in Medicine Qualitative interactions occur when a treatment effect or measure of association varies in sign by sub‐population. Of particular interest in many biomedical settings are absence/presence qualitative interactions, which occur when an effect is present in one sub‐population but absent in another. Absence/presence interactions arise in emerging applications in precision medicine, where the objective is to identify a set of predictive biomarkers that have prognostic value for clinical outcomes in some sub‐population but not others. They also arise naturally in gene regulatory network inference, where the goal is to identify differences in networks corresponding to diseased and healthy individuals, or to different subtypes of disease; such differences lead to identification of network‐based biomarkers for diseases. In this paper, we argue that while the absence/presence hypothesis is important, developing a statistical test for this hypothesis is an intractable problem. To overcome this challenge, we approximate the problem in a novel inference framework. In particular, we propose to make inferences about absence/presence interactions by quantifying the relative difference in effect size, reasoning that when the relative difference is large, an absence/presence interaction occurs. The proposed methodology is illustrated through a simulation study as well as an analysis of breast cancer data from the Cancer Genome Atlas. 10.1002/sim.10025 http://onlinelibrary.wiley.com/termsAndConditions#am
title Statistical inference on qualitative differences in the magnitude of an effect
topic Statistics in Medicine
url https://onlinelibrary.wiley.com/doi/10.1002/sim.10025