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Rare Diseases and Orphan Products: Accelerating Research and Development G Committee and Staff Biographies Thomas F. Boat, M.D. (Chair), is Executive Associate Dean, University of Cincinnati College of Medicine and CEO of UC Physicians. He is immediate past Director of the Children’s Hospital Research Foundation and past Chairman of the College’s Department of Pediatrics. He also was physician-in-chief of Children’s Hospital Medical Center of Cincinnati. A pediatric pulmonologist by training, Dr. Boat worked early in his career to define the pathophysiology of airway dysfunction and develop more effective therapies for chronic lung diseases of childhood, such as cystic fibrosis. More recently he has worked at local and national levels to improve research efforts, subspecialty training, and clinical care in pediatrics. Dr. Boat previously served as chairman of the Department of Pediatrics at the University of North Carolina, Chapel Hill and co-director of the Cystic Fibrosis Center at Rainbow Babies and Children’s Hospital in Cleveland. He is Immediate Past Board President of the Association of Accreditation of Human Research Protection Programs, Inc. He has also served as Chair of the American Board of Pediatrics and President of the Society for Pediatric Research as well as the American Pediatric Society. Dr. Boat is a member of the Institute of Medicine and has served as member or chair of a number of IOM and National Research Council committees. Peter C. Adamson, M.D., is Chief of the Division of Clinical Pharmacology and Therapeutics and Director of Clinical and Translational Research at the Children’s Hospital of Philadelphia (CHOP). For 8 years Dr. Adamson led the National Cancer Institute’s Children’s Oncology Group (COG) Developmental Therapeutics Program, and currently he is co-Director
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Rare Diseases and Orphan Products: Accelerating Research and Development of the University of Pennsylvania–CHOP Clinical Translational Science Award (CTSA). Dr. Adamson was recruited from the National Cancer Institute (NCI) where he was an Investigator in the Pharmacology and Experimental Therapeutics Section of the Pediatric Oncology Branch. He was a member of the IOM committee on shortening the timeline for new cancer treatments and co-edited the report Making Better Cancer Drugs for Children (2005). Carolyn Asbury, Sc.M.P.H., Ph.D., is a Senior Consultant at the Dana Foundation (a New York-based non-profit that supports translational and clinical research in neuroscience, immunology, and neuroimmunology) and is also an adjunct Senior Fellow, Leonard Davis Institute of Health Economics at the University of Pennsylvania. She has served as Vice Chair and Chair of the Board of the National Organization for Rare Disorders, is a member of the Board of the U.S. Pharmacopeia, and is a Trustee of the College of Physicians of Philadelphia. Dr. Asbury has a master’s degree in public health and a doctorate in health systems business. She served as an advisor on market exclusivity, tax credit, and regulatory provisions of the 1983 Orphan Drug Act and subsequently authored the book Orphan Drugs: Medical versus Market Value. She has also authored several journal articles and book chapters on orphan drug issues and policies. Prior to her role at Dana, she was Senior Program Officer at the Robert Wood Johnson Foundation and then Director of the Health and Human Services Program at the Pew Charitable Trusts. Paul Citron, M.S.E.E., retired in 2003 as Vice President of Technology Policy and Academic Relations at Medtronic, Inc., where he was previously Vice President of Science and Technology. He had responsibility for corporate-wide assessment and coordination of technology and for establishing and prioritization of corporate research. Currently he is adjunct professor at the Jacobs School of Engineering, University of California San Diego and an advisor to the Harvard-MIT Division of Health Sciences and Technology. He is also an advisor to several firms in the biotechnology sector. Mr. Citron has a B.S. and M.S. in electrical engineering. He was elected a Founding Fellow of the American Institute of Medical and Biological Engineering. He has authored numerous publications and holds eight U.S. medical device patents. He is a member of the National Academy of Engineering (NAE) and serves on the advisory group of the NAE’s Center for Engineering, Ethics, and Society. He served on the IOM committee on postmarket surveillance of pediatric medical devices. Peter B. Corr, Ph.D., is a Founder and General Partner of Celtic Therapeutics LLLP, a private equity firm focused on the development of innovative
