Addressing the Barriers to Pediatric Drug Development: Workshop Summary (2008)

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Appendix B Speaker Biographies William E. Evans, PharmD, is Director and Chief Executive Officer of St. Jude Children’s Research Hospital, and First Tennessee Bank Professor at the University of Tennessee Colleges of Medicine and Pharmacy. For the past 30 years, his research at St. Jude has focused on the pharmacoge- nomics of anticancer agents in children, for which he has received three consecutive National Institutes of Health (NIH) MERIT Awards from the National Cancer Institute. The major disease focus of his pharma- cogenomics research has been on acute lymphoblastic leukemia in chil- dren. Dr. Evans has authored more than 300 articles and book chapters, has been the editor of several textbooks and scientific journals, and has received several national awards for his research. He was elected to the Institute of Medicine (IOM) in 2002. George P. Giacoia, MD, is the Program Director of the National Insti- tute of Child Health and Development (NICHD) Pediatric Pharmacology Research Network and the coordinator and planner of the NICHD Pedi- atric Formulation Initiative. Dr. Giacoia is a neonatologist whose research interest has been in the area of neonatal pharmacology. Richard L. Gorman, MD, is a partner in the private practice of pediatrics. In prior years, he ran a pediatric emergency department and an ambula- tory center and was the Medical Director of the Maryland Poison Center. A graduate of Catholic University, he received his MD from Downstate Medical School in Brooklyn, NY. His pediatric residency was at Children’s 46 

APPENDIX B 47 National Medical Center in Washington, DC. A general pediatric aca- demic development fellowship at Johns Hopkins followed. He has been active in the American Academy of Pediatrics (AAP), serving on the Task Force on Terrorism, the Committee on Drugs, and the Section of Clinical Pharmacology and Therapeutics. Dr. Gorman is currently chair of that section. He has worked with AAP to ensure that drugs are both tested and labeled for the pediatric patient. Thomas Hassall, RPh, MS, holds a bachelor of science degree in pharmacy from the University of Iowa and a master’s degree in hospital pharmacy administration from the University of Minnesota. He served as a Com- missioned Officer in the U.S. Public Health Service for 26 years, including 16 years at the Food and Drug Administration (FDA). Mr. Hassall’s FDA experience includes regulatory positions in the Division of Cardio Renal Drug Products, the Division of Gastrointestinal and Coagulation Drug Products, the Division of Over the Counter Drug Products, the Office of the Center Director, and the Office of Drug Evaluation IV (ODE IV). While in ODE IV he worked on the implementation of the pediatric exclusivity provisions of the Food and Drug Administration Modernization Act and promulgation of the “pediatric rule.” Mr. Hassall has 6 years of regulatory policy experience in the pharmaceutical industry, where he has contin- ued his interest in pediatric drug development. He currently holds the position of Senior Director in Global Scientific, Medical, and Regulatory Affairs at Abbott. Natasha D. Jarrett graduated from Oxford Brookes University with an honors degree in biology and has worked in regulatory affairs since 1997. Ms. Jarrett started her regulatory career at GlaxoWellcome, UK and sub- sequently moved to Hoffmann-La Roche in Hertfordshire, UK, where she worked on a range of local marketing and development projects. For the past 4 years, Ms. Jarrett has been working at Hoffmann-La Roche in the United States, with U.S. and global responsibility for a range of virology and dyslipidemia development projects. Ms. Jarrett is also the regulatory representative for U.S. pediatric strategy and development. She is cur- rently Director, Regulatory Affairs at Hoffmann-La Roche, Nutley, NJ. Michael Katz, MD, is Senior Vice President for Research and Global Programs, March of Dimes Birth Defects Foundation, Reuben S. Carpen- tier Professor, Emeritus of Pediatrics and Professor, Emeritus of Public Health at Columbia University. He is also consultant, emeritus to the New York–Presbyterian Hospital. He received an AB degree from the University of Pennsylvania, an MD degree from State University of New York, Downstate Medical Center, and an MS degree in tropical medicine 

