This session focuses on the conceptual framework and rationale underlying patient reported outcomes (PRO) for clinical development programs aimed at achieving PRO labeling or promotional claims. Articulating the rationale for measuring PRO domains and providing the conceptual model demonstrating the mechanisms, links, and content of PRO measures demonstrates the importance of the PROs for assessing product effectiveness. We will also explicate the translation of PRO findings from clinical trials into labeling and promotional claim statements. Examples from recently approved labels will be used to illustrate these concepts. Best practice guidelines will be discussed for describing conceptual frameworks and for making statements about PRO labeling and promotional claims.

This session focuses on best practices for PRO instrument development and validation, in light of the FDA PRO draft guidelines. Specifically, we will address the following questions: what is the optimal approach for developing a new PRO instrument? What is the evidence necessary to demonstrate that an existing instrument can be used in a valid way without going through a full-scale validation process? What needs to be done when an instrument is adapted or modified? What are the implications of changing various design features of an instrument in order to meet the demands of a particular study, including recall period, response options, item stems, etc.? What changes are relatively inconsequential, and what changes require major re-validation efforts? What are some sample size considerations in validation studies? To the extent possible, practical examples will be utilized.