Background: Necrotizing enterocolitis (NEC) is a multifactorial intestinal disease that causes 1 in 10 deaths in US NICUs. NEC affects medically fragile babies in their first weeks and months of life. Prevention of NEC has been neglected for decades. The disease is often regarded as inevitable. NEC awareness, resources, and research are inadequate. Without sufficient research to compare existing treatments to guide standards of care, or support guidelines on how to effectively partner with parents of vulnerable infants, or how to best prevent and treat NEC, patient-families receive variable care and often feel disempowered. Currently, NEC research lacks a patient-family voice.

Proposed Solution: The NEC Society is a 501(c)(3) nonprofit organization that brings together diverse stakeholders committed to building a world without NEC. This capacity-building project will prepare the NEC Society to drive patient-centered research (PCOR) and comparative clinical effectiveness research (CER) in the NEC community and serve as a model for neonatal rare disease communities. The NEC Society’s patient-families and clinician-researchers will pursue intensive training to learn how to lead and effectively participate in patient-centered research. These stakeholders will develop a process to identify, prioritize, design, implement, evaluate, and disseminate PCOR/CER. Patient-families will be fully integrated throughout the entire process.

Objectives:

• Create and train a dedicated PCOR team, consisting of NEC patient-families and clinician-researchers.
• Develop methods and strategies to determine and drive priorities for PCOR research in the NEC community.
• Establish a model framework for patient-centered research in the neonatal rare disease community, which can be disseminated to and used by other diverse groups and stakeholders.

Activities: The NEC Society will work with the UC Davis Center for Healthcare Policy and Research (CHPR), which is an interdisciplinary research unit that leads community-based participatory research, PCOR, and CER. CHPR will provide tailored mentorship and training for stakeholders, including authentic engagement, qualitative research methods, study design, data collection, analysis, and dissemination. CHPR will provide practical strategies for strengthening and sustaining patient-family and clinician-researcher engagement, as well as create a curriculum with topics including optimizing collaboration, developing PCOR questions, selecting appropriate research methods, assessing and ensuring rigor, and research ethics. The project will be evaluated and the results disseminated.

Outcomes: This project’s long-term outcomes and impact include the NEC Society driving patient-centered research in the NEC community, serving as a leader in PCOR for other neonatal rare disease communities, and fundamentally improving how medically fragile babies and their families experience care in the NICU, leading to groundbreaking new research and best practices.

Engagement Plan: The NEC Society will recruit patient-families and clinician-researchers to serve on the project’s NEC PCOR Leadership Team, with an emphasis on diversity. The NEC PCOR team will meet in person and virtually as they participate in training, identify tools, develop infrastructure, create a PCOR/CER research agenda, disseminate findings, and create a model for neonatal rare disease communities. Patient-families will be fully integrated.

Project Collaborators: The NEC Society will continue to collaborate with international NEC partners, including the United Kingdom’s NEC charities, Brazil’s Instituto Pequenos Grandes Guerreiros, and Australia’s NEC Alliance.