The Patient-Centered Outcomes Research Institute (PCORI) has awarded $14.4 million to a UNC researcher to investigate novel treatment strategies for pediatric inflammatory bowel diseases (PIBD). Michael D. Kappelman, MD, MPH, professor of pediatrics and epidemiology at the UNC School of Medicine, will lead the study, which aims to generate real-world evidence on the effectiveness and safety of off-label medications for PIBD patients who have not responded to anti-TNF therapies.
Comparative Effectiveness Study
The study, titled "Comparative Effectiveness of Emerging Medications in Children with Inflammatory Bowel Disease," will utilize PCORnet, a national patient-centered clinical research network. Kappelman emphasized the high-impact potential of the project, stating, "By systematically studying how we care for children with PIBD today, we will determine how best to care for these patients in the future."
Collaboration with Duke Clinical Research Institute
The Duke Clinical Research Institute (DCRI) will serve as the Clinical Coordinating Center and Data Coordinating Center. Keith Marsolo, PhD, Laine Thomas, PhD, and Brian Smith, MD, will oversee participant recruitment and data collection across 30 sites participating in PCORnet, as well as the comprehensive analysis of study data. Marsolo noted the study's significance in generating real-world evidence to guide treatment decisions for children unresponsive to standard therapies. "By leveraging the power of PCORnet and collaborating with leading experts at UNC, we aim to provide clinicians, patients, and their families with the evidence they need to make more informed decisions and ultimately improve care and outcomes for the thousands of children affected by these chronic diseases," said Marsolo.
Addressing Unmet Needs in PIBD Treatment
Crohn’s disease and ulcerative colitis, common forms of PIBD, cause inflammation of the gastrointestinal tract, leading to abdominal pain, diarrhea, and growth delays in pediatric patients. While anti-TNF therapy is the only FDA-approved treatment class for these young patients, it often proves ineffective for up to half of those treated. Newer treatment options, though available, lack FDA approval for pediatric use and are primarily studied in adults. This study seeks to address the critical gap in evidence regarding the safety and effectiveness of these medications in children.
Study Design and Potential Impact
The study will follow PIBD patients treated with newer medications at 30 U.S. sites within PCORnet for up to three years. This will generate real-world evidence about the safety and comparative effectiveness of these treatments. Researchers believe the study has the potential to transform care for over 100,000 children in the U.S. living with PIBD. By providing evidence-based treatment decisions, the findings will reduce uncertainty and improve outcomes for patients facing limited options and treatment challenges.
