Considerations for the Generalizability of Clinical Outcome Assessment Validation Data Across Rare Diseases
Presented at RARE Patient Advocacy Summit 2019
Development, validation and selection of clinical outcome assessments (COAs) requires:
- A clear understanding of concepts of interest (COIs) for measurement and intended context of use (COU)
- Evidence for content validity
- Evidence for psychometric validity and reliability
The ability to reuse or adapt existing COAs for novel COUs is valuable in reducing cost and timelines. In rare disease, availability of patients and issues of disease course and severity may make adaptation an imperative.