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P059. Development of patient reported outcome (PRO) instruments in ulcerative colitis and Crohn's disease

P.D.R. Higgins1, T. Bhuket2, R. Cross3, M. Flasar3, M. Docherty4, K. Isaacs5, A. Rustgi3, P.A. Wren6

1University of Michigan, Ann Arbor, MI, United States; 2Alameda County Hospital, Oakland, CA, United States; 3University of Maryland, Baltimore, MD, United States; 4University of California San Diego, San Diego, CA, United States; 5University of North Carolina, Chapel Hill, NC, United States; 6Oakland University, Detroit, MI, United States

Aims: Ulcerative colitis (UC) and Crohn's disease (CD) impact >3 million people in the U.S. and Europe. Measurement of disease activity in IBD increasingly relies on objective measures of intestinal inflammation like endoscopy, imaging, and biomarkers, and limits patient input. The FDA has determined that the IBDQ is not valid for labeling claims in IBD. This is a report of the process used to develop FDA- and EMEA-approvable Patient Reported Outcome (PRO) measures for IBD, our preliminary results, and draft instruments.

Methods: UC and CD PRO development requires: (1) focus groups with patients; (2) item development and refinement; (3) patient testing and cognitive debriefing; (4) further item development and refinement; and (5) longitudinal clinical testing. Clinical sites in Oakland, CA; Chapel Hill, NC; San Diego, CA; Baltimore, MD; and Ann Arbor, MI were selected to maximize regional differences and ensure sociodemographic variability. Eligible subjects were males and females between 18 and 75 years of age, fluent in English, and presenting with a range of disease severity. Focus groups discussed broad, open-ended questions about symptoms, impact on daily activities, monitoring flares and remission, coping strategies, and patient-derived definitions of meaningful improvement. Participants then engaged in an item importance exercise to rank symptoms identified through the discussion. Participants also rated their present disease state and worst ever disease state on 100-point disease activity meter.

Results: A total of 38 UC and 42 CD patients participated in the focus groups. Efforts to recruit minority patients were successful; 35% of UC and 38% of CD participants were drawn from communities of color. NVivo qualitative analysis of transcripts yielded symptom domains including stool frequency, stool consistency, pain, blood and mucus, nausea, and weight loss. Concept frequency and saturation across disease type and clinical site were assessed. The item importance exercise demonstrated key differences in the disease experience reported by patients with UC and CD. Testing and refinement of the proposed PRO measures is currently underway with individual cognitive debriefing interviews at the original clinical sites.

Conclusions: Validated PRO Instruments for UC and CD will provide an important complement to objective measures of intestinal inflammation for clinical trials of new IBD therapies. Future plans include weighted scoring of severity and impact, adaptation of the instrument format to personal electronic devices, determination of instrument responsiveness and reliability, and identifying endpoints for response and remission.