Transcript for the January 11, 2019 Joint Meeting of the Arthritis
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1 1 FOOD AND DRUG ADMINISTRATION 2 CENTER FOR DRUG EVALUATION AND RESEARCH 3 4 5 JOINT MEETING OF THE 6 ARTHRITIS ADVISORY COMMITTEE (AAC) AND THE 7 DRUG SAFETY AND RISK MANAGEMENT 8 ADVISORY COMMITTEE (DSaRM) 9 10 11 12 Friday, January 11, 2019 13 8:00 a.m. to 5:15 p.m. 14 15 16 17 College Park Marriott Hotel and Conference Center 18 General Vessey Ballroom 19 Building 31 Conference Center 20 3501 University Boulevard East 21 Hyattsville, Maryland 22 A Matter of Record (301) 890-4188 2 1 Meeting Roster 2 DESIGNATED FEDERAL OFFICER (Non-Voting) 3 Yinghua Wang, PharmD, MPH 4 Division of Advisory Committee and Consultant 5 Management 6 Office of Executive Programs, CDER, FDA 7 8 ARTHRITIS ADVISORY COMMITTEE MEMBERS (Voting) 9 Jeffrey Curtis, MD, MS, MPH 10 Marguerite Jones Harbert – Gene Ball Endowed 11 Professor of Medicine 12 Division of Clinical Immunology & Rheumatology 13 University of Alabama at Birmingham 14 Birmingham, Alabama 15 16 17 18 19 20 21 22 A Matter of Record (301) 890-4188 3 1 Jennifer Horonjeff, PhD 2 (Consumer Representative) 3 Founder and Patient Advocate 4 Savvy Cooperative 5 Patient Outcome and Quality Consultant 6 Division of Rheumatology 7 Department of Medicine 8 Columbia University Medical Center 9 Sunnyside, New York 10 11 Martha C. Nason, PhD 12 Mathematical Statistician 13 Division of Clinical Research 14 National Institute of Allergy and Infectious 15 Diseases, National Institutes of Health 16 Rockville, Maryland 17 18 Alyce M. Oliver, MD, PhD 19 Joseph P. Bailey MD Chair in Rheumatology 20 Professor of Medicine 21 Medical College of Georgia at Augusta University 22 Augusta, Georgia A Matter of Record (301) 890-4188 4 1 Veena K. Ranganath, MD, MS 2 Associate Clinical Professor 3 Division of Rheumatology 4 David Geffen School of Medicine at UCLA 5 Los Angeles, California 6 7 Jose U. Scher, MD (via telephone) 8 Assistant Professor in Medicine and Rheumatology 9 New York University School of Medicine 10 New York, New York 11 12 ARTHRITIS ADVISORY COMMITTEE MEMBER (Non-Voting) 13 James B. Chung, MD, PhD 14 (Industry Representative) 15 Executive Medical Director 16 US Medical Organization 17 Inflammation Therapeutic Area Head 18 Amgen, Inc. 19 Thousand Oaks, California 20 21 22 A Matter of Record (301) 890-4188 5 1 DRUG SAFETY AND RISK MANAGEMENT ADVISORY COMMITTEE 2 MEMBERS (Voting) 3 Marie R. Griffin, MD, MPH 4 Professor, Health Policy and Medicine 5 Director, Vanderbilt MPH Program 6 Department of Health Policy 7 Vanderbilt University Medical Center 8 Nashville, Tennessee 9 10 Laurel A. Habel, MPH, PhD 11 Associate Director, Cancer Research 12 Division of Research 13 Kaiser Permanente Northern California 14 Oakland, California 15 16 Martin Kulldorff, PhD 17 Professor of Medicine and Biostatistician 18 Division of Pharmacoepidemiology and 19 Pharmacoeconomics 20 Department of Medicine 21 Harvard Medical School and Brigham & Women's Hospital 22 Boston, Massachusetts A Matter of Record (301) 890-4188 6 1 Steven B. Meisel, PharmD 2 System