The purpose of this guidance is to assist sponsors in the clinical development of drugs for the treatment of ulcerative colitis (UC) in adult and pediatric patients. Specifically, this guidance addresses the Food and Drug Administration’s (FDA’s) current thinking regarding efficacy endpoints for UC clinical trials. This draft guidance is intended to serve as a focus for continued discussions among the Division of Gastroenterology and Inborn Errors Products (DGIEP), pharmaceutical sponsors, the academic community, and the public.
This guidance does not address the treatment or prevention of long-term complications of UC; for example, this guidance is not intended to discuss endpoints for prevention or reduction in risk of colorectal cancer.
This guidance also does not contain discussion of the general issues of statistical analysis or clinical trial design. Those topics are addressed in the ICH guidances for industry E9 Statistical Principles for Clinical Trials and E10 Choice of Control Group and Related Issues in Clinical Trials, respectively.