Sharing Clinical Research Data: Workshop Summary (2013)

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C Participant Biographies Sharon F. Terry, M.A. (Workshop Chair), is president and CEO of the Genetic Alliance, a network of more than 10,000 organizations, 1,200 of which are disease advocacy organizations. Genetic Alliance improves health through the authentic engagement of communities and individuals. It develops innovative solutions through novel partnerships, connecting consumers to smart services. She is the founding CEO of PXE Interna- tional, a research advocacy organization for the genetic condition pseudoxanthoma elasticum (PXE). As codiscoverer of the gene associat- ed with PXE, she holds the patent for ABCC6 and has assigned her rights to the foundation. She developed a diagnostic test and is conducting clin- ical trials. Ms. Terry is also a cofounder of the Genetic Alliance Registry and Biobank. She is the author of more than 90 peer-reviewed articles. In her focus at the forefront of consumer participation in genetics research, services, and policy, she serves in a leadership role on many of the major international and national organizations, including the Institute of Medi- cine (IOM) Health Sciences Policy Board, the National Coalition for Health Professional Education in Genetics board, and the International Rare Disease Research Consortium Interim Executive Committee. She is a member of the IOM Roundtable on Translating Genomic-Based Re- search for Health. She is on the editorial boards of several journals. She was instrumental in the passage of the Genetic Information Non- discrimination Act. In 2005, she received an honorary doctorate from Iona College for her work in community engagement; the first Patient Service Award from the University of North Carolina Ins- titute for Pharmacogenomics and Individualized Therapy in 2007; the Research!America Distinguished Organization Advocacy Award in 105 

106 SHARING CLINICAL RESEARCH DATA 2009; and the Clinical Research Forum and Foundation’s Annual Award for Leadership in Public Advocacy in 2011. She is also an Ashoka Fellow. Myles Axton, Ph.D., is editor of Nature Genetics. He was a university lecturer in molecular and cellular biology at the University of Oxford and a fellow of Balliol College from 1995 to 2003. He obtained his degree in genetics at Cambridge in 1985, and his doctorate at Imperial College in 1990. Between 1990 and 1995, he did postdoctoral research at Dundee and at the Massachusetts Institute of Technology’s (MIT’s) Whitehead Institute. His research made use of the advanced genetics of Drosophila to study genome stability by examining the roles of cell cycle regulators in life cycle transitions. His interests broadened into human genetics, genomics, and systems biology through lecturing and from tutoring bio- chemists, zoologists, and medical students from primary research papers. Helping to establish Oxford’s innovative research M.Sc. in integrative biosciences led Dr. Axton to realize the importance of the integrative overview of biomedical research. As a full-time professional editor, he is now in a position to use this perspective to help coordinate research in genetics. Jesse A. Berlin, Sc.D., spent 15 years as a faculty member at the Univer- sity of Pennsylvania, in the Center for Clinical Epidemiology and Biosta- tistics, under the direction of Dr. Brian Strom. He left the University of Pennsylvania to join Janssen Research & Development, where he is cur- rently vice president of epidemiology. He has authored or coauthored more than 230 publications in a wide variety of clinical and methodolog- ical areas, including papers on the study of meta-analytic methods as ap- plied to both randomized trials and epidemiology. He served on an Institute of Medicine committee that developed recently released rec- ommendations for the use of systematic reviews in clinical effectiveness research. He currently serves on the Scientific Advisory Committee to the Observational Medical Outcomes Partnership, a public-private part- nership aimed at understanding methodology for assessing drug safety in large administrative databases. He is also a fellow of the American Sta- tistical Association. Josephine P. Briggs, M.D., is director of the National Center for Com- plementary and Alternative Medicine (NCCAM), and acting director, Division of Clinical Innovation, National Center for Advancing Transla- 

APPENDIX C 107 tional Sciences, National Institutes of Health (NIH). An accomplished researcher and physician, Dr. Briggs received her A.B. in biology from Harvard–Radcliffe College and her M.D. from Harvard Medical School. She completed her residency training in internal medicine and nephrolo- gy at the Mount Sinai School of Medicine, followed by a fellowship at Yale University. She then worked as a research scientist at the Physiolo- gy Institute at the University of Munich. In 1985, Dr. Briggs moved to the University of Michigan, where she held several academic positions, including associate chair for research in the Department of Internal Med- icine and professorships in the Division of Nephrology, Department of Internal Medicine, and the Department of Physiology. She joined the NIH in 1997 as director of the Division of Kidney, Urologic, and Hema- tologic Diseases at the National Institute of Diabetes and Digestive and Kidney Diseases. In 2006, Dr. Briggs accepted a position as senior scien- tific officer at the Howard Hughes Medical Institute. In 2008, she re- turned to the NIH as director of the NCCAM. Dr. Briggs has published more than 175 research articles, book chapters, and scholarly publica- tions. She has served on the editorial boards of several journals, and was deputy editor for the Journal of Clinical Investigation. Dr. Briggs is an elected member of the American Association of Physicians and the American Society of Clinical Investigation and a fellow of the American Association for the Advancement of Science. She is a recipient of many awards and prizes, including the Volhard Prize of the German Nephrological Society, the Alexander von Humboldt Scientific Exchange Award, and NIH Director’s Awards for her role in the development of the Trans-NIH Type I Diabetes Strategic Plan and her leadership of the Trans-NIH Zebrafish committee. Dr. Briggs is also a member of the NIH Steering Committee, the highest governing board at the NIH. Robert M. Califf, M.D., is the vice chancellor for Clinical and Transla- tional Research, director of the Duke Translational Medicine Institute (DTMI), and professor of medicine in the Division of Cardiology at Duke University Medical Center in Durham, North Carolina. He leads a multifaceted organization that seeks to transform how scientific discover- ies are translated into improved health outcomes. Prior to leading the DTMI, he was the founding director of the Duke Clinical Research Insti- tute (DCRI). He is editor in chief of the American Heart Journal, the oldest cardiovascular specialty journal, and a practicing cardiologist at Duke University Medical Center. Dr. Califf attended Duke University, graduating summa cum laude and Phi Beta Kappa. He remained at Duke 

108 SHARING CLINICAL RESEARCH DATA for medical school, where he was selected for the Alpha Omega Alpha medical honor society. After graduating from Duke University School of Medicine, he completed a residency in internal medicine at the Universi- ty of California, San Francisco, then returned to Duke for a cardiology fellowship. Dr. Califf is board certified in internal medicine and cardiol- ogy, and was named a Master of the American College of Cardiology in 2006. An international leader in the fields of cardiovascular medicine, health care outcomes, quality of care, and medical economics, he has authored or coauthored more than 1,000 peer-reviewed articles and is among the most frequently cited authors in medicine. He is also a con- tributing editor for TheHeart.org, an online information resource for health care professionals working in the field of cardiovascular medicine. As founder and a decade-long director of the DCRI, Dr. Califf led many landmark clinical trials and health services research projects, and remains actively involved in designing, leading, and conducting multinational clinical trials. Under his guidance, DCRI grew into an organization with more than 1,000 employees and an annual budget of more than $100 mil- lion; its umbrella organization, the DTMI, now has an annual budget of more than $300 million. Supported in part by a Clinical and Translational Science Award (CTSA) from the National Institutes of Health, the DTMI works with government agencies, academic partners, research founda- tions, and the medical products industry to conduct innovative research spanning multiple therapeutic arenas and scientific disciplines. Dr. Califf serves as co-chair of the first Principal Investigators Steering Committee of the CTSA. He has served on the Food and Drug Administration’s (FDA’s) Cardiorenal Advisory Panel, and on the Institute of Medicine’s Committee on Identifying and Preventing Medication Errors, and Com- mittee on Nutritional Biomarkers. In 2008, he was part of the subcom- mittee of the FDA’s Science Board that recommended sweeping reform of the agency’s science base. He was also a member of the IOM commit- tees that recommended Medicare coverage of clinical trials and the re- moval of ephedra from the market. Dr. Califf is currently a member of the Institute of Medicine (IOM) Forum on Drug Discovery, Develop- ment, and Translation and a member of the National Advisory Council on Aging. Reflecting his interests in health care quality, Dr. Califf was the founding director of the coordinating center of the Centers for Educa- tion & Research on Therapeutics, a public-private partnership that seeks to improve the use of medical products through research and education. He is currently co-chair of the Clinical Trials Transformation Initiative, a public-private partnership focused on improving the clinical trials sys- 