Director of Medication Safety 3 Fairview Health Services/Healtheast Care System 4 Minneapolis, Minnesota 5 6 Anne-Michelle Ruha, MD, FACMT 7 Vice Chief 8 Department of Medical Toxicology 9 Banner University Medical Center 10 Professor of Internal Medicine and Emergency Medicine 11 University of Arizona College of Medicine 12 Phoenix, Arizona 13 14 Terri L. Warholak, PhD, RPh, CPHQ, FAPhA 15 Professor and Assistant Dean 16 Academic Affairs and Assessment 17 College of Pharmacy 18 University of Arizona 19 Tucson, Arizona 20 21 22 A Matter of Record (301) 890-4188 7 1 TEMPORARY MEMBERS (Voting) 2 John Cush, MD 3 Director of Clinical Rheumatology 4 Professor of Medicine and Rheumatology 5 Baylor Research Institute 6 Baylor University Medical Center 7 Dallas, Texas 8 9 David T. Felson, MD, MPH 10 Professor of Medicine and Epidemiology 11 Boston University School of Medicine 12 Boston, Massachusetts 13 14 C. Michael Gibson, MS, MD 15 Professor of Medicine 16 Harvard Medical School 17 President and CEO 18 Baim Institute for Clinical Research 19 Founder and Chairman 20 PERFUSE Core Laboratories and Data Coordinating 21 Center 22 Boston, Massachusetts A Matter of Record (301) 890-4188 8 1 Matthew H. Liang, MD, MPH 2 Study Director 3 VA Cooperative Studies Program 4 Professor of Medicine, Harvard Medical School 5 Professor of Health Policy and Management Harvard TH 6 Chan School of Public Health 7 Boston, Massachusetts 8 9 Mara McAdams DeMarco, PhD 10 Assistant Professor of Epidemiology and Surgery 11 Johns Hopkins Bloomberg School of Public Health and 12 School of Medicine 13 Baltimore, Maryland 14 15 Donald R. Miller, PharmD 16 Professor of Pharmacy Practice 17 College of Health Professions 18 North Dakota State University 19 Fargo, North Dakota 20 21 22 A Matter of Record (301) 890-4188 9 1 Steven E. Nissen MD 2 Chair, Department of Cardiovascular Medicine 3 Cleveland Clinic 4 Professor, Cleveland Clinic Lerner College of 5 Medicine 6 Cleveland, Ohio 7 8 Bruce M. Psaty, MD, PhD, MPH 9 Professor, Departments of Medicine, Epidemiology and 10 Health Services 11 Co-Director, Cardiovascular Health Research Unit 12 University of Washington 13 Seattle, Washington 14 15 Maria E. Suarez-Almazor, MD, PhD 16 (Acting Chairperson) 17 Barnts Family Distinguished Professor 18 Head, Section of Rheumatology and Clinical Immunology 19 University of Texas MD Anderson Cancer Center 20 Houston, Texas 21 22 A Matter of Record (301) 890-4188 10 1 Gene H. Weiner 2 (Patient Representative) 3 Plymouth, Massachusetts 4 5 FDA PARTICIPANTS (Non-Voting) 6 Sally Seymour, MD 7 Acting Division Director 8 Division of Pulmonary, Allergy, and Rheumatology 9 Products (DPARP) 10 Office of Drug Evaluation II (ODE-II) 11 Office of New Drugs (OND), CDER, FDA 12 13 Nikolay P. Nikolov, MD 14 Associate Director for Rheumatology 15 DPARP, ODE-II, OND, CDER, FDA 16 17 Rosemarie Neuner, MD, MPH 18 Clinical Reviewer 19 DPARP, ODE-II, OND, CDER, FDA 20 21 22 A Matter of Record (301) 890-4188 11 1 Ya-Hui Hsueh, PhD 2 Statistical Reviewer 3 Division of Biometrics VII 4 Office of Biostatistics 5 Office of Translational Sciences, CDER, FDA 6 7 