Inflammatory Bowel Disease Patients’ Perspectives of Clinical Trials: A Global Quantitative and Qualitative Analysis

Abstract

Background Despite recent progress, inflammatory bowel disease (IBD) therapies with pronounced long-term efficacy and improved safety are needed. IBD clinical trials face challenges with patient recruitment because of study designs, competitive or overlapping trials, and limited numbers of eligible patients. We aimed to better understand patients’ awareness of, attitudes toward, and experience with IBD clinical trials. Methods This multinational, cross-sectional cohort study of adults with IBD recruited online consisted of 2 components: a quantitative 15-minute online survey completed by all participants and a qualitative 30-minute telephone interview completed by a subset of patients from the United States. Results Quantitative survey respondents (N=226) included patients with ulcerative colitis (52%) and Crohn’s disease (48%) from the United States (n=100, 21 of whom were interviewed), Brazil (n=26), Canada (n=25), France (n=25), Germany (n=25), and Spain (n=25); 96% of respondents reported at least a basic understanding of clinical trials. Patients rated conversations with health care providers most helpful for researching trials, but during interviews patients discussed their desire for increased patient–physician communication about trials. Major barriers to participation included invasive screening/monitoring (35% of quantitative responses) and concern over receiving placebo (35%) or suboptimal treatment (33%). Most respondents (68%) reported that clinical trial participants are “guinea pigs” for an experimental treatment. Conclusions Opportunities to improve participation in IBD trials include improved communication with health care providers, further patient education, and alternative trial designs. Ultimately, a better understanding of the patient perspective will be important for more informed patients and more successful recruitment and enrollment.

Crossing barriers: the burden of inflammatory bowel disease across Western Europe

“…87 Consequently, the failure to recruit patients quickly and/or in sufficient numbers is a leading cause of clinical trial discontinuation with concerns with the ability to deliver future trials in IBD. 86,88 Rubin et al 88 demonstrated that patients identified healthcare providers as the most helpful resource for researching clinical trials. The study further stated that physicians rarely initiate conversations about clinical trials and patients typically do not ask.…”

Editorial: shedding new light on extraintestinal manifestations in inflammatory bowel disease—authors’ reply

“…Based on the costs and barriers associated with clinical trial design, we suspect that much of the future data in this space will be derived from observational studies or investigator initiated clinical studies. 4,5 This explains the focus of our work to strengthen the quality of observational assessment of EIMs. Our modified Delphi consensus panel provides guidance for the practicing clinician while building a robust framework for conducting observational studies in this space.…”

“…In addition, operationalizing required subspecialty involvement with EIM specific follow‐up in clinical trials focused on luminal disease is possible, but not trivial. Based on the costs and barriers associated with clinical trial design, we suspect that much of the future data in this space will be derived from observational studies or investigator initiated clinical studies 4,5 …”

Review article: Externally derived control arms—An opportunity for clinical trials in inflammatory bowel disease?

“…However, the prospect of placebo assignment disincentivises patients from trial participation and is a major barrier for more than a third of IBD patients. 8,9 Studies employing active comparators may sidestep the ethical concerns of a placebo group but are hampered by the need for significantly greater patient numbers to demonstrate differences against the test drug, which is costly and time consuming. Therefore, novel study design and delivery strategies must be considered to accommodate testing of the ever-expanding IBD therapeutic pipeline to make necessary advances.…”

“…The FDA also strongly recommends that more than one phase III RCT is conducted to independently substantiate experimental results. However, the prospect of placebo assignment disincentivises patients from trial participation and is a major barrier for more than a third of IBD patients 8,9 . Studies employing active comparators may sidestep the ethical concerns of a placebo group but are hampered by the need for significantly greater patient numbers to demonstrate differences against the test drug, which is costly and time consuming.…”