APPENDIX C 109 tem. He is also chair of the Clinical Research Forum, an organization of academic health and science system leaders devoted to improving the clinical research enterprise. Michael N. Cantor, M.D., M.A., FACP, is senior director, Information Strategy and Analytics, in Pfizer’s Clinical Informatics and Innovation group. His work focuses on leveraging data reuse and integration to sup- port future horizons of scientific decision support for precision medicine. He is currently coleading several initiatives around the secondary use of clinical data, including Pfizer’s ePlacebo/eControls database, as well as its comprehensive Clinical Lab Data Catalog. He created and coleads the MEDIC (Multisite Electronic Data Infectious Disease Consortium) pro- ject, which aims to partner with academic medical centers to perform observational studies using data from electronic medical record (EMR) systems. He has served as an advisor to programs across each of Pfizer’s Business Units, as well as the Worldwide Research and Development organization, on the role of health care IT in advancing their strategic priorities. Dr. Cantor previously led Pfizer Business Technology’s “Data Without Borders” strategy, with the aim of advancing data sharing and reuse, both internally and externally, to advance Precision Medicine. He has been a member of American Medical Informatics Association’s pub- lic policy committee for 6 years, and led the committee’s initiative to update its positions on data stewardship and reuse. Prior to joining Pfiz- er, Dr. Cantor was the chief medical information officer for the South Manhattan Healthcare Network of the New York City Health and Hospi- tals Corporation, based at Bellevue Hospital in Manhattan. His work there focused on developing the network’s EMR system to improve pa- tient safety and on using the network’s clinical data warehouse for re- search. He continues to see patients at Bellevue, and is a clinical assistant professor of medicine at the New York University School of Medicine. Dr. Cantor completed his residency in Internal Medicine and Informatics Training at Columbia. He has an M.D. from Emory University and an A.B. from Princeton. Carolyn Compton, M.D., Ph.D., is the president and chief executive officer of Critical Path Institute. She was most recently the director of the Office of Biorepositories and Biospecimen Research and the executive director of the Cancer Human Biobank project at the National Cancer Institute (NCI). In these capacities, she had leadership responsibility for strategic initiatives that included the Innovative Molecular Analysis 

110 SHARING CLINICAL RESEARCH DATA Technologies for Cancer program; the Biospecimen Research Network program; and the NCI Community Cancer Centers project. She is an ad- junct professor of pathology at the Johns Hopkins School of Medicine. She received her M.D. and Ph.D. from Harvard Medical School and the Harvard Graduate School of Arts and Sciences. She trained in pathology at Harvard’s Brigham and Women’s Hospital and is boarded in both An- atomic Pathology and Clinical Pathology. She came to the NCI from McGill University, where she had been the Strathcona Professor and Chair of Pathology and the pathologist in chief of McGill University Health Center from 2000 to 2005. Prior to this, she had been a professor of pathology at Harvard Medical School, the director of gastrointestinal pathology at the Massachusetts General Hospital, and the pathologist in chief of the Shriners Hospital for Crippled Children, Boston Burns Unit for 15 years. During this time she served as the chair of the Pathology Committee of the Cancer and Leukemia Group B for 12 years. Her re- search interests are in colon and pancreatic cancer as well as epithelial biology and wound healing. Dr. Compton has held many national and international leadership positions in pathology and cancer-related profes- sional organizations. She is a fellow of the College of American Pathologists (CAP) and a fellow of the Royal Society of Medicine. Cur- rently, she is chair of the American Joint Committee on Cancer, serves on the Executive Committee of the Commission on Cancer (COC) of the American College of Surgeons, and serves as the pathology section edi- tor for Cancer. She is a past chair of the Cancer Committee of the Col- lege of American Pathologists and was editor of the first edition of the CAP Cancer Protocols (Reporting on Cancer Specimens) used as stand- ards for COC accreditation. Among her awards are the International So- ciety for Biological and Environmental Repositories Award for Outstanding Achievement in Biobanking, the National Institutes of Health (NIH) Director’s Award, the NIH Award of Merit, and the CAP Frank W. Hartman Award. She has published more than 500 original scientific papers, reports, review articles, and books. Neil de Crescenzo, M.B.A., is senior vice president and general manag- er for Health Sciences at Oracle. He is responsible for managing Oracle’s solution groups, strategic planning, product development, and sales, ser- vice, and support for the industry solutions sold into the health care and life sciences markets worldwide. Mr. de Crescenzo brings more than 20 years of operational and information technology (IT) leadership across health care and life sciences to his work with customers and partners 

APPENDIX C 111 worldwide. Prior to joining Oracle, Mr. de Crescenzo held a number of leadership positions during his decade at IBM Corporation, working with health care and life sciences clients throughout the world. Prior to enter- ing the IT industry, he held leadership positions in health care operations at multiple medical centers and a major health insurer. Mr. de Crescenzo began his career in investment banking, working with U.S. and European clients in the areas of corporate finance and mergers and acquisitions. Mr. de Crescenzo has been a keynote speaker at numerous industry con- ferences worldwide and is quoted frequently on industry issues. In 2005, he was named one of the “Top 25 Most Influential Consultants” by Con- sulting magazine. Mr. de Crescenzo has a B.A. in political science from Yale University and an M.B.A. in high technology from Northeastern University. Peter Doshi, Ph.D., is a postdoctoral fellow in comparative effectiveness research at the Johns Hopkins University School of Medicine. His over- arching research interests are in improving the basis for credible evi- dence synthesis to support and improve the quality of evidence-based medical and health policy–related decision making. In 2009, he joined a Cochrane systematic review team evaluating neuraminidase inhibitors for the treatment and prevention of influenza. Rather than focusing on publications, the review evaluates regulatory information, including clin- ical study reports. He received his A.B. in anthropology from Brown University, A.M. in East Asian studies from Harvard University, and Ph.D. in history, anthropology, and science, technology and society from the Massachusetts Institute of Technology. Kelly Edwards, Ph.D., is an associate professor in the University of Washington (UW) School of Medicine’s Department of Bioethics and Humanities. Dr. Edwards also is a core faculty member for the UW Insti- tute for Public Health Genetics. She received both her M.A. in medical ethics and her Ph.D. in philosophy of education from the UW. Dr. Edwards’ work incorporates communication and public engagement as an ethical obligation for clinicians and researchers. She is the director of the Ethics and Outreach Core for the UW Center for Ecogenetics and Environmental Health, which is funded by the National Institute of Envi- ronmental Health Sciences. She also is a codirector of the Regulatory Support and Bioethics Core for the Institute for Translational Health Sci- ences (ITHS), a partnership of the UW, Fred Hutchinson Cancer Re- search Center, Seattle Children’s, and other regional institutions and 

112 SHARING CLINICAL RESEARCH DATA community and tribal groups. Funded by the National Institutes of Health (NIH), the ITHS assists researchers with translating their scientific dis- coveries into practice. In addition, Dr. Edwards is a lead investigator with the UW Center for Genomics and Healthcare Equality, funded by the NIH’s National Human Genome Research Institute. Since 2004, she has been the faculty advisor for the Forum on Science, Ethics and Policy, groups of graduate and professional students and postdoctoral fellows at the UW and University of Colorado who promote dialogue on issues concerning science and society. To further engage people in conversa- tions about ethical dimensions of science and medicine, Dr. Edwards has facilitated Community Conversations and the Public Health Café, a se- ries of events hosted in Seattle by the Northwest Association for Bio- medical Research. Dr. Edwards contributes to issues of ethical research practices with the Genetic Alliance, a health advocacy organization; Sage Bionetworks, a local nonprofit; and the Institute of Medicine. Her cours- es include “Inquiry-Based Science Communication,” “Applied Research Ethics,” “Community-Based Participatory Research: A Model for Genet- ics Research with Native American Communities?” and “Public Com- mentary on Ethical Issues in Public Health Genetics.” She is associate editor of BMC Medical Research Methodology and a reviewer for several journals. Dr. Edwards serves on the UW School of Medicine’s Continu- ous Professional Improvement Committee and is a former member of Medicine’s Standing Committee on Issues of Women Faculty, the Stu- dent Progress Committee, and the Committee on Research and Graduate Education. She is a current member of the UW Graduate School Com- mittee on Interdisciplinary Education. Hans-Georg Eichler, M.D., M.Sc., is the senior medical officer at the European Medicines Agency (EMA) in London, where he is responsible for coordinating activities among the EMA’s scientific committees and advising on scientific and public health issues. In 2011, Dr. Eichler was the Robert E. Wilhelm Fellow at the Massachusetts Institute of Technol- ogy (MIT) Center for International Studies, participating in a joint re- search project under the MIT’s New Drug Development Paradigms initiative. He divided his time between the MIT and the EMA in London. Prior to joining the EMA, Dr. Eichler was at the Medical University of Vienna in Austria for 15 years. He was vice rector for Research and In- ternational Relations since 2003, and professor and chair of the Depart- ment of Clinical Pharmacology since 1992. His other previous positions include president of the Vienna School of Clinical Research and co-chair 

