Research Project
10242197
Project Summary/Abstract Clinical trials are the highest standard of evidence for the effectiveness and safety of medical interventions. Unfortunately, children often receive medical therapies that have never been studied in clinical trials involving pediatric subjects. Children often respond differently from adults to medications due to differences in physiology and drug metabolism, placing children at increased risk of experiencing adverse, even life- threatening events from off-label use of medical treatments. Additionally, one-fifth of US children suffer from at least one chronic disease and face the risks of receiving treatments based on scant data. Children from rural and medically underserved communities bear a disproportionately large risk of not benefiting from medical treatments as researchers often fail to engage individuals from these communities in research. However, when asked, children enrolled in clinical trials state that their participation provided them with access to more treatment options, improved their understanding of their disease, and provided them with the opportunity to help other people. Since 2016 the IDeA States Pediatric Clinical Trials Network (ISPCTN) has engaged children, families, and health care providers from rural and medically underserved communities in high-quality pediatric clinical trials. The Nebraska Pediatric Clinical Trials Unit (NPCTU) plays a leading role in defining and ensuring best practices in clinical trials design, initiation, and conduct within the ISPCTN and serves as a transformative resource for pediatric research in Nebraska and neighboring IDeA states. The objective of the current application is to further extend clinical trials participation opportunities and infrastructure to rural and underserved communities across Nebraska. The NPCTU team will achieve this objective by expanding institutional and community partnerships regionally and nationally. Specifically, we will increase rural engagement in clinical trials through partnership with researchers at the University of Nebraska at Kearney and health care providers, children, and families at the Children?s Physicians Kearney clinic, and through regular communication with a rural community advisory board. In partnership with the Great Plains IDeA-CTR and other ISPCTN sites regionally we will train pediatric health care providers and research staff to participate in ISPCTN-approved clinical trials and provide junior faculty with mentorship and training that enables them to advance their careers as researchers in child health. Finally, as a core resource within the Child Health Research Institute, the NPCTU will help increase research infrastructure access and utilization across the University of Nebraska Medical Center and Children?s Hospital & Medical Center. The NPCTU administrative team will measure and track key performance indicators of clinical trials engagement, conduct, and capacity- building using metrics organized according to the RE-AIM framework. Renewing the NPCTU will: 1) enhance access to innovative medical therapies for Nebraska children; and 2) improve workforce capacity in Nebraska to conduct clinical trials, which will provide better health care options for pediatric patients.
