Creating a multi-center rare disease consortium – the Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR)

Abstract

Eosinophilic gastrointestinal disorders (EGIDs) affect various segments of the gastrointestinal tract. Since these disorders are rare, collaboration is essential to enroll subjects in clinical studies and study the broader population. The Rare Diseases Clinical Research Network (RDCRN), a program of the National Center for Advancing Translational Sciences (NCATS), funded the Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR) in 2014 to advance the field of EGIDs. CEGIR facilitates collaboration among various centers, subspecialties, patients, professional organizations and patient-advocacy groups and includes 14 clinical sites. It has successfully initiated two large multi-center clinical studies looking to refine EGID diagnoses and management. Several pilot studies are underway that focus on various aspects of EGIDs including novel therapeutic interventions, diagnostic and monitoring methods, and the role of the microbiome in pathogenesis. CEGIR currently nurtures five physician-scholars through a career training development program and has published more than 40 manuscripts since its inception. This review focuses on CEGIR’s operating model and progress and how it facilitates a framework for exchange of ideas and stimulates research and innovation. This consortium provides a model for progress on other potential clinical areas.

Revisiting the NIH Taskforce on the Research needs of Eosinophil-Associated Diseases (RE-TREAD)

“…RDCRN has collaborated with PAGs and the pharmaceutical industry on government‐sponsored clinical research studies to be completed in a timely fashion and to bring novel therapeutics to market. An EAD‐associated registry was borne out of RDCRN through the CEGIR (U54 AI117804), an initiative of the Office of Rare Disease Research (ORDR) that is jointly funded by NCATS, NIAID, and NIDDK . This registry (part of a longitudinal observational study) is designed to understand the natural history of EGID and will help standardize histopathologic evaluation and assessment of these rare disorders.…”

A novel approach to conducting clinical trials in the community setting: utilizing patient-driven platforms and social media to drive web-based patient recruitment

“…This study presents one of the first formal applications of previous data to inform the theory based creation of a comprehensive approach to conducting Web-based direct-to-patient recruitment for research. The development of PRISM builds upon the positive findings in the TAPIR trial, seeks to avoid the challenges identified in the approach used for TAPIR, and advances innovative approaches to clinical research by: (1) expanding the use of social media and other online recruitment strategies through the use of novel technologies and marketing campaigns to target populations of patients with rare diseases previously unavailable to researchers [20][21][22][23][24]; (2) identifying and attempting to remove barriers to enrollment and participation in clinical research studies through web-based referral [53]; and (3) demonstrating the feasibility of the proposed methods for clinical research studies in rare disease populations.…”

“…A principal component of our strategy relied upon soliciting buy-in and commitment from PAG representatives to share and promote our recruitment content via their respective social media platform accounts in order to further promote our efforts. As previously reported by the RDCRN, PAGs act as research partners in ensuring the feasibility and success of various protocols via their collaboration with patient recruitment, support training programs, and study design [2,44]. PAG representatives were asked to provide what they felt would be the most important information to be considered about their respective target population, including the population's geographic characteristics, imagery or messaging that should be utilized or avoided, and most successful recruitment strategies to date.…”

“…While medical research continues to provide great benefit to society in terms of advancing knowledge and innovation, achieving target rates for study recruitment and accrual remains a challenge. Inability to reach eligible patients for recruitment ultimately reduces the statistical power of studies, incurs economic costs, and may jeopardize funding [1][2][3][4][5][6][7][8].…”

Increasing Rates of Diagnosis, Substantial Co-occurrence, and Variable Treatment Patterns of Eosinophilic Gastritis, Gastroenteritis and Colitis Based on 10 Year Data Across a Multi-Center Consortium

“…We conducted a retrospective study of subjects from multiple centers in the Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR) 19-20 , including pediatric and adult tertiary care centers in different geographical regions. Subjects were included from the University of Arkansas for Medical Sciences/Arkansas Children’s Hospital, the University of North Carolina, Children’s Hospital of Philadelphia, Cincinnati Children’s Hospital Medical Center, the University of Pennsylvania, and Children’s Hospital of Colorado.…”