48 ADDRESSING THE BARRIERS TO PEDIATRIC DRUG DEVELOPMENT from Columbia University. He served an internship at the University of California Hospital, Los Angeles, and a residency at Babies Hospital in New York. He was an Assistant Professor of Pediatrics at the University of Pennsylvania and an associate member of the Wistar Institute of Anatomy and Biology in Philadelphia prior to his appointment as Professor of Pub- lic Health and Head of the Division of Tropical Medicine in the School of Public Health at Columbia University in 1970. In 1972, he became Professor of Pediatrics and Director of the Division of Infectious Diseases at Babies Hospital. From 1977 to 1992, he was the Reuben S. Carpentier Professor and Chairman of the Department of Pediatrics at Columbia University’s College of Physicians and Surgeons and Director of Pediat- ric Service at Presbyterian Hospital (Babies Hospital) in New York. He has been in his current position since 1992. Dr. Katz is an internationally recognized expert in parasitic diseases, the relationship between malnutri- tion and infection, and viral pathogenesis. He is the author of numerous publications and has received a number of awards and honors, including the Senior U.S. Scientist Award of the Alexander von Humboldt Founda- tion in Germany and a Distinguished Service Award of the College of Physicians & Surgeons of Columbia University. He has served on advi- sory committees and as a consultant to the World Health Organization, UNICEF, USAID, and the IOM for his expertise in virology and in tropical diseases. Dr. Katz is a fellow of the American Society for the Advancement of Science, a fellow of the Infectious Diseases Society of America, and a member of the IOM. Lisa Lee Mathis, MD, is a board-certified pediatrician in the U.S. Public Health Service with special interest and experience in counterterrorism and medical care of refugees and displaced persons. Dr. Mathis attended medical school at the Uniformed Services University of the Health Sci- ences and then did a residency in pediatrics at the University of Califor- nia, Davis. After that, she practiced general pediatrics in inner cities, and currently practices at the National Naval Medical Center in Bethesda, MD. Dr. Mathis is also an Associate Professor of Pediatrics at the Uniformed Services University, where she teaches first- and second-year medical students in the classroom and third- and fourth-year medical students in the pediatric clinic. Mary Dianne Murphy, MD, is the Director of the Office of Pediatric Therapeutics in the FDA’s Office of the Commissioner. Previously, in the Center for Drug Evaluation, Dr. Murphy was Director of the Office of Counter-terrorism and Pediatric Drug Development (2001–2004), Asso- ciate Director for Pediatrics (1998–2001), and Director of the Office of Drug Evaluation with oversight for all of the divisions involved with 

APPENDIX B 49 antimicrobial therapeutics (1998–2001). Dr. Murphy received her medical degree from the Medical College of Virginia. After completing a pediatric residency at the University of Virginia, she spent 3 years at the National Naval Medical Center as a Navy pediatrician before completing a fel- lowship in pediatric infectious diseases at the University of Colorado. Dr. Murphy was an Assistant Professor for Pediatrics at the University of Texas Health Science Center at San Antonio, an Associate Professor of Pediatrics and medical consultant to the Diagnostic Virology Laboratory at the University of Tennessee Medical Center at Knoxville, and Professor of Pediatrics and Chief of General Pediatrics at the University of Florida Health Science Center at Jacksonville. Dr. Murphy has numerous articles in refereed publications on pediatric infectious diseases, pediatric drug development, residency teaching, and laboratory diagnosis and is the editor of a book on office laboratory procedures. Robert M. Nelson, MD, PhD, is currently Associate Professor of Anes- thesiology and Critical Care at The Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania School of Medicine. After receiving his MD from Yale University in 1980, Dr. Nelson trained in pediatrics (Massachusetts General Hospital) and neonatology and pediat- ric critical care (University of California, San Francisco). He has received formal training in theology and religious and medical ethics, receiving a Master of Divinity from Yale Divinity School in 1980 and a PhD in The Study of Religion from Harvard University in 1993. Dr. Nelson has lec- tured and published widely on ethical and regulatory issues in pediatric research and clinical care. He is Chair of the Pediatric Advisory Com- mittee (PAC) of the FDA, and former Chair of the PAC Pediatric Ethics Subcommittee. Dr. Nelson is a member of the Human Studies Review Board of the Environmental Protection Agency, and the Subcommittee on Research Involving Children of the Secretary’s Advisory Committee on Human Research Protections, U.S. Department of Health and Human Services. He was a member of the IOM Committee on Clinical Research Involving Children (through March 2004), and former Chair of the AAP Committee on Bioethics (through 2001). Currently he is Director of the Center for Research Integrity, established at CHOP to further the respon- sible conduct of pediatric research. Dr. Nelson’s current research explores different aspects of child assent and parental permission, such as adoles- cent risk perception, the development of a child’s capacity to assent, and the degree to which parental choice is perceived as voluntary. His research has been funded by NIH, the Greenwall Foundation, and the National Science Foundation. 

