Landmark Study to Compare the Effectiveness of Biologic or Small Molecule Therapies in Inflammatory Bowel Disease (IBD) Receives Funding
Published: May 17, 2018
New York, NY – Crohn’s disease (CD) and ulcerative colitis (UC), collectively referred to as inflammatory bowel disease (IBD), which may affect as many as 3 million Americans, cost over $6 billion annually, and cause substantial patient morbidity, missed work and school, and diminished quality of life. Currently, anti-TNF therapy is considered first line treatment for moderate-to-severe IBD. However, up to 80 percent of patients do not respond or, ultimately, lose response to anti-TNF. New treatments provide additional options from which to choose, however, there are no head-to-head studies that compare these new treatments.
The Crohn’s & Colitis Foundation (Foundation) is a leader in a $2.4 million funding award recently approved by the Patient-Centered Outcomes Research Institute (PCORI) to study comparative effectiveness of biologic or small molecule therapies in inflammatory bowel diseases. Leveraging the research infrastructure developed by PCORnet, the National Patient-Centered Clinical Research Network, and by IBD Plexus, the Foundation’s new research exchange platform, this project will compare the effectiveness of these newer treatment options, providing needed data that will help patients and physicians make more informed healthcare decisions.
“Selecting the most effective treatment is incredibly important to our patients,” said Angela Dobes, the Foundation’s senior director of IBD Plexus and one of the study’s principal investigators. “This study will engage key stakeholders, most importantly patients, at each phase to ensure that the results provide patients, and their physicians, with strong evidence to support important treatment decisions.”
The aims of the study are to compare the effectiveness of newer biologic agents (ustekinumab versus vedolizumab) among patients with Crohn’s disease who are anti-TNF primary or secondary non-responders; and to compare the effectiveness of vedolizumab versus a small molecule (tofacitinib) among anti-TNF refractory ulcerative colitis patients.
The study will recruit 180 patients across the country with each condition who are starting one of the treatments being compared. Participants may also be referred by partner health plans, Anthem Blue Cross Blue Shield and Humana, or join the study directly through IBD Partners, the Foundation’s patient-powered research network (www.ibdpartners.org). The study will focus on patient-reported outcomes including pain, fatigue, and other symptoms, collected through electronic surveys. The study will also analyze the real-world data collected on thousands of patients by the health plan partners to further compare the effectiveness of these agents.
“This study focuses on outcomes that really matter to patients, including various aspects related to quality of life. We will also evaluate the ability to prevent hospitalization and surgery, and the length of time patients remain on treatment,” said the study’s principal investigator Michael Kappelman, MD, MPH, Professor at the University of North Carolina at Chapel Hill and a co-principal investigator of IBD Partners. “Using varied real-world data sources, our study findings will be broadly generalizable and directly inform these important patient treatment decisions.”
“This project was selected for PCORI funding not only for its scientific merit and commitment to engaging patients and other stakeholders, but also for its potential to improve our nation’s capacity to conduct clinical research more efficiently and to answer important questions that patients and clinicians face,” said PCORI Executive Director Joe Selby, MD, MPH. “We look forward to following the study’s progress and working with the Foundation to share the results.”
This study was selected for PCORI funding through a highly competitive review process in which patients, clinicians, and other stakeholders joined clinical 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.
About the Patient-Centered 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, visit www.pcori.org.
PCORnet is an innovative initiative funded by PCORI that is designed to make it faster, easier, and less costly to conduct clinical research than is now possible. It is doing this by harnessing the power of large amounts of health data and patient partnerships. In the process, it is transforming the culture of clinical research from one directed by researchers to one driven by the needs of patients and those who care for them.
Crohn's & Colitis Foundation
The Crohn's & Colitis Foundation is a non-profit, volunteer-fueled organization dedicated to finding the cures for Crohn's disease and ulcerative colitis, and to improving the quality of life of children and adults affected by these diseases. It was founded in 1967 by Irwin M. and Suzanne Rosenthal, William D. and Shelby Modell, and Henry D. Janowitz, M.D.