APPENDIX C 113 of the Committee on Reimbursement of Drugs of the Austrian Social Security Association. His industry experience includes time spent at Ciba-Geigy Research Labs in the United Kingdom, and Outcomes Re- search at Merck & Co. in New Jersey. Dr. Eichler graduated with an M.D. from Vienna University Medical School and an M.Sc. in toxicolo- gy from the University of Surrey in Guildford, United Kingdom. He trained in internal medicine and clinical pharmacology at the Vienna University Hospital as well as at Stanford University. Ron Fitzmartin, Ph.D., M.B.A., is senior advisor, Office of Planning and Informatics, Center for Drug Evaluation and Research, Food and Drug Administration (FDA). Prior to joining FDA, Dr. Fitzmartin held scientific and technical leadership positions at Decision Analytics, Daiichi Sankyo, Inc., Daiichi Medical Research, Inc., and Purdue Pharma L.P. In addition, he served as statistician at both the U.S. Census Bureau and the U.S. Department of the Navy. Dr. Fitzmartin was elected a member of the Board of Directors and President of the Drug Infor- mation Association from 2007 to 2009. Dr. Fitzmartin has been a fre- quent presenter at many industry meetings and has authored numerous articles in areas such as informatics, pharmacovigilance, clinical data management, regulatory compliance, and R&D strategy. Dr. Fitzmartin received a Ph.D. in statistics from the University of Maryland, an M.B.A. from the University of New Haven, and an M.S. and B.S. from Southern Connecticut State University. Jennifer S. Geetter, J.D., is a partner in the law firm of McDermott Will & Emery LLP and is based in the firm’s Washington, DC, office. She focuses her practice on emerging biotechnology and safety issues, advis- ing hospital, industry, insurance, and provider clients on matters relating to research, drug and device development, off-label use, personalized medicine, formulary compliance, privacy and security, electronic health records and data strategy initiatives, patient safety, conflicts of interest, scientific review and research misconduct, internal hospital disciplinary proceedings, and emerging issues in secondary research concerning bio- logical samples and data warehousing. She also assists health care clients in implementing research strategies, structuring research operational and compliance infrastructure, and developing guidelines for the appropriate relationships between providers and industry. She is a frequent speaker on these topics. Ms. Geetter is a member of the firm’s Life Sciences Af- finity Group and Personalized Medicine Team. She is also a co-chair of 

114 SHARING CLINICAL RESEARCH DATA the Pro Bono and Community Service Committee for the Washington, DC, office. She sits on McDermott’s National Pro Bono Committee and the American Health Lawyers Association Life Sciences Section Steer- ing Committee. Ms. Geetter is listed as a leading individual in health care in Washington, DC, in Chambers USA 2008: America’s Leading Lawyers for Business. She was recognized as a STAR Mentor in the McDermott University Mentoring Program for 2006-2007. She is also a member of McDermott’s Gender Diversity Committee. In 2003, Ms. Geetter was awarded the ACE Founders Award for her pro bono efforts as part of a team of McDermott lawyers on behalf of a group of low- income residents of a Boston neighborhood. She received the firm’s Out- standing Achievement Award for Commitment to Pro Bono and Service to the Community in 2004 and serves on the firm’s national coordinating committee for pro bono activities. Ms. Geetter is admitted to practice in Massachusetts, New York, and Washington, DC. Steven N. Goodman, M.D., M.H.S., Ph.D., is professor of medicine and health policy, and research and associate dean for clinical and transla- tional research at Stanford University. Before joining Stanford in 2011, Dr. Goodman spent two decades on the Johns Hopkins medical faculty as professor of oncology in the division of biostatistics, with appointments in the departments of Pediatrics, Biostatistics, and Epidemiology in the Johns Hopkins Schools of Medicine and Public Health. He has been edi- tor of Clinical Trials: Journal of the Society for Clinical Trials since 2004 and senior statistical editor for the Annals of Internal Medicine since 1987. He has served on a wide range of Institute of Medicine committees, including Agent Orange and Veterans, Immunization Safety, the Committee on Alternatives to the Daubert Standards, Treatment of PTSD (Posttraumatic Stress Disorder) in Veterans, and most recently co- chaired the Committee on Ethical and Scientific Issues in Studying the Safety of Approved Drugs, whose report was released in 2012. Dr. Goodman was appointed by the Government Accountability Office to serve on the Methodology Committee of the Patient Centered Outcomes Research Institute and is a scientific advisor to the Medical Advisory Panel of the National Blue Cross/Blue Shield Technology Evaluation Center. He served on the Surgeon General’s committee to write the 2004 report on the health consequences of smoking. Dr. Goodman received a B.A. from Harvard; an M.D. from New York University; trained at Washington University in pediatrics, in which he was board certified; and received an M.H.S. in biostatistics and a Ph.D. in epidemiology from 

APPENDIX C 115 Johns Hopkins University. He writes and teaches on evidence evaluation and inferential, methodologic, and ethical issues in epidemiology and clinical research. Robert A. Harrington, M.D., is the Arthur L. Bloomfield Professor of Medicine and chair of the Department of Medicine at Stanford Universi- ty. He received his undergraduate degree in English from the College of the Holy Cross in Worcester, MA. He attended Dartmouth Medical School and received his M.D. from Tufts University School of Medicine in 1986. He was an intern, resident, and the chief medical resident in in- ternal medicine at the University of Massachusetts Medical Center. He was a fellow in cardiology at Duke University Medical Center, where he received training in interventional cardiology and research training in the Duke Databank for Cardiovascular Diseases. Dr. Harrington was previ- ously the director of the Duke Clinical Research Institute. His research interests include evaluating antithrombotic therapies to treat acute is- chemic heart disease and to minimize the acute complications of percu- taneous coronary procedures; studying the mechanism of disease of the acute coronary syndromes; understanding the issue of risk stratification in the care of patients with acute ischemic coronary syndromes; and trying to better understand and improve on the methodology of clinical trials. He is the recipient of a National Institutes of Health Roadmap contract to investigate “best practices” among clinical trial networks. He has authored more than 400 peer-reviewed manuscripts, reviews, book chapters, and editorials. He is an associate editor of the American Heart Journal and an editorial board member for the Journal of the Ameri- can College of Cardiology. He is a senior editor of the 13th edition of Hurst’s The Heart. He is a fellow of the American College of Cardiology, the American Heart Association, the European Society of Cardiology, the So- ciety of Cardiovascular Angiography and Intervention, and the American College of Chest Physicians. He recently served as a member and the chair of the Food and Drug Administration Cardiovascular and Renal Drugs Advisory Committee. Lynn D. Hudson, Ph.D., serves as the chief science officer for the Criti- cal Path Institute and executive director of the Multiple Sclerosis Consor- tium. She received a B.S. in biochemistry at the University of Wisconsin, a Ph.D. in genetics and cell biology at the University of Minnesota, and postdoctoral training at Harvard Medical School and Brown University. For most of her career, Dr. Hudson has been a bench neuroscientist at the 