50 ADDRESSING THE BARRIERS TO PEDIATRIC DRUG DEVELOPMENT Walter A. Orenstein, MD, joined Emory University’s School of Medicine in March 2004 as Director of a new Emory Program for Vaccine Policy and Development and as Associate Director of the Emory Vaccine Center. Dr. Orenstein retired from his 26-year career at the Centers for Disease Control and Prevention (CDC), where he led the National Immuniza- tion Program, a $1.6 billion effort with more than 450 staff, dedicated to reducing vaccine-preventable disease burdens around the world, includ- ing elimination of some of the greatest causes of childhood mortality and disability. Dr. Orenstein’s primary appointment is in the Division of Infec- tious Diseases in the Department of Medicine at the Emory University School of Medicine. He holds faculty appointments in Pediatrics and in the Departments of International Health and Epidemiology in Emory’s Rollins School of Public Health. During Dr. Orenstein’s tenure at the National Immunization Program, he has led successful efforts to combat and markedly reduce the occurrence of once-common childhood diseases, including measles, rubella, mumps, meningitis from Haemophilus influ- enzae type b (Hib), varicella, and invasive pneumococcal disease. The Immunization Program also has made major contributions: protecting adults from vaccine-preventable diseases through eliminating barriers to vaccination and developing new vaccine strategies, expanding vaccine safety efforts, improving risk communication, and promoting the use of immunization registries. William J. Rodriguez, MD, PhD, received his MD and his PhD in micro- biology from Georgetown University. He completed his internship and residency at University Hospital in San Juan, Puerto Rico, and completed a fellowship in infectious disease at the Children’s National Medical Cen- ter. He has been Professor of Pediatrics at The George Washington Univer- sity (GWU) since 1985 and was Chairman of the Department of Infectious Diseases at Children’s from 1983 to 2000. The author of more than 130 papers and book chapters, as well as numerous abstracts, Dr. Rodriguez has distinguished himself with his research in the areas of new antibiotic development, the treatment of middle ear infections, and the study of monoclonal antibodies in the prevention and treatment of respiratory syncytial virus in infants and young children. In 2000, Dr. Rodriguez retired from Children’s National Medical Center and was appointed Pro- fessor Emeritus in GWU’s School of Medicine and the Health Science. Dr. Rodriguez joined the FDA in July 2000, when he was appointed Science Director for Pediatrics in the Office of Counter-Terrorism and Pediatric Drug Development in the Center for Drug Evaluation and Research. He is currently the Science Director for Pediatrics in the Office of New Drugs in an assignment in the Office of Pediatric Therapeutics in the Office of the Commissioner. He has participated in the pediatric initiatives that have 

