The Challenge of Measuring Renodes Phenomenon

Renodes phenomenon, a common manifestation of scleroderma, has no FDA-approved treatments due to difficulties in measuring changes in patient reported outcomes (PROs) in clinical trials. Developing a new PRO instrument became essential to assess renodes. The AsRAP questionnaire was developed through a five-year research program, following FDA guidance and best practices. It aimed to capture the patient experience of renodes, including physical symptoms, emotional impact, functional ability, social participation, work participation, and quality of life. The questionnaire underwent rigorous statistical analyses such as factor analysis, item response theory, and graded response modeling to ensure validity and reliability.