What do patients rank as more important in quality-of-life (QL) and patient-reported outcome (PRO) evaluation: Symptoms (Sx) or summative assessments? Results of a 3,860-patient survey in lung, breast, and prostate cancers with implications for drug development.

Abstract

9093 Background: Patient (pt) involvement in establishing content validity (CV) is critical for QL and PRO instrument development. Instruments usually contain both multidimensional summative items and single Sx items. As endpoints in drug development, the FDA has emphasized Sx assessment in PRO guidance and has questioned the CV of many instruments as not having sufficient pt input. This survey was conducted to 1) provide in depth CV in several major cancers, and 2) provide an opportunity to assess whether pts agree with the FDA in prioritizing Sx over summative items in PRO/QL evaluation. Methods: Lung, breast, or prostate cancer pts registered with the web-based NexCura resource were surveyed. Pts ranked 20 to 21 issues, including general, physical, functional, psychosocial and summative items, on a 5-point scale. 3,860 pts completed the anonymous web survey. Multiple demographic factors were also assessed to allow subgroup analysis. Results: The Table shows the 5 highest ranked items as rated in the si...