APPENDIX B 51 encouraged pediatric drug development. Information from some of the initiatives’ early findings has been communicated in scientific journals. Wayne R. Snodgrass, MD, PhD, is a clinical pharmacologist, medical tox- icologist, and pediatrician. He is the Medical Director of the Texas Poison Center–Houston/Galveston. He is Chair, AAP Committee on Drugs; Chair, Data Safety Monitoring Board of Pediatric Pharmacology Research Units, NICHD, NIH; Chair, Scientific Advisory Committee, American Associa- tion of Poison Control Centers; Chair, Neuroprotection Review Panel, U.S. Army; former Chair, Pediatric Expert Panel, U.S. Pharmacopeia; member, Best Pharmaceuticals for Children Act drug listing committee; member, Network Steering Committee, Obstetric Pharmacology Research Units, NICHD, NIH; member, Advisory Committee on Childhood Lead (Pb) Poisoning & Prevention, CDC; and former member, Non-Prescription Drug Advisory Committee, FDA. His clinical activities include Attend- ing Physician for the clinical pharmacology–toxicology consult service at University Hospital Galveston; formerly Attending Physician for the pediatric intensive care unit; and formerly Attending Physician in the neo- natal intensive care unit. His research interests include cytochrome P450 isozyme and allele patterns to predict individual toxicity risk for drugs that undergo metabolic activation; sedation and analgesia in infants and children; and development of drugs of choice criteria for use by physi- cians providing medical care for infants and children. Stephen P. Spielberg, MD, PhD, has been Dean of Dartmouth Medical School since July 2003. He received an AB from Princeton University and an MD and a PhD (Pharmacology) from the University of Chicago. He did a pediatric internship and residency at Boston Children’s Hospital and a fellowship in genetics at NICHD. He held faculty positions in pediatrics and in pharmacology at Johns Hopkins and the Hospital for Sick Children in Toronto, where he was a Director of Clinical Pharmacology and Toxicol- ogy. At Merck Research Laboratories and Johnson & Johnson, he was Vice President for Pediatric Drug Development prior to going to Dartmouth. He was Rapporteur for ICH E-11 and served on the FDA Pediatric Advi- sory Subcommittee while he was in industry. He is currently President of the American Society for Clinical Pharmacology and Therapeutics; on the Board of the Foundation for the NIH; on the Council of Convention of the U.S. Pharmacopeia; on the External Advisory Boards of the NICHD PPRU Network; and in the Elizabeth Glaser Pediatric Research Network. Robert M. Ward, MD, completed medical school at Johns Hopkins Uni- versity and trained in pediatrics, neonatology, and clinical pharmacology at the University of Minnesota. He was appointed to Assistant Professor 

52 ADDRESSING THE BARRIERS TO PEDIATRIC DRUG DEVELOPMENT of Pediatrics and Pharmacology at Pennsylvania State University in 1979. His research interests focused on neonatal and fetal pharmacology and drug therapy. Dr. Ward moved to the University of Utah in 1985 as an Associate Professor of Pediatrics. He served as Medical Director of the Primary Children’s Medical Center Newborn Intensive Care Unit from 1989 to 1997. He was promoted to Professor of Pediatrics in 1995. In 1997, he began the Pediatric Pharmacology Program at the University of Utah, a clinical trials program for the study of medications in children. He is currently PI of one of 13 sites in the NIH Pediatric Pharmacology Research Unit Network. In 1997, Dr. Ward became the Chair of the AAP Commit- tee on Drugs and consulted in the development of the Food and Drug Administration Modernization Act, Best Pharmaceuticals for Children Act, and Pediatric Research Equity Act. He has consulted with the FDA and USP and testified before Congress regarding the study and approval of new drugs for pediatric patients. As a professor of pediatrics, he has authored more than 80 manuscripts, book chapters, and editorials. Annic Weyersberg, MD, worked in the University Children’s Hospital of Cologne before joining the European Agency for the Evaluation of Medicinal Products in November 2005 as a national expert for pediatrics for the preparation of the Paediatric Regulation.