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Patient-Reported Outcome Assessments in Gastrointestinal Clinical Trials: A Framework for Development and Validation
Ist Teil von
The American journal of gastroenterology, 2022-07, Vol.117 (7), p.1019-1021
Ort / Verlag
United States: Wolters Kluwer Health Medical Research, Lippincott Williams & Wilkins
Erscheinungsjahr
2022
Link zum Volltext
Beschreibungen/Notizen
To enable the demonstration of benefit, clinical trials for GI disorders frequently use alternative outcome measures such as patient-reported outcome (PRO) assessments (1), which measure how patients feel and function through the assessment of clinical signs/symptoms, and biomarkers, which measure the molecular, histologic, radiographic, or physiologic characteristics of the studied disorder. [...]PRO assessments have frequently been positioned as primary or coprimary endpoints to demonstrate substantial evidence of efficacy for GI drug development programs. STEP 4: PILOT TEST THE PRO ASSESSMENT The refined PRO assessment should be pilot tested in an early-phase clinical trial to ensure its reliability, validity, and ability to detect a treatment effect, and to identify potential performance issues before use in late-phase clinical studies intended to support the demonstration of substantial evidence of efficacy, as described in the US FDA guidance for industry (8). Development of PRO assessments is typically a multiyear process involving multiple stages of patient input, pilot testing, and iterative refinement of the assessment itself. [...]it is important to begin the development process early to allocate sufficient time for implementation and testing.