Clinical Research in Real-World Settings
Randomized controlled trials—in which participants are randomly assigned to either receive a treatment or not—are considered the gold standard of scientific studies.
While such trials are necessary, they can be very expensive and take a long time to complete, plus it can be challenging to find enough people to take part in them. What’s more, entrance criteria are often highly restrictive, so participants in the study often end up being very similar to each other.
There is another kind of research that’s often easier and more cost-effective to conduct: research done in real-world settings. Called pragmatic research, this type of research can help fill important knowledge gaps and begin to answer key questions not addressed in randomized controlled trials.
In pragmatic research, patients still provide informed consent to agree to participate in the study. Often, clinicians collect data for these studies during routine appointments with the patients who have agreed to participate. This allows them to collect data on a broad range of topics and examine trends in treatment. This type of research may also be used after a clinical trial to evaluate how well various approaches work in the real world.
Other types of pragmatic research rely on evaluating insurance claims data or other large data sets; in those instances, patient identification has been removed.
The Foundation is currently prioritizing pragmatic research to fill in knowledge gaps in several areas, including figuring out how many people have IBD, finding predictive factors to determine which patients will respond or not respond to different types of treatments, and developing standardized questionnaires to use in IBD surgical practice.
Determining IBD Prevalence
A groundbreaking study, the Diversity within the INcidence, Prevalence, Treatment, and OUTcomes in Patients with IBD (INPUT), funded by the Centers for Disease Control and Prevention led by the Crohn’s & Colitis Foundation estimates the incidence, prevalence, and racial-ethnic distribution of physician-diagnosed IBD in the United States, using comprehensive health insurance claims data. The study, conducted by principal investigators at the Foundation, the University of Pennsylvania, University of North Carolina at Chapel Hill, and Carelon Research, finds that IBD is diagnosed in more than 0.7% of Americans, with 721 cases per 100,000 people, or nearly 1 in 100. This study is unique in having pulled data that are representative of nearly the entire U.S. population with health insurance.
Importantly, the study found significant differences in prevalence among different racial groups, with Whites having a rate of IBD that is 7 times higher than Blacks, 6 times higher than Hispanics, and 21 times higher than Asian Americans. More research is needed to understand the reasons for the racial-ethnic differences in IBD prevalence.
Developing Standardized Questionnaires
Nearly 30% of patients with ulcerative colitis (UC) will have surgery during their lifetime. The most common type of surgery for patients with UC is proctocolectomy with ileal pouch-anal anastomosis, which is more commonly known as J-pouch surgery.
While this type of surgery can help improve disease symptoms, many patients will develop new symptoms or experience complications from their J-pouch. To date, bowel function and other outcomes post-J-pouch surgery have been studied, but there is no standardized assessment of symptoms or complications. The Foundation is supporting the PROPS study (Patient Reported Outcomes after Pouch Surgery) to develop and validate a standardized patient questionnaire that can be used in clinical practice to more easily and accurately assess a patient’s bowel function and other outcomes post-surgery.
