April 24, 2015
Three University of North Carolina School of Medicine faculty have been awarded contracts from the Patient-Centered Outcomes Research Institute (PCORI) to fund patient-centered clinical comparative clinical effectiveness research (CER) studies.
The largest of the three contacts, a five-year award totaling nearly $8 million, was awarded to Michael Kappelman, MD, MPH. It aims to answer one of the most pressing questions parents of children diagnosed with Crohn’s disease face: which treatment will be most effective and cause the least side effects?
Kelli Allen, PhD, research professor in the UNC Thurston Arthritis Research Center, was awarded a two-year, $1.9 million contract for a study aimed at developing and disseminating an evidence-based pain coping skills training (CST) intervention among African Americans with osteoarthritis to reduce disparities in outcomes.
Donna M. Evon, PhD, assistant professor in the Division of Gastroenterology and Hepatology and the UNC Liver Center, was awarded a three-year, $2.4 million contract for a study aimed at helping patients with chronic hepatitis C viral infection (HCV) and their providers make more-informed treatment decisions. This study to compare two new all-pill treatments is the first head-to-head study of short-term and longer-term outcomes that were selected by patients with HCV, and will matter most to patients making future decisions about HCV treatment.
Crohn’s disease is an inflammatory bowel disease that affects approximately 38,000 children in the U.S., causing debilitating abdominal pain and bloody diarrhea and leading to frequent hospitalizations and surgery.
“Treatment typically involves suppressing inflammation with drug therapies, balancing potential benefits against risks that can be life threatening,” explained the study’s principal investigator, Dr. Kappelman, an associate professor of pediatrics in the UNC School of Medicine. “Parents turn to their child’s gastroenterologist to guide them in health decisions, but there are gaps in research where we can’t definitively say which therapy is better.”
One of those “gaps in research” motivated Kappelman to apply for funding through PCORI’s Pragmatic Clinical Studies Initiative. Anti-tumor necrosis factor, or anti-TNF, drugs are a class of drugs that has been used for more than a decade in the treatment of inflammatory diseases like rheumatoid arthritis. More recently, they have had a major impact in the treatment of Crohn’s disease. What remains unknown, however, is whether using anti-TNF drugs alone or in combination with methotrexate, another immune suppressant, leads to better outcomes.
The multi-disciplinary research team, which includes investigators from more than 50 pediatric gastroenterology programs across the country, will recruit participants through ImproveCareNow, a network of more 575 gastroenterologists in 71 practices. They will enlist 425 individuals under 18 years old with moderate to severe Crohn’s disease who are initiating anti-TNF therapy. Participants in the double-blind study will be randomized to receive either anti-TNF plus low dose oral methotrexate (combination therapy) or anti-TNF alone (monotherapy), and they will be followed for two years.
“This study will address one of the most challenging treatment decisions faced by patients with pediatric Crohn's disease, their families, and their physicians,” said Dr. Kappelman. “It presents a tremendous opportunity to generate strong scientific knowledge that will directly inform and impact patient care—and for the pediatric gastroenterology community to further integrate research and clinical care so that we can learn from each and every patient encounter.”
Unique among clinical trials, parents and patients will be involved in all aspects of planning and conducting the trial and will also guide the development of recruitment materials and the informed consent process.
“Time and again, basic questions about children’s illnesses are understudied,” said David Wohl, MD, a UNC physician and Crohn’s disease father who will lead the study’s parent engagement core. “This trial represents an unprecedented collaboration between researchers and parents as partners. As the parent of a child who struggles with Crohn’s disease, I am excited to see this study launched and hope parents at UNC and the other centers will help us learn what works best for our kids.”
PCORI’s Pragmatic Clinical Studies Initiative is 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. Pragmatic clinical studies test a treatment’s effectiveness in “real-life” practice situations, such as typical hospitals and outpatient clinics, and can include a wider range of study participants, making their findings more generally applicable.
“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 fill a crucial evidence gap and answer an important question about the treatment of pediatric Crohn’s disease,” said Joe Selby, MD, MPH, executive director of PCORI. “We look forward to following the study’s progress and working with UNC to share its results.”
UNC’s award has been approved pending completion of a business and programmatic review by PCORI staff and issuance of a formal award contract.
About the Patient-Center Outcomes Research Institute
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 at pcori.org.