Development of Patient-reported Outcome Measures for Ulcerative Colitis and Crohn’s Disease.
A number of modalities are available for objective measurement of inflammatory bowel disease (IBD) activity (endoscopy, histopathology, imaging, biomarkers) however the goals of therapy must also include improvements in disease characteristics that are important to patients–those which improve their function and well-being. Patient-reported outcomes (PROs) are considered the gold standard for quantifying patient experience by regulatory agencies and are currently recommended as a component of disease assessment in registration trials of therapies for IBD. Although PROs are widely used to study chronic diseases, a limitation to drug development in IBD specifically has been a lack of PROs that meet the regulatory standards for index development. We developed the Symptoms and Impacts Questionnaire for Crohn’s Disease and Ulcerative Colitis (SIQ-CD and SIQ-UC, respectively) to address this need using guidelines recommended by the United States Food and Drug Administration and International Society for Pharmacoeconomics and Outcomes Research for the development of PROs. The SIQ-CD and SIQ-UC are also critically different from other PROs in that centrally-read endoscopy was used to qualify patients based upon a range of endoscopic disease activity during index development. These draft questionnaires are available for use in Crohn’s disease and ulcerative colitis clinical trials, and hold promise for evaluating IBD-related symptoms and impacts in patients with a range of clinical and endoscopic disease severity.