The Patient-Centered Outcomes Research Institute (PCORI) announced the approval of funding totaling $14.4 million to study new treatment options for pediatric inflammatory bowel diseases (PIBD). The study, “Comparative Effectiveness of Emerging Medications in Children with Inflammatory Bowel Disease,” led by Michael D. Kappelman, MD, MPH, professor of pediatrics and epidemiology at the UNC School of Medicine, will leverage PCORnet® to generate real-world evidence to compare the clinical effectiveness and safety of off-label medications in patients with PIBD for whom anti-TNF treatments are ineffective.
“This will be a really exciting project with high impact,” said Kappelman, who is the principal investigator. “By systematically studying how we care for children with PIBD today, we will determine how best to care for these patients in the future.”
The Duke Clinical Research Institute (DCRI) will serve as the Clinical Coordinating Center and Data Coordinating Center, with Keith Marsolo, PhD, Laine Thomas, PhD, and Brian Smith, MD, overseeing participant recruitment and data collection across 30 sites participating in PCORnet, as well as the comprehensive analysis of study data.
“This study represents a pivotal step forward in PIBD research, as we work to generate much-needed real-world evidence that can guide treatment decisions for children whose condition does not respond to standard therapies,” said Marsolo, associate professor in Population Health Sciences at Duke University. “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.”
Crohn’s disease and ulcerative colitis are two common chronic inflammatory bowel diseases that can inflame the lining of the gastrointestinal tract and cause significant suffering in pediatric patients, including debilitating abdominal pain and diarrhea, impact on psychosocial functioning, and delays in growth or puberty. Despite the burden of these conditions, treatment options for children with moderate to severe PIBD remain limited. Currently, the only FDA-approved treatment class for these young patients is anti-TNF therapy, which often proves ineffective for up to half of those treated, leading to a return of symptoms over time.
While newer treatment options exist, they have primarily been studied in adults and lack FDA-approval for pediatric use. Nevertheless, these medications are often prescribed to treat children with PIBD who do not respond to anti-TNF therapy. Thus, the absence of robust, objective evidence, about their safety and effectiveness in children is a critical gap in the literature.
To address this gap, the current study will follow patients with PIBD treated with these newer medications at 30 U.S. sites participating in PCORnet for up to three years, generating much needed real-world evidence about the safety and comparative effectiveness of these treatments. PCORnet is a national resource, funded by PCORI, that enables insights from high quality health data, patient partnership, and research expertise to deliver fast, trustworthy answers that advance health outcomes. With connections to data from everyday healthcare encounters with more than 47 million people across the U.S. each year, PCORnet offers the study team a powerful platform for understanding the real-world effectiveness and safety of PIBD medications.
Researchers say this study has the potential to transform care for the over 100,000 children in the U.S. living with PIBD, helping clinicians and families make more informed, evidence-based treatment decisions for patients who have not responded to first-line therapies. By filling a critical knowledge gap, the findings will guide treatment decisions, reduce uncertainty, and ultimately improve outcomes for PIBD patients who currently face limited options and significant treatment challenges.
