Interpreting Change and Responder Analyses for Patient-Responder Outcomes (Part I)
*Richard Gershon, Northwestern University Feinberg School of Medicine  
*Laura Lee Johnson, NIH 
*Irene Katzan, Cleveland Clinic and The MetroHealth System 
*Nan Rothrock, Northwestern University Feinberg School of Medicine 

Keywords:

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.