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March 6 Transcript UNITED STATES FOOD AND DRUG ADMINISTRATION PEDIATRIC ADVISORY COMMITTEE MEETING Silver Spring, Maryland Monday, March 6, 2017 2 1 PARTICIPANTS: Welcome and Introductory Remarks: 2 MARK HUDAK, MD 3 Chair of Pediatric Advisory Committee (PAC) Assistant Dean of Managed Care for the 4 University of Florida College of Medicine - Jacksonville 5 Assistant Medical Director Neonatal Intensive Care Unit 6 University of Florida Health Science Center Jacksonville, Florida 7 Review of Agenda and Introduction of Dr. McCune, 8 the New Director of the Office of Pediatric Therapeutics: 9 ROBERT "SKIP" NELSON, MD, PhD 10 Deputy Director, Office of Pediatric Therapeutics 11 Office of the Commissioner (OC) Food and Drug Administration 12 Opening Statement: 13 MARIEANN R. BRILL, MBA, RAC, MT (ASCP) 14 Designed Federal Official, PAC Office of Pediatric Therapeutics 15 Office of the Commissioner (OC) Food and Drug Administration 16 Silver Spring, Maryland 17 Pediatric Focused Safety Review Update - Exjade (deferasirox): 18 PETER WALDRON, MD 19 Division of Pharmacovigilance II Office of Pharmacovigilance and Epidemiology 20 Center for Drug Evaluations and Research (CDER), FDA 21 22 3 1 PARTICIPANTS (CONT'D): 2 KATE GALPERIN, MD, Medical Officer Division of Epidemiology I 3 Office of Surveillance and Epidemiology, (CDER), FDA 4 5 Standard Review of Adverse Event Presentation Kuvan (sapropteria dihydrochloride): 6 JACQUELINE SPAULDING, MD 7 Division of Pediatric and Maternal Health Office of New Drugs, CDER, 8 Food and Drug Administration 9 Nitropress (sodium nitroprusside): 10 LILY (YERUK) MULUGETA, Pharma D Division of Pediatric and Maternal Health 11 Office of New Drugs Food and Drug Administration 12 The Role of Pharmacogenomic Data in 13 Pediatric Therapeutics: 14 ROBERT "SKIP" NELSON, MD, PhD Deputy Director, Office of Pediatric 15 Therapeutics Office of the Commissioner (OC) 16 Food and Drug Administration 17 Pharmacogenomics in Pediatric Product Development and Labeling: 18 DIONNA GREEN, MD 19 Medical Officer/Policy Lead Guidance And Policy Team 20 Office of Clinical Pharmacology Food and Drug Administration 21 22 4 1 PARTICIPANTS (CONT'D): 2 Case Studies in Pharmacogenomics: 3 MICHAEL PACANOWSKI, Pharm D, MPH Office of Office of Clinical Pharmacology 4 Center for Drug Evaluation and Research Food and Drug Administration 5 Analytical and Clinical Validation of 6 Pharmacogenetic Tests: 7 KELLIE B. KELM, PhD Chief, Cardio-Renal Diagnostic Devices Branch 8 Division of Chemistry and Toxicology Devices Office of In Vitro Diagnostic Devices 9 And Radiological Health Food and Drug Administration 10 Clinical Implementation of Precision Therapeutics 11 In Children: 12 J. STEVEN LEEDER, PharmaD, PhD Director, Division of Clinical Pharmacology, 13 Toxicology and Therapeutic Innovation Associate Chair-Research 14 Department of Pediatrics Deputy Director Children's Research Institute 15 Children's Mercy Kansas City Professor of Pediatrics and Pharmacology 16 UMK Schools of Medicine and Pharmacy 17 Discussion: 18 MARK HUDAK, MD Chair of Pediatric Advisory Committee 19 Summary and Wrap-up: 20 ROBERT "SKIP" NELSON, MD, PhD 21 Deputy Director, Office of Pediatric Therapeutics 22 Office of the Commissioner (OC) Food and Drug Administration 5 1 PARTICIPANTS (CONT'D): 2 Adjournment: 3 MARK HUDAK, MD Chair of Pediatric Advisory Committee 4 5 6 7 * * * * * 8 9 10 11 12 13 14 15 16 17 18 19 20 21 22 6 1 P R O C E E D I N G S 2 (8:30 a.m.) 3 DR. HUDAK: Good morning. I think we'll 4 get started. It's 8:30. Welcome to the meeting 5 of the Pediatric Advisory Committee. I'm Mark 6 Hudak and I have the privilege of chairing this 7 meeting. So we have a very full and interesting 8 agenda today as always. A couple of 9 administrative items we need to do this morning. 10 But we'll start by going around the table and 11 having the members around the table introduce 12 themselves. We have some new members and some new 13 consultants. So this will be informative for 14 everybody. So, I guess we'll start with Dr. 15 Portman. Caught you unaware there. Sorry. 16 DR. PORTMAN: You did. You did indeed. 17 So I'm Ron Portman. I'm a Pediatric Nephrologist. 18 And I represent industry, working at the Pediatric 19 Therapeutic Area of Novartis. 20 DR. TURER: I'm Christy Turer. I am a 21 combined Internal Medicine Pediatric attending at 22 UT of Southwestern and the Director of the 7 1 Academic General Pediatric Scholarship Program. 