Online Program

Interpreting Change and Responder Analysis for Patient-reported Outcomes

*Joseph Cappelleri, Pfizer 
*Lisa A Kammerman, U.S. Food and Drug Administration 

Keywords: patient-reported outcomes, health measurement scales, interpretation, responder analysis

Patient-reported outcome (PRO) measures used for labeling and promotional claims must have: 1) evidence documenting their responsiveness; and 2) interpretation guidelines (e.g., responder definition) to be most useful as effectiveness endpoints in clinical trials. The recommended approach is to estimate the responder definition based on anchor-based methods, which will be discussed during the workshop. However, this workshop will also discuss how distribution-based methods can provide some insights on interpreting the amount of change that signifies an important change in PROs. Confidence in a specific responder change threshold evolves over time and is confirmed by additional research evidence, including clinical trial experience; the responder change threshold may vary by population and context, and no one responder change threshold will be valid for all study applications involving a PRO instrument. During this workshop, the speakers will explain how to demonstrate and identify thresholds for specific study populations in an effort to pursue labeling and promotional claims.