Objective: The importance of symptom assessment using patient-reported outcomes (PROs) is becoming increasingly recognized in the management of upper gastrointestinal (GI) disease. The authors aimed to review systematically the methodological aspects of PRO instrument development and use in the GERD or dyspepsia literature, and to assess these instruments' properties in light of the Food and Drug Administration's (FDA) guidance.
Material and methods: Systematic PubMed and Embase searches (using terms based on the FDA guidance) identified studies that reported methodological aspects in developing or using PRO instruments for GERD or dyspepsia symptom measurement.
Results: Ten studies were identified (six systematically and four from citation lists). Studies reported the development or use of a relevant PRO instrument, with a focus on methodological aspects that the FDA guidance describes as important for patient understanding. Studies demonstrated heterogeneity of recall periods, symptoms and response options. Two studies demonstrated that a lack of consistent vocabulary may contribute to discrepancy in symptom reporting between investigators and patients. Two studies indicated that symptoms must be described in a manner that is relevant to patients. One study described the development of a PRO instrument separately in two languages, acknowledging linguistic and cultural differences between populations. One study demonstrated changes in symptom severity based on the recall period.
Conclusions: There is considerable heterogeneity in the methodology used to develop PRO instruments for upper GI disease. Adherence to best practices in PRO development and validation may improve the quality and utility of these measures, leading to improved communication in clinical practice.