Welcome/Opening Remarks FDA Guidance on Patient Reported Outcomes: Discussion, Dissemination, and Operationalization Chantilly, VA, February 23 – 25, 2006 Jeff. A. Sloan Mayo Clinic Comprehensive Cancer Center Rochester, MN

I was a lot younger when we started planning this meeting…

In fact we were all a lot younger… marlenemichelelaurie jane kristymartha

…at times it was challenging…

…but the guidance is out and we are all here

Thanks FDA & Mayo partnership FDA & Mayo partnership Laurie Burke, Jane Scott Laurie Burke, Jane Scott Writers and Reviewers Writers and Reviewers Funding from Corporate Sponsors Funding from Corporate Sponsors North Central Cancer Treatment Group North Central Cancer Treatment Group Mayo QOL Team Mayo QOL Team Marlene Frost, Michele Halyard Marlene Frost, Michele Halyard Kristy Vierling, Kara Curry Kristy Vierling, Kara Curry Martha Hoag and Mayo CME Martha Hoag and Mayo CME

Corporate Sponsors LEADER:Amgen Eli Lilly Takeda PATRON: GlaxoSmithKline Genetic, Inc. Genetic, Inc. Roche Laboratories, Inc. Roche Laboratories, Inc. Wyeth Wyeth CONTRIBUTOR: Merck Research Laboratories NeoPharm, Inc.

Syllabus Schedule, Faculty bios, financial disclosures Schedule, Faculty bios, financial disclosures FDA draft guidance FDA draft guidance Draft manuscripts, produced before the guidance was released Draft manuscripts, produced before the guidance was released Slide presentations Slide presentations

Housekeeping: Facilities Breaks / Meals Breaks / Meals Phones, copier, fax, messaging, wireless Phones, copier, fax, messaging, wireless Emergency phone number: Emergency phone number:

Work Hard: Play Hard Reception tonight 6:00 – 9:00 Reception tonight 6:00 – 9:00 Dinner hosted by George and Martha Washington Dinner hosted by George and Martha Washington Can still sign up at registration desk Can still sign up at registration desk

Work Hard: Play Hard Reception tomorrow 7:00 – 9:30 Hockey/Movie Night in Canada Hockey/Movie Night in Canada Heavy hors d’oeuvres Heavy hors d’oeuvres Trivia Trivia “Strange Brew” “Strange Brew”

Meeting Survival Facilitation Massage therapists Massage therapists Free chair massage Free chair massage Signup at registration desk Signup at registration desk

Primary Meeting Goals Provide a focused process to facilitate discussion among all stakeholders INPUT and FEEDBACK INPUT and FEEDBACK Present conclusions of each paper Present conclusions of each paper Delineate ways to best operationalize the guidance into clinical trials

Meeting Format 90 minutes per paper 90 minutes per paper minute presentation of findings minute presentation of findings 0-5 minute FDA commentary 0-5 minute FDA commentary minute moderated Q&A / discussion minute moderated Q&A / discussion Audience may submit written questions Audience may submit written questions Team members / FDA panelists respond Team members / FDA panelists respond

Ground Rules Brevity is the soul of wit: 1-minute wisdom Brevity is the soul of wit: 1-minute wisdom Disagreements are not personal Disagreements are not personal Keep to time schedule Keep to time schedule We have a path already chosen, the time for going back to square one has past We have a path already chosen, the time for going back to square one has past People can be nominated for massages People can be nominated for massages

Mayo/FDA Meeting on PRO: Faculty Paper I: Conceptual Issues Margaret Rothman, Ph.D.Johnson & Johnson Philippe Beltram, Pharm.D.Sanofi-Aventis Joe Cappelleri, Ph.D.Pfizer Joe Lipscomb, Ph.D.Emory University Bonnie Teschendorf, Ph.D.American Cancer Society Paper II: PRO Instrument Selection Michele Halyard, M.D.Mayo Clinic Dave Cella, Ph.D.Northwestern Joseph Jackson, Ph.D.Bristol-Myers Squibb Claire Snyder, Ph.D.Johns Hopkins Maria Watson, Ph.D.GlaxoSmithKline Paper III: PRO Instrument Development Issues Ralph Turner, Ph.D.Phase V Technologies Charles Cleeland, Ph.D.MD Anderson Joel Kallich, Ph. D.Amgen Bhash Parasuraman, Ph.D.Astra Zeneca Alexandra Quittner, Ph.D.University of Miami

Mayo/FDA Meeting on PRO: Faculty Paper IV: PRO Validation Marlene Frost, Ph.D.Mayo Clinic Ron Hays, Ph.D.UCLA Astra Liepa, PharmDEli Lilly & Company Bryce Reeve, Ph.D.National Cancer Institute Joseph Stauffer, Ph.D.Alpharma Paper V: Interpretation of Results Based on PRO’s Jeff Sloan, Ph.D.Mayo Clinic Amylou Dueck, Ph.D.Mayo Clinic Pennifer Erickson, Ph. D.Pennsylvania State Univ. Harry Guess, Ph.D.Univ. of North Carolina Dennis Revicki, Ph.D.MEDTAP International Nancy Santanello, M.D.Merck

Mayo/FDA Meeting on PRO: Faculty FDA Response: Laurie Burke, Jane Scott, Donald Patrick et al Canadian Perspective (NCIC): David Osoba, M.D. et al European Perspective (EORTC): Neil Aaronson, Ph.D. et al European Perspective (ERICA): Catherine Acquadro, Ph.D. et al

In Memorium Harry A. Guess, M.D.,Ph.D.