116 SHARING CLINICAL RESEARCH DATA National Institutes of Health (NIH), where she directed the Office of Sci- ence Policy Analysis from 2006 to 2011. As a major source for policy analysis within the NIH Office of the Director, her office covered a wide spectrum of sensitive and emerging issues and oversaw a number of pro- grams, including the American Association for the Advancement of Sci- ence/NIH Science Policy Fellowship program, the NIH’s contract with the National Academy of Sciences, and the Public–Private Partnership Program. Her policy team’s awards cite contributions to the NIH’s con- gressional justification, biennial report, implementation of the NIH Re- form Act, stem cell guidelines, and comparative effectiveness research. Dr. Hudson has conducted research in the National Institute of Neurolog- ical Disorders and Stroke (NINDS) intramural program, where she was chief of the Section of Developmental Genetics for 16 years. She re- ceived the NIH Merit Award for her discovery of the causative mutations in the neurologic disorder Pelizaeus-Merzbacher Disease (PMD), and an NINDS Award for educational outreach efforts. Her research focused on defining the network of genes involved in the development of glial cells, with the goal of designing strategies to overcome glial dysfunction in inherited or acquired neurological diseases. She served as an officer for the American Society for Neurochemistry, as an officer on the scientific advisory board of the PMD Foundation, and as an advisor for a number of granting agencies and disease foundations, including the National Multiple Sclerosis Society. Charles Hugh-Jones, M.D., is vice president and head, Medical Affairs North America, Oncology, Hematology, and Solid Organ Transplant at Sanofi. He has previously served as vice president and head, Oncology Medical Affairs, North America, for Sanofi-Aventis; vice president of brand management and vice president of medical affairs at Enzon Phar- maceuticals; executive director, Medical Affairs, at Schering AG/ Berlex/Bayer-Schering; director of medical affairs for Schering AG, Glob- al Business Unit; senior medical advisor for Schering AG, United King- dom; and specialist registrar at Hammersmith Hospital, United Kingdom. Dr. Hugh-Jones received his M.D. from the University of London. He is board certified in internal medicine and has additional Higher Specialist Training (United Kingdom) in diagnostic and interventional radiology. John P. A. Ioannidis, D.Sc., M.D., holds the C.F. Rehnborg Chair in Disease Prevention at Stanford University and is professor of medicine, professor of health research and policy, and director of the Stanford Pre- 

APPENDIX C 117 vention Research Center at Stanford University School of Medicine, pro- fessor of statistics (by courtesy) at Stanford University School of Hu- manities and Sciences, member of the Stanford Cancer Center and of the Stanford Cardiovascular Institute, and affiliated faculty of the Woods Institute for the Environment. From 1999 until 2010, he chaired the De- partment of Hygiene and Epidemiology at the University of Ioannina School of Medicine in Greece, where he had been a tenured professor since 2003. Dr. Ioannidis graduated in the top rank of his class from the School of Medicine, University of Athens, in 1990 and earned a doctor- ate in biopathology. He trained at Harvard and Tufts, specializing in in- ternal medicine and infectious diseases, and then held positions at the National Institutes of Health, Johns Hopkins University School of Medi- cine, and Tufts University School of Medicine before returning to Greece in 1999. He has been adjunct faculty for the Tufts School of Medicine since 1996, with the rank of professor since 2002. Since 2008 he led the Genetics/Genomics component of the Tufts Clinical and Translational Science Institute and the Center for Genetic Epidemiology and Modeling of the Tufts Institute for Clinical Research and Health Policy Studies at Tufts Medical Center. He is also an adjunct professor of epidemiology at the Harvard School of Public Health and a visiting professor of epidemi- ology and biostatistics at Imperial College London. Dr. Ioannidis is a member of the executive board of the Human Genome Epidemiology Network. He has served as president of the Society for Research Synthe- sis Methodology, as a member of the editorial board of 27 leading inter- national journals (including PLoS Medicine, Lancet, Annals of Internal Medicine, Journal of the National Cancer Institute, Science Translation- al Medicine, Molecular and Cellular Proteomics, AIDS, International Journal of Epidemiology, Journal of Clinical Epidemiology, Clinical Trials, Cancer Treatment Reviews, Open Medicine, and PLoS ONE), and as editor in chief of the European Journal of Clinical Investigation from 2010 to 2014. He has given more than 250 invited and honorary lectures. He has received several awards, including the European Award for Ex- cellence in Clinical Science for 2007, and was inducted into the Associa- tion of American Physicians in 2009 and into the European Academy of Cancer Sciences in 2010. His PLoS Medicine paper “Why Most Pub- lished Research Findings Are False” has been the most accessed article in the history of the Public Library of Science. The Atlantic selected Ioannidis as the Brave Thinker scientist for 2010, claiming that he “may be one of the most influential scientists alive.” 

118 SHARING CLINICAL RESEARCH DATA Charles Jaffe, M.D., is the CEO of Health Level 7, where he serves as the organization’s global ambassador, fostering relationships with key industry stakeholders. A 37-year veteran of the health care IT industry, Dr. Jaffe was previously the senior global strategist for the Digital Health Group at Intel Corporation, vice president of life sciences at Science Ap- plications International Corporation, and director of medical informatics at AstraZeneca Pharmaceuticals. He completed his medical training at Johns Hopkins and Duke Universities, and was a postdoctoral fellow at the National Institutes of Health and at Georgetown University. Former- ly, he was president of InforMed, a consultancy for research informatics. Over the course of his career, he has been the principal investigator for more than 200 clinical trials, and has served in various leadership roles in American Medical Informatics Association. He has been a board member of leading organizations for information technology standards, and served as chair of a national institutional review board. Most recently, he held an appointment in the Department of Engineering at Penn State University. Dr. Jaffe has been the contributing editor for several journals and has published on a range of subjects, including clinical management, informatics deployment, and health care policy. Sachin H. Jain, M.D., M.B.A., is chief medical information and innova- tion officer (CMIO) at Merck and a lecturer in health care policy at Har- vard Medical School. He also serves as an attending hospitalist physician at the Boston VA-Boston Medical Center. In his capacity as Merck’s CMIO, Dr. Jain is working to establish and manage global alliances to use health data to improve medication safety, efficacy, use, and adher- ence. In addition, Dr. Jain is leading several efforts for new ventures and business development. Previously, he was senior advisor to the adminis- trator of the Centers for Medicare & Medicaid Services (CMS). At CMS, he helped lead the launch of the Center for Medicare & Medicaid Inno- vation, briefly serving as its acting deputy director for policy and pro- grams. At CMS, Dr. Jain advocated for speedier translation of health care delivery research into practice and an expanded use of clinical registries. Dr. Jain also served as special assistant to the National Coordinator for Health Information Technology at the Office of the National Coordinator for Health Information Technology (ONC). At ONC, Dr. Jain worked with David Blumenthal to implement the Health Information Technology for Economic and Clinical Health provisions of the American Recovery and Reinvestment Act that provide incentives for physicians and hospi- tals to become meaningful users of health information technology. He 

APPENDIX C 119 received his undergraduate degree magna cum laude in government from Harvard College; his M.D. from Harvard Medical School; and his M.B.A. from Harvard Business School. He was a recipient of the Paul and Daisy Soros Fellowship and the Dean’s Award at Harvard Business School. He completed his residency in internal medicine at Brigham and Women’s Hospital, where he was honored with the Resident Mentor Award. Dr. Jain is a founder of several nonprofit health care ventures, including the Homeless Health Clinic at the Harvard Square Homeless Shelter; the Harvard Bone Marrow Initiative; and ImproveHealthCare.org. He worked with DaVita-Bridge of Life to bring charity dialysis care to rural Rajasthan, India, and Medical Missions for Children to bring cleft lip and palate surgery to that region. He maintained a faculty appoint- ment at Harvard Business School’s Institute for Strategy and Competi- tiveness and worked with Strategy Professor Michael Porter on a new case literature on health care delivery innovation. He presently serves as an honorary senior institute associate at Harvard Business School. Dr. Jain has worked at WellPoint, McKinsey & Co., and the Institute for Healthcare Improvement. He has also served as an expert consultant to the World Health Organization. He has authored more than 50 publica- tions on health care delivery innovation and health care reform in journals such as the New England Journal of Medicine, JAMA, and Health Af- fairs. His work has been cited in the New York Times, CNN, the Wall Street Journal, and other media outlets. He serves on the editorial boards of the American Journal of Managed Care, Harvard Medicine, and Wing of Zock. The book he coedited with Susan Pories and Gordon Harper, The Soul of a Doctor, has been translated into Chinese. Beth Kozel, M.D., Ph.D., is an instructor of pediatrics in the Division of Genetics and Genomic Medicine at St. Louis Children’s Hospital and Washington University School of Medicine. Clinically, she cares for children with genetic conditions that include chromosomal changes, in- herited disorders, and multiple medical problems of unknown etiology. In her research, she has studied the biology of elastic fiber formation and works with patients with elastic fiber diseases such as Williams-Beuren syndrome, supravalvular aortic stenosis, and cutis laxa. Her current re- search is aimed at identifying modifiers of vascular disease severity among affected individuals. Her work is supported by a K08 award from the National Institutes of Health and she is a scholar of the Children’s Discovery Institute at Washington University. She is a member of the American Society of Matrix Biology and the American Society of Hu- 