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Rare Diseases and Orphan Products: Accelerating Research and Development therapeutics, the development of alliances that advance solutions for diseases of the developing world, and global advocacy for biomedical innovation. Dr. Corr retired from Pfizer Inc. at the end of 2006, where he served as Senior Vice President for science and technology. Before that, he served as Executive Vice President of Pfizer Global Research and Development and President of Worldwide Development. Prior to joining Pfizer in 2000, Dr. Corr was President of pharmaceutical research and development at Warner Lambert/Parke Davis (until the merger with Pfizer), and he previously served as Senior Vice President of discovery research at Monsanto/Searle. Dr. Corr also spent 18 years as a researcher in molecular biology and pharmacology at Washington University in St. Louis, Missouri, where he was a professor of medicine (cardiology) and a professor of pharmacology and molecular biology. Dr. Corr serves on the Board of Governors of the New York Academy of Sciences, the Board of Regents of Georgetown University, and several other nonprofit and for-profit boards. He is also a member of the IOM Forum on Drug Discovery, Development, and Translation and served on the IOM committee on conflict of interest in medical research, education, and practice. Michael DeBaun, M.D., is Director of the Sickle Cell Medical Treatment and Education Center at St. Louis Children’s Hospital and Professor of Pediatrics, Biostatistics, and Neurology, Washington University in St. Louis. Dr. DeBaun’s research has focused on understanding the etiology, pathogenesis, and management of cerebrovascular injury in children with sickle cell disease. He was among the first clinical investigators to carefully document the epidemiology, cognitive and clinical significance of silent cerebral infarcts in children with sickle cell anemia and to demonstrate that both size and location of cerebral infarcts result in specific cognitive loss in children. These studies subsequently led to the basis of the first international clinical trial in sickle cell disease, Silent Cerebral Infarct Multi-Center Trial. In oncology, Dr. DeBaun has focused on understanding the epidemiology, optimal management and molecular basis for overgrowth syndromes associated with cancer in children, specifically Beckwith Wiedemann Syndrome (BWS). Dr. DeBaun established an international BWS registry. The clinical work has been coupled with molecular genetic analysis documenting phenotype and epigenotype correlations in BWS. Dr. DeBaun and his colleagues were the first to describe the association between in vitro fertilization (IVF), congenital malformation syndromes, and epigenotype mutations in children born after IVF. Harry Dietz, M.D., is Victor A. McKusick Professor of Genetics and Medicine in the Institute of Genetic Medicine and the Departments of Pediatrics, Medicine, and Molecular Biology and Genetics, Johns Hopkins University
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Rare Diseases and Orphan Products: Accelerating Research and Development School of Medicine. He is Director of the University’s William S. Smilow Center for Marfan Syndrome Research and Investigator, Howard Hughes Medical Institute. Dr. Dietz studies how blood vessel walls develop and are maintained with a focus on processes that contribute to inherited forms of cardiovascular disease. His work on Marfan syndrome, a rare and potentially fatal connective tissue disease, has led him from discovery of the molecular basis of the disease to a current clinical trial of a surprising potential treatment: a medication used to treat high blood pressure. He has received awards from the Society for Pediatric Research, the American Society of Human Genetics, and the National Marfan Foundation. Dr. Dietz is a member of the Institute of Medicine. Ellen J. Flannery, J.D., is a partner at Covington & Burling LLP, Washington, DC. She advises clients regarding the regulation of medical devices, pharmaceuticals, and biological products, as well as on product liability law. She has chaired the American Bar Association (ABA) Section of Science & Technology Law and the ABA Coordinating Group on Bioethics and the Law. Ms. Flannery has been counsel in cases involving the scope of the Food and Drug Administration’s legal authority and has taught Food and Drug Law seminars at Boston University, University of Maryland, and University of Virginia Law Schools. She serves on the editorial boards of the Guide to Medical Device Regulation and the Food and Drug Administration Enforcement Manual and has published a number of articles related to medical device regulation. She served on the IOM committee on postmarket surveillance of pediatric medical devices. Pat Furlong, R.N., B.S.N., is the Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), the largest nonprofit organization in the United States solely focused on Duchenne muscular dystrophy (Duchenne). Its mission is to improve the treatment, quality of life, and long-term outlook for all individuals affected by Duchenne through research, advocacy, education, and compassion. Ms. Furlong is the mother of two sons who lost their battle with Duchenne in their teenage years. She is on the board of the Genetic Alliance and the Muscular Dystrophy Coordinating Committee at the U.S. Department of Health and Human Services, and she is also a committee member on the Collaboration in Education and Test Translation Program, which was developed by the NIH Office of Rare Diseases to promote the development and clinical and research use of tests for rare genetic diseases. She serves on the data safety monitoring board for both the Rare Diseases Clinical Research Network and Cooperative International Neuromuscular Research Group and on the Steering Committee for TREAT-NMD.
