Michael D. Kappelman, MD, MPH of the University of North Carolina at Chapel Hill (read the UNC press release here) and the ImproveCareNow Network has been awarded $7.9 million in funding from the Patient Centered Outcomes Research Institute (PCORI) to conduct a five year study comparing Anti-TNF Monotherapy versus Combination Therapy with Low Dose Methotrexate in Pediatric Crohn’s Disease.

The ImproveCareNow Network will serve as hub for this large pragmatic clinical trial, which aims to compare combination versus monotherapy with biologic medications. “This is an important day for children with IBD. ImproveCareNow will address one of the highest priority questions faced by patients, families and clinicians with the disease.” said Scientific Director, Peter Margolis, MD, PhD.

“This will be a tremendous opportunity to generate strong scientific knowledge that will directly inform and impact patient care.  This study will address one of the most challenging treatment decisions faced by patients with pediatric Crohn's disease, their families, and their physicians. It is a wonderful opportunity for the pediatric gastroenterology community to further integrate research and clinical care, so that we can learn from each and every patient encounter.”  

- Michael D. Kappelman, MD, MPH, Principal Investigator 

“This award is significant is so many ways. Foremost, it addresses a huge knowledge gap in the treatment of this dreadful disease in children - time and again, basic questions about children’s illnesses are understudied. This also represents an unprecedented collaboration between researchers and parents as partners. As a parent of a child who has to struggle with IBD, I am excited to see this study launched and hope other parents here and at other centers will help us learn what works best for our kids.”

- David A. Wohl, MD, Parent Partner

 “This project was selected for PCORI funding not only for its scientific merit and commitment to engaging patients and other healthcare stakeholders in a major study conducted in real-world settings, but also for its potential to answer an important question about pediatric Crohn’s disease and fill a crucial evidence gap,” said PCORI Executive Director Joe Selby, MD, MPH. “We look forward to following the study’s progress and working with Dr. Kappelman, the University of North Carolina at Chapel Hill and the ImproveCareNow Network to share its results.”

Dr. Kappelman’s study was selected for funding through PCORI’s Pragmatic Clinical Studies Initiative, an effort to produce results that are broadly applicable to a diverse range of patients and care situations and can be more quickly taken up in routine clinical practice. Many clinical studies test whether a treatment works under optimized conditions in specialized research centers, but health care is rarely delivered in such idealized situations and settings. Pragmatic clinical studies test a treatment’s effectiveness in “real-life” practice situations, such as typical hospitals and outpatient clinics, and also can include a wider range of study participants, making their findings more generally applicable.

This study and the other projects approved for PCORI funding were selected through a highly competitive review process in which patients, caregivers, and other stakeholders joined scientists to evaluate the proposals. Applications were assessed for scientific merit, how well they will engage patients and other stakeholders, and their methodological rigor among other criteria.

The award has been approved pending completion of a business and programmatic review by PCORI staff and issuance of a formal award contract.

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PCORI is an independent, nonprofit organization authorized by Congress in 2010. Its mission is to fund research that will provide patients, their caregivers, and clinicians with the evidence-based information needed to make better-informed healthcare decisions. For more information about PCORI’s funding awards, visit the Research and Results page on www.pcori.org.

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