Pragmatic Statistical Approaches for Power Analysis, Causal Inference, and Biomarker Detection
Creators: name: Wu, Fan
Publisher: name: Purdue University Graduate School
Year: 2023
Subjects: Statistical data science

description: Mediation analyses play a critical role in social and personality psychology research. However, current approaches for assessing power and sample size in mediation models have limitations, particularly when dealing with complex mediation models and multiple mediator sequential models. These limitations stem from limited software options and the substantial computational time required. In this part, we address these challenges by extending the joint significance test and product of coefficients test to incorporate the fourth-pathed mediated effect and generalized kth-pathed mediated effect. Additionally, we propose a model-based bootstrap method and provide convenient R tools for estimating power in complex mediation models. Through our research, we demonstrate that power decreases as the number of mediators increases and as the influence of coefficients varies. We summarize our results and discuss the implications of power analysis in relation to mediator complexity and coefficient influence. We provide insights for researchers seeking to optimize study designs and enhance the reliability of their findings in complex mediation models. Matching is a crucial step in causal inference, as it allows for more robust and reasonable analyses by creating better-matched pairs. However, in real-world scenarios, data are often collected and stored by different local institutions or separate departments, posing challenges for effective matching due to data fragmentation. Additionally, the harmonization of such data needs to prioritize privacy preservation. In this part, we propose a new hierarchical framework that addresses these challenges by implementing differential privacy on raw data to protect sensitive information while maintaining data utility. We also design a data access control system with three different access levels for designers based on their roles, ensuring secure and controlled access to the matched datasets. Simulation studies and analyses of datasets from the 2017 Atlantic Causal Inference Conference Data Challenge are conducted to showcase the flexibility and utility of our framework. Through this research, we contribute to the advancement of statistical methodologies in matching and privacy-preserving data analysis, offering a practical solution for data integration and privacy protection in causal inference studies. Biomarker discovery is a complex and resource-intensive process, encompassing discovery, qualification, verification, and validation stages prior to clinical evaluation. Streamlining this process by efficiently identifying relevant biomarkers in the discovery phase holds immense value. In this part, we present a likelihood ratio-based approach to accurately identify truly relevant protein markers in discovery studies. Leveraging the observation of unimodal underlying distributions of expression profiles for irrelevant markers, our method demonstrates promising performance when evaluated on real experimental data. Additionally, to address non-normal scenarios, we introduce a kernel ratio-based approach, which we evaluate using non-normal simulation settings. Through extensive simulations, we observe the high effectiveness of the kernel method in discovering the set of truly relevant markers, resulting in precise biomarker identifications with elevated sensitivity and a low empirical false discovery rate.