Project Summary

PCORI supports Patient-Powered Research Networks (PPRNs), communities of patients participating in clinical research, as part of the National Patient-Centered Clinical Research Network (PCORnet). In 2017, PCORI launched an initiative on Partnerships to Conduct Research within PCORnet to support the PPRNs in conducting comparative clinical effectiveness research on questions that are important to patients and other stakeholders. The initiative funded this project and others.

This research project is in progress. PCORI will post the research findings on this page within 90 days after the results are final.

What is the research about?

The immune system works to protect the body from infections. In inflammatory bowel disease, or IBD, the immune system attacks healthy cells by mistake. Crohn’s disease, or CD, and ulcerative colitis, or UC, are IBDs that cause long-term diarrhea and stomach pain. IBD can reduce quality of life and even lead to death. CD and UC affect more than 1.5 million Americans.

Biologics and small molecules are types of medicine that reduce inflammation. A group of biologics, called anti-TNFs, are the first option for treating moderate to severe CD and UC. But for up to 80 percent of patients, the medicine doesn’t work as well over time or stops working.

In this study, the research team is comparing how new biologic and small molecule treatments work for patients when anti-TNF medicines don’t work. The team is comparing a medicine called vedolizumab with another called ustekinumab for patients with CD. They are also comparing vedolizumab with a medicine called tofacitinib for patients with UC.

Who can this research help?

Results from this study can help patients who don’t respond to anti-TNFs and their doctors when considering treatment options.

What is the research team doing?

The research team is looking at data from adults age 18 or older. They are including at least 180 adults with CD and at least 180 adults with UC who are starting one of the newer medicines after not responding to an anti-TNF medicine. To identify patients, the team is working with 18 clinics across the country, health plans, a network of patients interested in research on IBD, and another research study. The team is following up with patients six months after they start their new medicine. The team is asking patients about their experiences of pain, fatigue, digestive symptoms, and other outcomes that matter to patients.

The research team is also looking at claims data for another group of patients who are starting one of the medicines after not responding to an anti-TNF medicine. Claims data is information from health insurance companies about the kinds of treatment a patient gets. It allows researchers to see how patients respond to treatment in the real world. The team is studying how long patients stay on their new medicine and whether they are hospitalized or have surgery over a one-year period.

Patients with CD and UC are working with the research team to plan the study.

Research methods at a glance

Design Elements Description
Design Aim 1: prospective cohort study
Aim 2: retrospective cohort study
Population Aim 1: adults with CD who are starting ustekinumab or vedolizumab after no response to an anti-TNF medicine
Aim 2: adults with UC who are starting tofacitinib or vedolizumab after no response to an anti-TNF medicine
Interventions/
Comparators
Aim 1:
  • Ustekinumab
  • Vedolizumab
Aim 2:
  • Tofacitinib
  • Vedolizumab
Outcomes Aim 1:
Primary: fatigue, pain interference
Secondary: disease activity and social satisfaction
Aim 2:
Persistence on therapy for at least 1 year, absence of hospitalization, absence of surgery
Timeframe Aim 1: 6-month follow-up for primary outcomes
Aim 2: At least 1-year follow-up from treatment initiation

Project Information

Michael Kappelman, MD, MPH^
The University of North Carolina at Chapel Hill^
$2,794,763
Comparative Effectiveness of Biologic or Small Molecule Therapies in IBD

Key Dates

April 2018
April 2023
2018

Study Registration Information

^Angela Dobes, MPH, was the original principal investigator on this project, and its original organization was the Crohn's & Colitis Foundation in New York.

Tags

Populations Populations PCORI is interested in research that seeks to better understand how different clinical and health system options work for different people. These populations are frequently studied in our portfolio or identified as being of interest by our stakeholders. View Glossary
Intervention Strategy Intervention Strategies PCORI funds comparative clinical effectiveness research (CER) studies that compare two or more options or approaches to health care, or that compare different ways of delivering or receiving care. View Glossary
State State The state where the project originates, or where the primary institution or organization is located. View Glossary
Last updated: March 4, 2022