Mo2025 Patient Preference for Defining Success in Fecal Incontinence Treatment Trials

Abstract

Background: Fecal incontinence (FI) affects 9% of non-institutionalized U.S. adults and may severely impact quality of life. In recent clinical trials evaluating new treatments for regulatory approval, the primary outcome is often the proportion of responders, defined as anyone with least a 50% reduction in FI frequency. However, some patients have objected that a 50% reduction is not enough to restore quality of life and would not be regarded by them as a Aim: To identify patient preferences for the best way to identify what constitutes success in the treatment of FI. Methods: 23 individuals with FI participated in one of six 2-hour online moderated chatroom discussions in groups of 2-7 patients. Participation was anonymous only arbitrarily assigned IDs were used to identify patients in the focus groups. Topics covered included patient preferences for the terminology used to refer to FI, what should be included in the definition of FI (reported separately), and patient views on how to define a successful outcome of treatment. Participants were recruited via online advertisements, mass e-mails to the students and staff of the university, and our Center's research participant registry. Volunteers first logged onto a website where they gave informed consent to the research and answered standardized questionnaires on FI severity (Fecal Incontinence Severity Index, Constipation and Incontinence Questionnaire) and quality of life impact (Fecal Incontinence Quality of Life scale). They also signed up for one of several scheduled chatroom sessions. The sessions were moderated by the study investigators who combined open-ended questions (e.g., How would you define success in a clinical trial?) with questions about whether specific responder definitions (e.g., at least a 50% reduction) were acceptable to them. Results: There were 23 participants, 22 were females. Only 41% of subjects felt that a 50% reduction in FI symptoms reflected a meaningful treatment success. When asked how much reduction would be necessary to regard the treatment as successful, the average was a 75% reduction. Only 4 out of 23 subjects reported that a cure (100% reduction in FI frequency) was necessary to count a treatment as successful. Adequate relief was endorsed by 52% of subjects as a good way to measure results, although several commented that they did not know what adequate relief means. Conclusions: The primary outcome that has been accepted by regulatory agencies for approval of new treatments 50% reduction in treatment is not considered to be sufficient for treatment to be successful by the majority of patients, but neither would they require a complete cure. On average, these FI patients felt that a 75% reduction in frequency would be required for a successful treatment outcome. [Supported by Salix Pharmaceuticals and AHRQ grant R01 HS018695]