# Patient experiences in ulcerative colitis: conceptual model and review of patient-reported outcome measures

## TL;DR

This paper reviews patient experiences with ulcerative colitis and evaluates tools for measuring patient-reported outcomes.

## Contribution

The study develops a conceptual model of UC symptoms and evaluates existing patient-reported outcome measures for their validity and usefulness.

## Key findings

- A conceptual model grouped 52 symptoms and 72 impacts into eight, two, and five dimensions respectively.
- Five PRO measures showed good or excellent content validity and met FDA and COSMIN criteria.
- All reviewed PRO measures require further psychometric evaluation to confirm their effectiveness.

## Abstract

To identify symptoms and their impacts on daily functioning and health-related quality of life (HRQoL) experienced by adult patients with ulcerative colitis (UC) and evaluate patient-reported outcome (PRO) measures for UC clinical studies.

A conceptual model of symptoms and impacts of UC were developed from a literature review. PRO measures were identified from the literature, clinical trials databases, health technology assessment submissions, and regulatory label claims, and were selected for conceptual analysis based on disease specificity and use across information sources. PRO measures covering the most concepts when mapped against the conceptual model were assessed for gaps in psychometric properties using Food and Drug Administration (FDA) guidance and consensus-based standards for the selection of health measurement instruments (COSMIN) criteria.

The conceptual model grouped the 52 symptom concepts and 72 proximal and distal impacts into eight, two, and five dimensions, respectively. Of 65 PRO measures identified, eight underwent conceptual analysis. Measures covering the most concepts and assessed for psychometric properties were the Inflammatory Bowel Disease Questionnaire, Symptoms and Impacts Questionnaire for UC, UC-PRO symptoms modules, UC-PRO impact modules, and Crohn’s and UC Questionnaire; all had good or excellent support for content validity. The UC-PRO Signs and Symptoms fully met FDA guidance and COSMIN criteria for content validity and most psychometric properties.

Existing PRO measures assess concepts relevant to patients with UC, but all PRO measures reviewed require further psychometric evaluation to demonstrate they are fit for purpose.

The online version contains supplementary material available at 10.1007/s11136-024-03612-4.