120 SHARING CLINICAL RESEARCH DATA man Genetics. She is a registered researcher with the Williams Syndrome Patient and Clinical Research Registry. Harlan Krumholz, M.D., is the Harold H. Hines, Jr., Professor of Med- icine and director of the Robert Wood Johnson Clinical Scholars Pro- gram at Yale University School of Medicine, and director of the Yale- New Haven Hospital Center for Outcomes Research and Evaluation. His research focuses on determining optimal clinical strategies and identify- ing opportunities for improving the prevention, treatment, and outcome of cardiovascular disease. Using applied clinical research methods, his work seeks to provide critical information to improve the quality of health care, monitor changes over time, guide decisions about the alloca- tion of scarce resources, and inform decisions made by patients and their clinicians. Studies from his research group have directly led to improve- ments in the use of guideline-based medications, the timeliness of care for acute myocardial infarction, the information available to patients who are making decisions about clinical options, the public reporting of out- comes measures, and the current national focus on reducing the risk of readmission. He has also advanced the application of quality improve- ment to the elimination of disparities. His work influenced several provi- sions in the health reform bill. Dr. Krumholz serves on the board of trustees of the American College of Cardiology, the board of directors of the American Board of Internal Medicine, and the board of governors of the Patient-Centered Outcomes Research Institute. He is an elected member of the Association of American Physicians, the American Socie- ty for Clinical Investigation, and the Institute of Medicine. He is a Dis- tinguished Scientist of the American Heart Association and received its Distinguished Service Award. He received a B.S. from Yale, an M.D. from Harvard Medical School, and a master’s in health policy and man- agement from the Harvard University School of Public Health. Richard E. Kuntz, M.D., M.Sc., is senior vice president and chief sci- entific, clinical, and regulatory officer of Medtronic, Inc. In this role, which he assumed in 2009, Dr. Kuntz oversees the company’s global regulatory affairs, health policy and reimbursement, clinical research, ventures and new therapies, and innovation functions. Dr. Kuntz joined Medtronic in 2005 as senior vice president and president of medtronic neuromodulation, which encompasses the company’s products and ther- apies used in the treatment of chronic pain, movement disorders, spastici- ty, overactive bladder and urinary retention, benign prostatic hyperplasia, 

APPENDIX C 121 and gastroparesis. In this role, he was responsible for the research, devel- opment, operations, and product sales and marketing for each of these therapeutic areas worldwide. Dr. Kuntz brings to Medtronic a broad background and expertise in many areas of health care. Prior to Medtron- ic he was the founder and chief scientific officer of the Harvard Clinical Research Institute, a university-based contract research organization that coordinates the National Institutes of Health and industry clinical trials with the Food and Drug Administration. He has directed more than 100 multicenter clinical trials and authored more than 200 original publica- tions. His major interests are traditional and alternative clinical trial de- sign and biostatistics. He also served as associate professor of medicine at Harvard Medical School, chief of the Division of Clinical Biometrics, and an interventional cardiologist in the division of cardiovascular dis- eases at the Brigham and Women’s Hospital. Dr. Kuntz graduated from Miami University, and received his M.D. from Case Western Reserve University School of Medicine. He completed his residency in internal medicine at the University of Texas Southwestern Medical School, and then completed fellowships in cardiovascular diseases and interventional cardiology at the Beth Israel Hospital and Harvard Medical School. Dr. Kuntz received his M.S. in biostatistics from the Harvard School of Pub- lic Health. Rebecca D. Kush, Ph.D., is founder, president, and CEO of the Clinical Data Interchange Standards Consortium (CDISC). CDISC is a nonprofit standards-developing organization with a mission to develop and support global, platform-independent standards that enable information system interoperability to improve medical research and related areas of health care and a vision of “informing patient care and safety through higher quality medical research.” She has more than 25 years of experience in the area of clinical research, including positions with the National Insti- tutes of Health, academia, a global contract research organization, and biopharmaceutical companies in the United States and Japan. She earned her doctorate in physiology and pharmacology from the University of California, San Diego, School of Medicine. She is lead author of the book eClinical Trials: Planning and Implementation and has authored numerous publications for journals including New England Journal of Medicine and Science Translational Medicine. She has developed a Pre- scription Education Program for elementary and middle schools and was named in PharmaVoice in 2008 as one of the 100 most inspiring individ- uals in the life sciences industry. Dr. Kush has served on the board of 

122 SHARING CLINICAL RESEARCH DATA directors for the U.S. Health Information Technology Standards Panel, Drug Information Association, and currently HL7. She was a member of the advisory committee for the World Health Organization International Clinical Trials Registry Platform. Dr. Kush served on the appointed Planning Committee for the Department of Health and Human Services Office of the National Coordinator for Health Information Technology sponsored Workshop Series on the “Digital Infrastructure for the Learn- ing Health System” for the Institute of Medicine. She is a member of the National Cancer Advisory Board IT Workgroup and was invited to rep- resent research as an appointed member of the U.S. Health Information Technology Standards Committee. Dr. Kush has developed a course, “A Global Approach to Accelerating Medical Research,” and has been a keynote speaker at numerous conferences in this arena in Australia, Brazil, China, Europe, Japan, Korea, and the United States. Elizabeth Loder, M.D., M.P.H., is a senior research editor at BMJ. She is also chief of the Division of Headache and Pain in the Department of Neurology at Brigham and Women’s Hospital, and an associate professor of neurology at Harvard Medical School. She is board certified in inter- nal medicine and headache medicine. Dr. Loder is the current president of the American Headache Society and has held leadership positions in national and international pain organizations and served as a member of the recent Institute of Medicine Committee on Advancing Pain Care, Re- search, and Education. Dr. Loder has worked as a clinician and research- er in the headache field for many years, and has extensive experience in the design and conduct of clinical trials. As a result of these experiences, Dr. Loder is deeply interested in problems associated with clinical trial reporting and interpretation, especially the consequences of missing clin- ical trial information for guidelines and treatment decisions. She has de- voted much of her recent career to the development, implementation and dissemination of standards for research reporting. She was responsible for a recent BMJ theme issue on unpublished clinical trial data. Research published in that issue of the journal has led several U.S. lawmakers to question whether the National Institutes of Health and Food and Drug Ad- ministration are doing enough to enforce research reporting requirements. Deven McGraw, J.D., is the director of the Health Privacy Project at Center for Democracy and Technology (CDT). The Project focuses on developing and promoting workable privacy and security protections for electronic personal health information. Ms. McGraw is active in efforts 

