June 28 - 29, 2006, Renaissance Mayflower Hotel, Washington, DC

Program and Presentation Materials

Meeting Chairs
Dennis Revicki, PhD, Bethesda, MD, USA
William Lenderking, PhD, Groton, CT, USA
Jeff Sloan, PhD, Rochester, MN, USA

Wednesday, June 28
6:00 - 7:30pm
Opening Reception

Thursday, June 29
9:00 - 10:30am
Opening Plenary Session
Draft FDA PRO Guidance and What it Means to You: Overview of the PRO Guidance, Current Status and Future Plans
FDA Scientists: John Powers, Edwin Rock, Sahar Dawisha
Moderators: Dennis Revicki, William Lenderking, Jeff Sloan

10:45 - 11:00am
Break

11:00 - 11:45am
Plenary Session 2
Conceptual Framework and Guidance on Statements about PRO Findings in Product Labels and Promotional Materials
Dennis Revicki, David Cella, Neil Aaronson, William Lenderking
Respondent: Albert Wu
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.

11:45am - 1:00 pm
Lunch on your own

1:00 - 1:45 pm
Plenary Session 3
Plenary Session 3 Respondent
Best Practices for PRO Instrument Development (Including Recall Period, etc.) and Validation
William Lenderking, Ron Hays, Jakob Bjorner, Peter Fayers, Neil Aaronson
Respondent: Nancy Santanello
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.

1:45 - 2:30pm
Plenary Session 4
Standards for Evaluation and Documenting Psychometric Qualities of PRO Instruments
Ron Hays, Neil Aaronson, Jakob Bjorner, Diane Fairclough
Respondent: Margaret Rothman
This session proposes standards for evaluating and documenting the psychometric qualities of PRO measure of use in medical product development and to support labeling claims. We will summarize methods for assessing reliability and validity (including responsiveness) of measures and provide guidance for evaluating these psychometric properties. The presentation will cover the kinds of evidence needed to indicate that a PRO measure has a sufficient level of reliability and validity, evaluation approaches that can be used when a measure is revised, and the types of reliability and validity evaluation that are appropriate during different phases of clinical trials.

2:30 - 3:15pm
Plenary Session 5
Plesary Session 5 Respondent
Statistical Analysis Issues for PROs: Missing Data, Multiplicity, and Longitudinal Data Structure
Jeff Sloan, Peter Fayers, Diane Fairclough, Dennis Revicki
Respondent: Donna Lamping

3:15 - 3:30pm
Break

3:30 - 4:15pm
Plenary Session 6
Interpreting PRO Results: Methods for Determining Responsiveness and MID
David Cella, Ron Hays, Dennis Revicki, Jeff Sloan
Respondent: Albert Wu
This session will review the state of the science in estimating minimally important differences or changes in patient reported outcome instruments, commonly referred to as MIDs. The MID is meant to reflect clinical or subjective importance as opposed to statistical significance, the latter of which is presumed to be necessary but not sufficient for claiming benefit of a new treatment relative to its comparator. Although MIDs are viewed as instrument-specific and perhaps even treatment context specific, experience with several established instruments suggests an MID range within which most applications would apply. Some historical myths about MIDs will be addressed with clarifications based on more recent experience. We will also discuss differences in MID applications when conducting group comparisons versus individual classification. A discussion of how MID information can help regulatory consideration of “substantial benefit,” and other recommendations based on available evidence and experience will be offered. For example, it is unlikely that any instrument has a single MID for every application, yet a fairly restricted range can usually be obtained through triangulation of anchor-based methods, distribution-based methods, and review of ancillary relevant information. The use of MID ranges, and the implications of the planned approach to MID applications in clinical trials will also be discussed.

4:30 - 5:30pm
Plenary Session 7
Commentary and Closing Session: PROs and You: Where Do We Go From Here?
Moderators: William Lenderking; Dennis Revicki; Jeff Sloan
Participants: Nancy Santanello, Margaret Rothman, Donna Lamping, Albert Wu
This session will include a mix of academic, contract research organization, and industry representatives who will comment on and discuss the presentations made during the day.