Related Experiment Video
Updated: Dec 30, 2025
The Dyspepsia Educational Tool As a Novel Aid in Dyspepsia Management
Published on: June 29, 2019
Concepts and Instruments for Patient-Reported Outcome Assessment in Celiac Disease: Literature Review and Experts'
Sarah Clifford1, Aliki Joanna Taylor2, Michele Gerber3
1Patient Centered Outcomes, Commercialisation and Outcomes, ICON Clinical Research, Los Angeles, CA, USA.
Background:
In diseases where there is a large subjective component, such as celiac disease (CD), patient reported-outcomes (PRO) endpoints are highly relevant. However, there is a gap in knowledge about which PRO endpoints and instruments should be used for clinical trials for treatment of celiac disease.
Objectives:
To identify patient-centered symptom, impact, and health-related quality of life (HRQoL) concepts in CD and relevant PRO instruments, and to gather expert input on concepts and instruments to inform selection of PRO endpoints for use in clinical trials of new CD treatments.
Methods:
A targeted literature review was conducted to identify symptom, impact, and HRQoL concepts, including those captured in PROs further reviewed against U.S. Food and Drug Administration standards for development and validation as endpoints. US and European clinicians, payers, and a patient advocate (n = 21) were interviewed to assess the identified concepts' relative importance in measuring treatment benefit and to gauge the value of potential PROs as endpoints for market access/reimbursement.
Results:
Thirty-four published studies were identified: 27 elucidated patient-centered concepts and 7 detailed the development or validation of PRO instruments. The Celiac Disease Symptom Diary and Celiac Disease Patient Reported Outcome instrument were deemed most appropriate for use as endpoints; however, each had limitations related to conceptual coverage, evidence for measurement properties, and feasibility for use in clinical trials. Experts reported gastrointestinal symptoms as most important to treat, with extra-intestinal symptoms burdensome from the patient perspective as well. Payers emphasized measuring both frequency and severity of symptoms and targeting patients nonresponsive to the gluten-free diet for treatment.
Conclusions:
With emerging treatment options for CD, further work is needed to operationalize PRO symptom endpoints that are meaningful to patients, valued by payers, and acceptable to regulators in demonstrating efficacy.
More Related Videos
05:56Implementation of Non-invasive Point of Care Transient Elastography for Evaluation of Liver Disease in Pediatric Populations with Cystic Fibrosis
Published on: August 29, 2025
06:28E-Patient Counseling Trial E-PACO: Computer Based Education versus Nurse Counseling for Patients to Prepare for Colonoscopy
Published on: August 1, 2019
Related Concept Videos
01:29Assessment of the Gastrointestinal System II: Health Perception Pattern
Health Perception Patterns
Health perception patterns offer valuable insights into a patient's lifestyle habits and how they may impact their GI health. These patterns include:
01:30Inflammatory Bowel Disease III: Diagnostic Studies and Management I-Nutritional Therapy
Diagnostic studies
A colonoscopy is the definitive screening test, distinguishing ulcerative colitis from other colon diseases with similar symptoms. During a colonoscopy test, inflamed mucosa with exudate ulcerations can be observed, and biopsies are taken to determine the histologic characteristics of the...
01:17Assessment of the Gastrointestinal System I: Subjective Data
Health History
The initial step in assessing the GI system is obtaining a comprehensive health history. This includes inquiring about the patient's history or presence of problems...
01:30Irritable Bowel Syndrome II: Clinical Features and Diagnostic Evaluation
Irritable Bowel Syndrome (IBS) is classified into subtypes based on the predominant bowel habits as determined by the Bristol Stool Form Scale (BSFS). The subtypes are:
01:20Taste Buds and Receptors
01:28Peptic Ulcer Disease III: Clinical Manifestations and Diagnostic Studies
Few clinical manifestations differentiate gastric ulcers from duodenal ulcers. Distinctions in the location, timing, and pain relief are crucial for healthcare providers in differentiating between gastric and duodenal ulcers during clinical assessments.