APPENDIX C 123 to advance the adoption and implementation of health information tech- nology and electronic health information exchange to improve health care. She was one of three persons appointed by the Department of Health and Human Services (HHS) Secretary Kathleen Sebelius to serve on the Health Information Technology Policy Committee, a federal advi- sory committee established by the American Recovery and Reinvestment Act of 2009. She co-chairs the committee’s Privacy and Security “Tiger Team” and serves as a member of its Meaningful Use, Information Ex- change, and Strategic Plan Workgroups. She also served on two key workgroups of the American Health Information Community, the federal advisory body established by HHS to develop recommendations on how to facilitate use of health IT to improve health. Specifically, she co- chaired the Confidentiality, Privacy and Security Workgroup and was a member of the Personalized Health Care Workgroup. She also served on the Policy Steering Committee of the eHealth Initiative and now serves on its Leadership Council. She is also on the Steering Group of the Markle Foundation’s Connecting for Health multistakeholder initiative. Ms. McGraw has a strong background in health care policy. Prior to join- ing CDT, Ms. McGraw was the chief operating officer of the National Partnership for Women & Families, providing strategic direction and oversight for all of the organization’s core program areas, including the promotion of initiatives to improve health care quality. Ms. McGraw also was an associate in the public policy group at Patton Boggs, LLP, and in the health care group at Ropes & Gray. She also served as deputy legal counsel to the Governor of Massachusetts and taught in the Federal Leg- islation Clinic at the Georgetown University Law Center. Ms. McGraw graduated magna cum laude from the University of Maryland. She earned her J.D., magna cum laude, and her L.L.M. from Georgetown University Law Center and was executive editor of the Georgetown Law Journal. She also has an M.P.H. from Johns Hopkins School of Hygiene and Public Health. Meredith Nahm, Ph.D., provides oversight and coordination of clinical research informatics projects undertaken by the Core, including Duke Translational Medicine Institute’s provision of infrastructure for clinical research data collection and management. She also oversees informatics for the MURDOCK community registry and the Duke Clinical Research Unit, as well as involvement in national efforts to develop and implement data standards. Additionally, Dr. Nahm supports efforts to advance the application of informatics capabilities to enhance medical practice. 

124 SHARING CLINICAL RESEARCH DATA Through her work and associated collaborations, Dr. Nahm helps to shape Duke’s Clinical Research Informatics strategy and direction. Prior to her appointment as the associate director for clinical research infor- matics, Dr. Nahm served as the director of clinical data integration at the Duke Clinical Research Institute in 2001 and has been there since 1998. She has more than 15 years of experience in clinical research informatics and quality control. Dr. Nahm has held numerous industry leadership roles, including serving as a member of the Board of Trustees of the Society for Clinical Data Management (SCDM), chair of the SCDM Good Clinical Data Management Practices (GCDMP) Committee, chair of the Clin- ical Data Interchange Standards Consortium Industry Advisory Board, and the data management content expert for the National Institutes of Health (NIH)-sponsored National Leadership Forum for Clinical Re- search Networks. Dr. Nahm authored the GCDMP sections on Measur- ing and Assuring Data Quality and has played a leadership role in data standards development and implementation efforts. She is currently working with the data standards development efforts in cardiology and tuberculosis on two NIH Roadmap projects, and the Data and Statistical Center for the National Institute on Drug Abuse Clinical Trials Network. She is a frequent presenter at industry meetings, and publishes in the clinical trial operational literature. Her research interests include quanti- tative evaluation of common clinical data management practices and data quality. She received her master’s in nuclear engineering from North Carolina State University, and her Ph.D. at the University of Texas at Houston School of Health Information Sciences. Jeffrey S. Nye, M.D., Ph.D., is vice president, neuroscience innovation and partnership strategy, at Janssen Research & Development LLC, a Johnson & Johnson (J&J) pharmaceutical company. He is responsible for the strategy and implementation of external R&D in the neurosciences, aiming to expand the pipeline with novel business relationships, venture, academic and public-private partnerships, and risk-shared outsourcing. Previously, Dr. Nye was chief medical officer and co-head of the East Coast Research and Early Development unit at J&J pharma. Prior roles include vice president of experimental medicine, vice president of compound development for Galantamine (Razadyne/Reminyl), a therapy for Alzheimer’s disease, and for Topamax, an epilepsy and migraine medicine. He previously served as a director of CNS Discovery Genomics and Biotechnology at Pharmacia. Prior to joining the pharmaceutical industry, Dr. Nye was a tenured associate professor of 

APPENDIX C 125 molecular pharmacology, biological chemistry, and pediatrics (neu- rology) at Northwestern University and Children’s Memorial Hospital. He received his bachelor’s degree in biochemistry and master’s degree in pharmacology from Harvard, and his M.D. and Ph.D. (with Solomon Snyder) from the Johns Hopkins University School of Medicine. He served as a pediatrics resident, postdoctoral fellow (with Richard Axel), and assistant professor of pediatrics in neurology at the College of Physicians and Surgeons of Columbia University. Dr. Nye has published more than 60 peer-reviewed papers on topics spanning basic and clinical research, and has led pharma programs in discovery through clinical development. He has served on numerous study sections and advisory panels, and currently serves as a councilor of the British Association of Psychopharmacology. He is the co-chair of the New York Academy Alzheimer’s Leadership council. Sally Okun, R.N., M.M.H.S., is a member of the Research, Data and Analytics Team at PatientsLikeMe, Inc. As the head of Health Data Integrity and Patient Safety, she is responsible for the site’s medical ontology and the integrity of patient-reported health data. She also dev- eloped and oversees the PatientsLikeMe Drug Safety and Pharmaco- vigilance Platform. Prior to joining PatientsLikeMe, Ms. Okun practiced as a palliative care specialist. In addition to working with patients and families, she was an independent consultant supporting multiyear cli- nical, research, and education projects focused on palliative and end-of- life care for numerous clients, including Brown University, Harvard Medical School, the Massachusetts Department of Mental Health, and the Robert Wood Johnson Foundation. Ms. Okun is a member of the Institute of Medicine Clinical Effectiveness Research Innovation Colla- borative, the Evidence Communication Innovation Collaborative, and the Best Practices Innovation Collaborative. She is a contributing author on two Institute of Medicine discussion papers: “Principles and Values for Team-Based Health Care” and “Communicating Evidence in Health Care: Engaging Patients for Improved Health Care Decisions.” Ms. Okun received her master’s degree from The Heller School for Social Policy & Management at Brandeis University; completed study of Palliative Care and Ethics at Memorial Sloan-Kettering Cancer Center; and was a Na- ional Library of Medicine Fellow in biomedical informatics. 

126 SHARING CLINICAL RESEARCH DATA Eric D. Perakslis, Ph.D., is chief information officer and chief scientist (informatics) at the Food and Drug Administration (FDA). In this role, Dr. Perakslis is responsible for modernizing and enhancing the IT capa- bilities as well as the in silico scientific capabilities at FDA. Prior to FDA, he was senior vice president of R&D information technology at Johnson & Johnson (J&J) Pharmaceuticals R&D and a member of the Corporate Office of Science and Technology. During his 13 years at J&J, Dr. Perakslis also held the posts of vice president R&D informatics, vice president and chief information officer, director of research information technology as well as assistant director and director of drug discovery research prior to his current role. Before joining J&J, he was the group leader of Scientific Computing at ArQule Inc. and he began his profes- sional career with the Army Corps of Engineers. Dr. Perakslis has a Ph.D. in chemical and biochemical engineering from Drexel University and also holds B.S. and M.S. degrees in chemical engineering. His cur- rent research interests are enterprise knowledge management, patient stratification, health care IT and translational informatics with the specif- ic focus on precompetitive data sharing, and open-source systems global- ization. Dr. Perakslis is a late-stage kidney cancer survivor and an avid patient advocate. He has served as the chair of the Survivor Advisory Board at the Cancer Institute of New Jersey and as the chief information officer of the King Hussein Institute for Biotechnology and Cancer in Jordan. He has also worked extensively with Lance Armstrong’s LiveStrong Foundation, the Kidney Cancer Association, the Scientist ↔ Survivor Program of the American Association for Cancer Research, OneMind4Research and several other prominent disease-based organiza- tions to further their agendas. Richard Platt, M.D., M.Sc., is a professor and chair of the Department of Population Medicine, and executive director of the Harvard Pilgrim Health Care Institute. He is an internist trained in infectious diseases and epidemiology. His research focuses on developing multi-institution au- tomated record linkage systems for use in pharmacoepidemiology, and for population-based surveillance, reporting, and control of both hospital- and community-acquired infections. He is principal investigator of the Food and Drug Administration’s (FDA’s) Mini-Sentinel program, of a Centers for Disease Control and Prevention (CDC) Center of Excellence in Public Health Informatics, of the Agency for Healthcare Research and Quality HMO Research Network DEcIDE center, and of a CDC Preven- tion Epicenter. He is a member of the Institute of Medicine Roundtable 