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Rare Diseases and Orphan Products: Accelerating Research and Development Marlene Haffner, M.D., M.P.H., is a consultant who has served as Executive Director of Regulatory Affairs at Amgen and as Director, Office of Orphan Products Development at the U.S. Food and Drug Administration from 1987 to 2006. She held the rank of Rear Admiral in the U.S. Public Health Service (PHS) and serves on the faculty of F. Edward Hébert School of Medicine, Uniformed Services University of the Health Sciences. She began her career in the PHS with service on the Navaho Reservation. Her medical training is in internal medicine, dermatology, and hematology. Dr. Haffner is an expert on rare disease research and treatment and international orphan product legislation. She has received awards or honors from the National Organization of Rare Disorders and the National Hemophilia Foundation. She served on the IOM Forum on Drug Discovery, Development, and Translation. Haiden Huskamp, Ph.D., is a Professor of Health Care Policy in the Department of Health Care Policy at Harvard Medical School. Her research focuses on prescription drug policy and the economics of the pharmaceutical industry, mental health policy, and the financing and utilization of end-of-life care. Dr. Huskamp has also developed a body of research on the impact of pharmacy management tools used to control drug costs. She recently completed a Career Development Award from the National Institute of Mental Health focused on the economics of psychotropic medications. She served on the IOM committee on pediatric palliative care. Anthony So, M.D., M.P.A., is Professor of the Practice of Public Policy Studies and Director, Program on Global Health and Technology Access at Duke University’s Terry Sanford Institute of Public Policy. Current projects include an NIH-funded study to conceptualize a technology trust in genomics, a study of U.S. tissue biobanking practices, a study on innovation and access to health technologies in developing countries, and a World Health Organization/World Alliance on Patient Safety report on research and development for health technologies combating antimicrobial resistance. Previously, Dr. So served as Associate Director of the Rockefeller Foundation’s Health Equity program, where he co-founded a program on intellectual property rights. Earlier, he served as Senior Advisor to the Administrator at the Agency for Health Care Policy and Research, U.S. Department of Health and Human Services and as a White House Fellow. A general internist by training, he also has an M.P.A. and completed a fellowship in the Robert Wood Johnson Clinical Scholars Program. He serves on the Advisory Board for TropIKA, a new web-based research and policy portal from the Special Programme for Research and Training in Tropical Diseases.
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Rare Diseases and Orphan Products: Accelerating Research and Development Robert D. Steiner, M.D., is Credit Unions for Kids Professor of Pediatric Research, Vice Chair for Research in Pediatrics, and, Faculty: Program in Molecular and Cellular Biosciences, Pediatrics, and Molecular and Medical Genetics at the Child Development and Rehabilitation Center/Doernbecher Children’s Hospital, Oregon Health & Science University (OHSU), in Portland. Dr. Steiner is a pediatrician and medical geneticist who specializes in inborn errors of metabolism, along with clinical and research interests in cholesterol disorders, osteogenesis imperfecta, and autism. Dr. Steiner has led and participated in clinical research and clinical trials in many rare disorders and has conducted research with funding from NIH, industry, and private foundations under OHSU contract terms that protect data access and publication rights. He is board-certified in pediatrics and both clinical genetics and clinical biochemical genetics. Nancy S. Sung, Ph.D., is a Senior Program Officer with the Burroughs Wellcome Fund (BWF). She oversees BWF’s Interfaces in Science Programs and Clinical Scientist Awards in Translational Research. Dr. Sung has also focused on building collaboration among other private foundations, government agencies, and professional societies who share BWF’s interests in strengthening training and career pathways for researchers in the clinical research as well as for physical and computational scientists entering biology areas. She is founding board chair of the Health Research Alliance, a consortium of 40 foundations and public charities. Her research has focused on gene regulation in Epstein-Barr virus and its link to nasopharyngeal carcinoma. She was a member of the IOM Clinical Research Roundtable and is currently a member of the IOM Forum on Drug Discovery, Development, and Translation. She chaired a Forum recent workshop on the topic of drug development for rare diseases. Study Staff Marilyn J. Field, Ph.D. (Study Director), is a senior program officer at the Institute of Medicine (IOM). Her recent projects at IOM have examined conflicts of interest in medical research, education, and practice and the safety of medical devices for children. Among earlier projects, she has directed three studies of the development and use of clinical practice guidelines, two studies of palliative and end-of-life care, and Congressionally requested studies of employment-based health insurance and Medicare coverage of preventive services. Past positions include Associate Director of the Physician Payment Review Commission, Executive Director for Health Benefits Management at the Blue Cross and Blue Shield Association, and Assistant Professor of Public Administration at the Maxwell School of Citi-
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Rare Diseases and Orphan Products: Accelerating Research and Development zenship and Public Affairs, Syracuse University. Her doctorate in political science is from the University of Michigan, Ann Arbor. Claire F. Giammaria, M.P.H. (from August 2010), is a research associate for the Board on Health Sciences Policy. Before joining the Institute of Medicine, she was the research associate for the Technology and Liberty Program at the ACLU’s Washington Legislative Office where she primarily worked on issues concerning genetics and privacy. Ms. Giammaria received her master’s degree from the Department of Health Management and Policy of the University of Michigan, Ann Arbor, and a certificate in public health genetics. Ms. Giammaria received her B.A. in biology from Grinnell College. Erin S. Hammers, M.P.H. (until May 2010), was a research associate on the Board on Health Sciences Policy at the Institute of Medicine. She completed her master’s of public health degree at Columbia University with a focus on socio-medical sciences and health promotion. Ms. Hammers is now a student at Georgetown University Law Center, pursuing her J.D. Robin E. Parsell is a senior program assistant for the Board on Health Sciences Policy. Her recent project at the IOM examined the conflict of interest in medical research, education, and practice. Before joining the Institute of Medicine, she gained 3 years of community-based preparatory research experience with special populations as a project director at the Johns Hopkins University Center on Aging and Health and other applied research experience at the Pennsylvania State University. Ms. Parsell graduated with a B.S. in biology (focus in molecular genetics and biochemistry) and a certificate in gerontology from the University of Alabama at Birmingham.
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