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Comparative effectiveness of biologic or small molecule therapies in inflammatory bowel disease (IBD)
Investigator (PI): Kappelman, Michael
Performing Organization (PO): (Current): University of North Carolina at Chapel Hill, School of Medicine, Department of Pediatrics, Division of Pediatric Gastroenterology / (919) 966-2435
Supporting Agency (SA): Patient-Centered Outcomes Research Institute (PCORI)
Initial Year: 2018
Final Year: 2023
Record Source/Award ID: PCORI/PaCR-2017C2-8172-IC
Funding: Total Award Amount: $2,435,558
Award Type: Contract
Award Information: PCORI: More information and project results (when completed)
Abstract: Background and significance: Crohn's disease (CD) and ulcerative colitis (UC), collectively referred to as inflammatory bowel disease (IBD), affect approximately 1.2 million individuals in the United States, cost over $6 billion annually, and cause substantial patient morbidity, missed work and school, and diminished quality of life. Currently, anti-TNF (tumor necrosis factor) therapy is considered first-line treatment for moderate to severe disease. Yet, up to 80% of patients do not respond or ultimately lose response to anti-TNF.?As the treatment armamentarium continues to grow, patients now have additional options to choose from. For patients with CD, these newer treatments include vedolizumab (alpha4 beta7 integrin antibody) and ustekinumab (anti-IL-12/23 antibody). For UC, new treatment options include vedolizumab or tofacitinib, a small molecule JAK (janus kinase) inhibitor that is anticipated to be approved by the Food and Drug Administration (FDA) to treat UC in March 2018. Unfortunately, anti-TNF refractory patients are often refractory to these newer treatments as well. Hence, selecting the most effective option is of paramount importance. Yet, there are no head-to-head studies to compare these options and help guide this common and important treatment decision. For this reason, the Institute of Medicine, the Crohn's & Colitis Foundation (the Foundation), and others have prioritized comparative effectiveness research that helps patients and their providers make more informed health choices. The overarching aim of this project is to compare the effectiveness of non-anti-TNF biologics and small molecules in patients who are refractory to anti-TNF therapy. To address this pressing need as quickly and efficiently as possible, we will leverage the research infrastructure that has been recently developed by PCORnet, the National Patient-Centered Clinical Research Network, and by IBD Plexus, the Foundation's new research exchange platform designed to accelerate inflammatory bowel disease (IBD) research and facilitate collaboration between stakeholders. Through the use of these varied sources of real-world data, our study findings will be broadly generalizable and directly inform these important patient treatment decisions. Study aims: Aim 1 is to compare the effectiveness of second-line biologic agents (ustekinumab versus vedolizumab) among patients with Crohn's disease (CD) who are anti-TNF primary or secondary non-responders. Aim 2 is to compare the effectiveness of a second-line biologic agent (vedolizumab) versus small molecule (tofacitinib) among patients with ulcerative colitis (UC) who are anti-TNF primary or secondary non-responders. Study description: Each aim will be addressed by two approaches. First, we will recruit 180 patients with each condition who are starting one of the treatments we are comparing. Participants will come from approximately 18 study centers across the country. Participants may also be referred by our partner health plans, Anthem Blue Cross Blue Shield and Humana, or join the study directly through our Patient Powered Research Network (www.ccfapartners.org). The study will focus on patient-reported outcomes including pain, fatigue, and other symptoms that will be collected through electronic surveys. The second approach will leverage real-world data collected by our health plan partners, including claims for office visits, hospital visits, medications, and other health care encounters. We will use this data to compare these treatment approaches focusing on outcomes such as length of time patients remain on treatment and the ability to prevent hospitalization and surgery. We will engage patients and other stakeholders at each phase of the study from conception and planning through analysis and dissemination. This will ensure that the results of this project provide patients and their physicians strong evidence that will directly inform this important treatment decision. Completion of these aims will not only answer two high-priority, patient-centered comparative effectiveness research questions, but will also leverage the existing research infrastructure of PCORnet and the Crohn's & Colitis Foundation and forge multi-stakeholder partnerships, contributing to the sustainability of both organizations.
MeSH Terms:
  • Biologics /*therapeutic use
  • Comparative Effectiveness Research
  • Decision Making
  • Food and Drug Administration
  • Foundations
  • Humans
  • Multicenter Studies as Topic
  • Outcome Assessment, Health Care
  • Patient-Centered Care
  • Quality of Life
  • Tumor Necrosis Factor /*antagonists & inhibitors
  • United States
Country: United States
State: North Carolina
Zip Code: 27599
UI: 20192432
Project Status: Ongoing