APPENDIX C 127 on Value & Science-Driven Health Care, and of the Association of American Medical Colleges’ Advisory Panel on Research. Dr. Platt for- merly chaired the FDA Drug Safety and Risk Management Advisory Committee, a National Institutes of Health study section (Epidemiology and Disease Control 2), and the CDC Office of Health Care Partnerships steering committee, and co-chaired the Board of Scientific Counselors of the CDC National Center for Infectious Diseases. William Z. Potter, M.D., Ph.D., earned his B.A., M.S., M.D., and Ph.D. at Indiana University, after which he held positions of increasing respon- sibility and seniority in translational neuroscience over the next 25 years at the National Institutes of Health (NIH). While there, Dr. Potter was widely published and appointed to many societies, committees, and boards, which enabled him to develop a wide reputation as an expert in psychopharmacological sciences and champion the development of novel treatments for central nervous system (CNS) disorders. Dr. Potter left the NIH in 1996 to accept a position as executive director and Research Fel- low at Lilly Research Labs, specializing in the Neuroscience Therapeutic Area. In 2004 he joined Merck Research Labs (MRL) as vice president of clinical neuroscience, and in 2006, he took on the newly created posi- tion of vice president of Translational Neuroscience, from which he re- tired in 2012. His experience at Lilly and MRL in identifying, expanding, and developing methods of evaluating CNS effects of compounds in hu- man brains cover state-of-the-art approaches across multiple modalities. These include brain imaging and cerebrospinal fluid proteomics (plus metabolomics) as well as development of more sensitive clinical, psy- chophysiological, and performance measures allowing a range of novel targets to be tested in a manner that actually addresses the underlying hypotheses. He has become a widely recognized champion for the posi- tion that more disciplined hypothesis testing of targets in humans is the best near-term approach to moving CNS drug development forward for important neurologic and psychiatric illnesses. Jonathan Rabinowitz, Ph.D., is the Elie Wiesel Professor and a former department chair at Bar Ilan University. He also holds a visiting profes- sorship in the Department of Psychiatry at Mount Sinai School of Medi- cine in New York. His current research focuses on understanding and minimizing placebo response in antipsychotic and antidepressant trials and developing statistical and methodological ways to improve efficien- cy of the central nervous system drug trials. His research has been sup- 

128 SHARING CLINICAL RESEARCH DATA ported by the National Institutes of Health, Federal Ministry of Educa- tion and Research (BMBF), Innovative Medicines Initiative, European Commission of the European Union, Brain & Behavior Research Foun- dation, formerly NARSAD, and major pharmaceutical companies. He is a member of the editorial board of European Neuropsychopharmacology and a member of the International Biometric Society, International So- ciety for Schizophrenia Research, and European College of Neuro- psychopharmacology. He serves on the Food and Drug Administration Clinical Expert Review Committee data standards development initia- tive, which will standardize clinical data elements for regulatory submis- sion. He is a member of the Drug Information Association scientific working group on missing data in clinical trials. He has published more than 150 scientific papers. Frank W. Rockhold, Ph.D., is senior vice president, global clinical safety and pharmacovigilance at GlaxoSmithKline (GSK) Pharmaceuti- cals Research and Development. This includes case management, signal detection, safety evaluation, and risk management for all development and marketed products. He is also co-chair of the GSK Global Safety Board. In his 20 years at GSK, he has also held management positions within the Statistics & Epidemiology Department and Clinical Opera- tions, both in R&D and in the U.S. pharmaceutical business, and most recently as interim head of the Cardiovascular and Metabolic Medicines Development Center. Dr. Rockhold previously held positions of research statistician, Lilly Research Laboratories, and executive director of biosta- tistics, data management, and health economics, Merck Research La- boratories. He has a B.A. in statistics from the University of Connecticut, an Sc.M. in biostatistics from Johns Hopkins University, and a Ph.D. in biostatistics from the Medical College of Virginia. He has held academic appointments at Butler University; Indiana University; adjunct professor of health evaluation sciences, Penn State University; and adjunct scholar in the Department of Epidemiology and Biostatistics at the University of Pennsylvania. Dr. Rockhold is currently affiliate professor of biostatistics at the Virginia Commonwealth University Medical Center. He is a past chair of the board of directors of the Clinical Data Interchange Standards Consortium and a member of the National Library of Medicine Advisory Group for ClinicalTrials.Gov. He is past president, Society for Clinical Trials; past chair, Pharmaceutical Research and Manufacturers Associa- tion (PhRMA) Biostatistics Steering Committee, and a member of the ICH E-9 and E-10 Expert Working Groups. He also served as associate 

APPENDIX C 129 editor for Controlled Clinical Trials. He is a fellow of the American Sta- tistical Association and a fellow of the Society for Clinical Trials. He is also a recipient of the PhRMA Career Achievement award. He has more than 150 publications and abstracts. Laura Lyman Rodriguez, Ph.D., is director of the Office of Policy, Communications, and Education at the National Institutes of Health (NIH) National Human Genome Research Institute (NHGRI). She works to develop and implement policy for research initiatives at the NHGRI, design communication and outreach strategies to engage the public in genomic science, and prepare health care professionals for the integration of genomic medicine into clinical care. Dr. Rodriguez is particularly interested in the policy and ethics questions related to the inclusion of human research participants in genomics and genetics research and shar- ing human genomic data through broadly used research resources (e.g., databases). Among other activities, Dr. Rodriguez has provided leader- ship for many policy development activities pertaining to genomic data sharing and the creation of the database for Genotypes and Phenotypes (dbGaP) at the NIH. Dr. Rodriguez received her B.S. with honors in biology from Washington and Lee University and earned a doctorate in cell biology from Baylor College of Medicine. Michael Rosenblatt, M.D., is executive vice president and chief medical officer of Merck & Co., Inc. He is the first person to serve in this role for Merck. Previously he served as dean of Tufts University School of Med- icine. Prior to that, he held the appointment of George R. Minot Profes- sor of Medicine at Harvard Medical School and chief of the Division of Bone and Mineral Metabolism Research at Beth Israel Deaconess Medi- cal Center (BIDMC). He served as the president of BIDMC from 1999 to 2001. Previously, he was the Harvard faculty dean and senior vice presi- dent for academic programs at CareGroup and BIDMC, and a founder of the Carl J. Shapiro Institute for Education and Research at Harvard Med- ical School and BIDMC, a joint venture whose mission is to manage the academic enterprise and promote academic innovation. Earlier, he served as director of the Harvard–Massachusetts Institute of Technology (MIT) Division of Health Sciences and Technology, during which time he led a medical education organization for M.D., Ph.D., and M.D.–Ph.D. train- ing jointly sponsored by Harvard and MIT. Previously, he was senior vice president for research at Merck Sharp & Dohme Research Laborato- ries, where he co-led the worldwide development team for alendronate 

130 SHARING CLINICAL RESEARCH DATA (FOSAMAX), Merck’s bisphosphonate for osteoporosis and bone disor- ders. In addition, he directed drug discovery efforts in molecular biology, bone biology and calcium metabolism, virology, cancer research, lipid metabolism, and cardiovascular research in the United States, Japan, and Italy. In leading most of Merck’s international research efforts, he estab- lished two major basic research institutes, one in Tsukuba, Japan, and one near Rome, Italy. He also headed Merck Research’s worldwide Uni- versity and Industry Relations Department. He is the recipient of the Fuller Albright Award for his work on parathyroid hormone and the Vincent du Vigneaud Award in peptide chemistry and biology, and the Chairman’s Award from Merck. His research is in the field of hormonal regulation of calcium metabolism, osteoporosis, and cancer metastasis to bone. His major research projects are in the design of peptide hormone antagonists for parathyroid hormone and the tumor-secreted parathyroid hormone-like protein; isolation/characterization of receptors and map- ping hormone–receptor interactions; elucidation of the mechanisms by which breast cancer “homes” to bone; and osteoporosis and bone biolo- gy. He has been an active participant in the biotechnology industry, serv- ing on the board of directors and scientific advisory boards of several biotech companies. He was a scientific founder of ProScript, the compa- ny that discovered bortezomib (Velcade), now Millennium Pharmaceuti- cal’s drug for multiple myeloma and other cancers. He was a member of the Board of Scientific Counselors of the National Institute of Diabetes and Digestive and Kidney Diseases at the National Institutes of Health. He has been elected to the American Society of Clinical Investigation and the Association of American Physicians; to fellowship in the Ameri- can Association for the Advancement of Science and the American Col- lege of Physicians; and as president of the American Society of Bone and Mineral Research. He has testified before the U.S. Senate on U.S. bio- medical research priorities, and served as a consultant to the U.S. Presi- dent’s Council of Advisors on Science and Technology. He previously served as Chief of the Endocrine Unit, Massachusetts General Hospital. He received his undergraduate degree summa cum laude from Columbia University and his M.D. magna cum laude from Harvard. His internship, residency, and endocrinology training were all at Massachusetts General Hospital. Vicki L. Seyfert-Margolis, Ph.D., is senior advisor for science innova- tion and policy in the Food and Drug Administration Commissioner’s Office. She focuses on initiatives in innovation, regulatory science, per- 