8 9 10 11 12 13 14 15 16 17 18 19 20 21 22 A Matter of Record (301) 890-4188 12 1 C O N T E N T S 2 AGENDA ITEM PAGE 3 Call to Order and Introduction of Committee 4 Maria Suarez-Almazor, MD, PhD 15 5 Conflict of Interest Statement 6 Yinghua Wang, PharmD, MPH 21 7 FDA Opening Remarks 8 Nikolay Nikolov, MD 26 9 Applicant Presentations - Takeda 10 Introduction and Regulatory History 11 Beth-Anne Knapp 37 12 Gout: Disease Burden, Background & 13 Treatment Landscape 14 Michael Becker, MD 44 15 CARES and Cardiovascular Safety of 16 Febuxostat 17 William White, MD 57 18 Efficacy of Febuxostat 19 Lhanoo Gunawardhana, MD, PhD 92 20 21 22 A Matter of Record (301) 890-4188 13 1 C O N T E N T S (continued) 2 AGENDA ITEM PAGE 3 Benefit/Risk Assessment 4 John Affinito, MD 101 5 Clinical Perspectives 6 N. Lawrence Edwards, MD, MACP, MACR 108 7 Clarifying Questions 116 8 FDA Presentations 9 Introduction and Regulatory History 10 Rosemarie Neuner, MD, MP 151 11 Statistical Assessment of 12 Cardiovascular Safety of CARES 13 Ya-Hui Hsueh, PhD 160 14 Characteristics of Febuxostat and 15 Allopurinol Users in Real World 16 Settings and Utilization Patterns 17 Marie Bradley, PhD, MScPH, MPharm 172 18 Clinical Considerations and Benefit-Risk 19 Assessment 20 Rosemarie Neuner, MD, MP 181 21 22 A Matter of Record (301) 890-4188 14 1 C O N T E N T S (continued) 2 AGENDA ITEM PAGE 3 Clarifying Questions 187 4 Open Public Hearing 233 5 Clarifying Questions (continued) 270 6 Charge to the Committees 7 Nikolay Nikolov, MD 292 8 Questions to the Committees and Discussion 297 9 Adjournment 427 10 11 12 13 14 15 16 17 18 19 20 21 22 A Matter of Record (301) 890-4188 15 1 P R O C E E D I N G S 2 (8:00 a.m.) 3 Call to Order 4 Introduction of Committee 5 DR. SUAREZ-ALMAZOR: Good morning. I would 6 like to start by reminding everyone to please 7 silence your cell phones, smartphones, and any 8 other devices, if you have not already done so. My 9 name is Maria Suarez-Almazor, and I'm the acting 10 chairperson this morning. I will now call today's 11 joint meeting of the Arthritis Advisory Committee 12 and the Drug Safety and Risk Management Advisory 13 Committee to order. 14 We'll start by going around the table and 15 introducing ourselves. We will start with the FDA 16 to my left, and then go around the table. 17 DR. SEYMOUR: My name is Dr. Sally Seymour. 18 I'm the acting director for the Division of 19 Pulmonary, Allergy, and Rheumatology Products at 20 the FDA. 21 DR. NIKOLOV: Morning, everyone. My name is 22 Nikolay Nikolov. I'm an associate director for A Matter of Record (301) 890-4188 16 1 rheumatology in the Division of Pulmonary, Allergy, 2 and Rheumatology Products at the FDA. 3 DR. NEUNER: Good morning. My name is 4 Rosemarie Neuner. I'm a medical officer in the 5 Division of Pulmonary, Allergy, and Rheumatology. 6 DR. HSUEH: Good morning. I'm Ya-Hui Hsueh, 7 statistical reviewer from the Office of 8 Biostatistics, FDA. 9 DR. GIBSON: Mike Gibson, a member of the 10 cardio/renal panel. 11 DR. HABEL: Laurie Habel, epidemiologist and 12 associate director for research at Northern 13 California, Kaiser Permanente.