2 DR. SAYEJ: Good morning. I am Wael 3 Sayej, Pediatric Gastroenterologist from 4 Connecticut Children's Medical Center in the 5 University of Connecticut. I am also the 6 Fellowship Director of the Pediatric 7 Gastroenterology fellowship there. 8 DR. KASKEL: Good morning. I'm Rick 9 Kaskel, Pediatric Nephrologist. I'm at Einstein 10 Montefiore, Director of Child Health for the CTSA. 11 DR. ANNE: Good morning. I'm Premchand 12 Anne, Pediatric Cardiologist. I'm at St. John 13 Hospital and Medical Center in Detroit, Michigan. 14 DR. WADE: Good morning. I'm Kelly 15 Wade. I'm a Neonatologist at Children's Hospital 16 of Philadelphia and the University of Pennsylvania 17 School of Medicine. 18 DR. CATALETTO: My name is Mary 19 Cataletto. I'm a Pediatric Pulmonologist at 20 Winthrop University Hospital in New York. 21 MS. MOORE: Good morning. My name's 22 Erin Moore. I'm a Healthcare Navigation 8 1 consultant. I have a six year old son who has 2 cystic fibrosis. And I work at Cincinnati 3 Children's Hospital on the Cystic Fibrosis 4 Learning Network. And also, I'm with Eli Lily 5 Pharmaceuticals on Clinical Trial Innovation. 6 DR. WHITE: Michael White from New 7 Orleans. I'm part of the UQ Ochsner Clinical 8 School, Pediatric Cardiologist. 9 DR. CALLAHAN: I'm David Callahan, I'm a 10 Child Neurologist, part of Washington University 11 Physicians in St. Louis. 12 MS. BRILL: I'm Marieann Brill. I'm the 13 Designated Federal Officer for this meeting. 14 DR. ZUPPA: Hi. I'm Athena Zuppa. I'm 15 a Pediatric Intensivist and Clinical 16 Pharmacologist from the Children's Hospital of 17 Philadelphia. 18 DR. CNAAN: Avital Cnaan. I'm a 19 Biostatistician, George Washington University, 20 D.C. 21 DR. COPE: Hi. Judy Cope, Pediatrician, 22 Epidemiologist. I head up the Safety Team in the 9 1 Office of Pediatric Therapeutics at FDA. 2 DR. HAUSMAN: Ethan Hausman, CEDR's 3 Division of Pediatric and Maternal Health. 4 Pediatrician and Pathologist. 5 DR. NELSON: Skip Nelson. I'm the 6 Deputy Director of the Office of Pediatric 7 Therapeutics. Formally in Neonatology and 8 Pediatric Critical Care. 9 DR. ALEXANDER: My name is John 10 Alexander. I'm the Deputy Director of the 11 Division of Pediatric and Maternal Health and the 12 Center for Drug Evaluation and Research at FDA. 13 MS. WEINEL: Hello. 14 MR. HUDAK: Let me check if there are 15 two people on the phone. 16 MS. WEINEL: Yes. This is Pam WEINEL. 17 I'm the Project Manager for this meeting. And 18 there are two people on the phone. And we're 19 going to see if they can come in and say hello. 20 DR. KISHNANI: Good morning. This is 21 Priya Kishnani. I'm a Clinical Advisor 22 (inaudible). 10 1 DR. HAVENS: I'm Peter Havens. 2 Pediatrician 3 (inaudible) Infectious Diseases at 4 the Medical College of Wisconsin 5 and Children's Hospital of 6 Wisconsin in Milwaukee. And 7 there's a lot of feedback on my 8 phone. I don't know what's going 9 on. 10 DR. KISHNANI: I caught the same thing. 11 I have a lot of feedback. 12 MS. WEINEL: We're trying to get the 13 sound right. So, just wait one minute and we're 14 going to see if you're --. You're sounding better 15 in here. Just wait one minute. Is it better? 16 DR. HUDAK: Yes. 17 DR. HAVENS: Yes. Now it's better. 18 DR. KISHNANI: Yes. Yes. 19 MS. WEINEL: Great. 20 DR. HUDAK: Welcome to those on the 21 phone. And if I forget to call you when it's 22 voting time for different matters, please speak 11 1 up. So, now I'll turn it over to Dr. Nelson, who 2 has some business to take care of. 3 DR. NELSON: Thanks Mark. So before I 4 review the Agenda, I thought I would introduce 5 Suzie McCune, who is our new Director of the 6 Office of Pediatric Therapeutics. Susie can -- 7 she likes short introductions. But let me just 8 say, Suzie's been around at the agency probably 9 for, I don't know, 10 15 years. She started, I believe, in 11 the Office of Pediatrics and Counterterrorism, 12 back in the days they called it Babes and Bombs, 13 before the Office of Pediatric Therapeutics was 14 founded, which was -- the OPT was founded in, I 15 think, 2002. So, I don't know if Suzie -- Suzie's 16 a Neonatologist by the way. And was at Children's 17 National Medical Center before joining FDA. So do 18 you want to just say hello Suzie, or is that --? 19 DR. MCCUNE: Hello. 20 DR. NELSON: (Laughter) 21 DR. MCCUNE: Skip told me that's all I 22 have to say, so. So, I just want to thank you all 12 1 for coming today. And I'm looking forward to the 2 discussion and it's really nice to be part of this 3 group (inaudible). 4 DR. NELSON: It's actually -- Suzie 5 reminded me, I think she actually presented some 6 of the safety stuff to the Committee back in 2003 7 and 2004.
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