Q4 – 2025 Research Roundup
We continue to proudly highlight the outstanding work of our faculty who have received external funding support. Their dedication and success are a testament to the strength and impact of our academic community. Below lists a snapshot of those endeavors.
Title: Trial of Sequential Medications AfteR TNF failure in JIA (SMART-JIA)
Amount: $57,193
Funding Agency: Duke University
Period: 6/1/2024-11/30/2025
Abstract: Despite a 30% non-response rate to TNFi, there are currently no data to guide the next step of therapeutic decisions, making this study absolutely critical and timely to patients with JIA and their families. The study will require a global collaborative effort, and NC State will be part of this highly productive and engaged multidisciplinary study team, including physicians and parent partners who have cared for patients with JIA. Each member of the team will contribute a unique and valuable perspective, informed by personal and professional experience that will prove indispensable in helping generate the knowledge that will best serve parents and patients with JIA who need to make decisions about how to manage their disease.
Title: Spatial and Tensor Methods for Estimating the Health Effects of PFAS Mixtures
Amount: $474,639
Funding Agency: National Institutes of Health (NIH)
Period: 6/10/2025-3/30/2029
Abstract: Perfluoroalkyl and Polyfluoroalkyl Substances (PFAS) are synthetic compounds found in the environment and linked to adverse health outcomes. However, understanding the causal effects of PFAS mixtures on various health outcomes remains limited. This project addresses this critical knowledge gap by employing innovative spatial and high-dimensional causal inference methods to enhance our understanding of PFAS health effects. By bringing scientific rigor to observational studies, the project aims to overcome the challenges posed by spatial dependence, various sources of bias, and high dimensionality in PFAS studies.
This project leverages two valuable health outcomes data sources relating PFAS in drinking water: (i) a nationwide cohort of Medicare beneficiary data, and (ii) a North Carolina electronic health records cohort. These cohorts have been linked to PFAS concentrations in the ground water sources and in the tap water used for drinking. These data of health outcomes contain individual-level information and provide comprehensive resources for enhancing the understanding of the effects of PFAS on population health. Based on previous epidemiological analysis of associations with PFAS exposure in the two cohorts we have identified three statistical challenges. First, spatially-dependent outcomes violate standard assumptions of causal inference, necessitating a rethinking of the potential outcomes framework. Second, various sources of bias are present such as preferential sampling and non-random actualization of PFAS exposures. Third, the high dimensionality of the PFAS chemical mixture and health responses introduces computational and stability issues, requiring new dimension-reduction methods. By developing a suite of methods tailored to address these challenges, this project marks a significant advancement in the field of epidemiological research.
Title: Enhancing Long-Term Treatment Effect Estimation Through External Control Borrowing
Amount: $145,117
Funding Agency: Eli Lilly and Co.
Period: 8/16/2024-12/31/2025
Abstract: This funding will be to support a collaboration between Eli Lilly’s Real-world analytics team and North Carolina State University (NCSU) Department of Statistics to compare and develop methodology to integrate randomized placebo-controlled trials, single-arm extension trials, and real-world placebo or historical trial information to estimate long-term treatment effects. The project will review existing approaches for constructing synthetic control arms while mitigating biases from differences in population characteristics and outcome progression and extend existing approaches to the scenario where “short-term” outcomes are available for the RCT control arm, but “long-term” outcomes are missing. The operating characteristics of approaches to impute the hypothetical outcomes for RCT control subjects in the extension period will also be evaluated.
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