APPENDIX C 131 sonalized medicine, scientific computing, and health informatics. Previ- ously, she served as chief scientific officer at Immune Tolerance Net- work (ITN), a nonprofit consortium of researchers seeking new treatments for diseases of the immune system. At ITN, she oversaw the development of more than 20 centralized laboratory facilities, and the design and execution of biomarker discovery studies for more than 25 Phase II clinical trials. As part of the biomarker efforts, she established construction of a primer library of 1,000 genes that may be involved in establishing and maintaining immunologic tolerance and codiscovered genes that may mark kidney transplant tolerance. Dr. Seyfert-Margolis was also an adjunct associate professor with the Department of Medicine at the University of California, San Francisco. She also served as director of the Office of Innovative Scientific Research Technologies at the Na- tional Institute of Allergy and Infectious Diseases at the National Insti- tutes of Health, where she worked to integrate emerging technologies into existing immunology and infectious disease programs. Dr. Seyfert- Margolis completed her Ph.D. in immunology at the University of Penn- sylvania’s School of Medicine. Jay M. Tenenbaum, Ph.D., is the founder and chair of Cancer Com- mons, a nonprofit, open science community that compiles and continual- ly refines information about cancer subtypes and treatments, based on the literature and actual patient outcomes. Dr. Tenenbaum’s background brings a unique perspective of a world-renowned Internet commerce pio- neer and visionary. He was founder and CEO of Enterprise Integration Technologies, the first company to conduct a commercial Internet trans- action, secure Web transaction, and Internet auction. In 1994, he founded CommerceNet to accelerate business use of the Internet. In 1997, he co- founded Veo Systems, the company that pioneered the use of XML for automating business-to-business transactions. Dr. Tenenbaum joined Commerce One in 1999, when it acquired Veo Systems. As chief scien- tist, he was instrumental in shaping the company’s business and technol- ogy strategies for the Global Trading Web. After Commerce One, Dr. Tenenbaum was an officer and a director of Webify Solutions, which was sold to IBM in 2006, and Medstory, which was sold to Microsoft in 2007. Dr. Tenenbaum was also the Founder and Chair of CollabRx, a provider of Web-based applications and services that help cancer patients and their physicians select optimal treatments and trials; it was acquired by Tegal in 2012. Earlier in his career, Dr. Tenenbaum was a prominent artificial intelligence (AI) researcher and led AI research groups at SRI 

132 SHARING CLINICAL RESEARCH DATA International and Schlumberger Ltd. Dr. Tenenbaum is a Fellow and former board member of the American Association for Artificial Intelli- gence, and a former consulting professor of computer science at Stan- ford. He currently serves as a director of Efficient Finance, PatientsLikeMe, and the Public Library of Science, and is a consulting professor of information technology at Carnegie Mellon’s new West Coast campus. Dr. Tenenbaum holds a B.S. and an M.S. in electrical en- gineering from the Massachusetts Institute of Technology, and a Ph.D. from Stanford University. Andrew Vickers, Ph.D., is attending research methodologist in the De- partment of Epidemiology and Biostatistics at Memorial Sloan-Kettering Cancer Center (MSKCC). He obtained a first in natural science from the University of Cambridge and has a doctorate in clinical medicine from the University of Oxford. Dr. Vickers’ clinical research falls into three broad areas: randomized trials, surgical outcomes research, and molecu- lar marker studies. A particular focus of his work is the detection and initial treatment of prostate cancer. Dr. Vickers has analyzed the “learn- ing curve” for radical prostatectomy, is working on a series of studies demonstrating that a single measure of prostate-specific antigen taken in middle age can predict prostate cancer up to 25 years later, and has de- veloped a statistical model to predict the result. His work on randomized trials focuses on methods for integrating randomized trials into routine surgical practice so as to compare different approaches to surgery. As part of this work, he has pioneered the use of Web interfaces for obtain- ing quality-of-life data from patients recovering from radical prostatec- tomy. Dr. Vickers’ methodological research centers primarily on novel methods for assessing the clinical value of predictive tools. In particular, he has developed decision-analytic tools that can be directly applied to a dataset, without the need for data gathering on patient preferences or util- ities. Dr. Vickers has a strong interest in teaching statistics. He is course leader for the MSKCC biostatistics course, teaches on the undergraduate curriculum at Weill Medical College of Cornell University, and writes the statistics column for Medscape. He is author of the introductory sta- tistics textbook What Is a P-Value Anyway? 34 Stories to Help You Ac- tually Understand Statistics. John A. Wagner, M.D., Ph.D., received his M.D. from Stanford Univer- sity School of Medicine and his Ph.D. from the Johns Hopkins University School of Medicine. Postgraduate training included an internship and resi- 

APPENDIX C 133 dency in internal medicine as well as molecular and clinical pharmacolo- gy postdoctoral fellowships. Currently, Dr. Wagner is vice president and head, Early Development Pipeline and Projects, and head, Global Project Management, at Merck & Co., Inc. Previously, he was vice president and head, clinical pharmacology, at Merck. Dr. Wagner is also an adjunct assistant professor, Division of Clinical Pharmacology, Department of Medicine, Jefferson Medical College, and visiting clinical scientist, Harvard–Massachusetts Institute of Technology Division of Health Sci- ences and Technology, Center for Experimental Pharmacology and Therapeutics. He is the past chair of the PhRMA Clinical Pharmacology Technical Group, past chair of the adiponectin workgroup for the Bi- omarkers Consortium, past committee member of the Institute of Medi- cine (IOM) Committee on Qualification of Biomarkers and Surrogate Endpoints in Chronic Disease, current member of the Board of Directors and current chair of the 2012 Scientific Program Committee for the American Society for Clinical Pharmacology and Therapeutics, and cur- rent full member of the IOM National Cancer Policy Forum. His more than 150 peer-reviewed publications include work in the areas of bi- omarkers and surrogate endpoints, experimental medicine, modeling and simulation, pharmacokinetics, pharmacodynamics, precompetitive col- laboration, and drug–drug interactions across a variety of therapeutic areas. John Wilbanks, B.A., is a senior fellow in entrepreneurship with the Ewing Marion Kauffman Foundation. He works on open-content, open- data, and open-innovation systems. Mr. Wilbanks also serves as a re- search fellow at Lybba. He has worked at Harvard Law School, the Mas- sachusetts Institute of Technology Computer Science and Artificial Intelligence Laboratory, the World Wide Web Consortium, the U.S. House of Representatives, and Creative Commons, as well as starting a bioinformatics company. He sits on the board of directors for Sage Bio networks and iCommons, and the advisory board for Boundless Learn- ing. Mr. Wilbanks holds a degree in philosophy from Tulane University and also studied modern letters at the University of Paris (La Sorbonne). Janet Woodcock, M.D., is the director, Center for Drug Evaluation and Research (CDER), Food and Drug Administration (FDA). Dr. Woodcock held various leadership positions within the Office of the Commissioner, FDA, including deputy commissioner and chief medical officer, deputy commissioner for operations, and chief operating officer and director, 

134 SHARING CLINICAL RESEARCH DATA Critical Path Programs. Previously, Dr. Woodcock served as director, CDER, from 1994 to 2005. She also held other positions at FDA, includ- ing director, Office of Therapeutics Research and Review, and acting deputy director, Center for Biologics Evaluation and Research. A promi- nent FDA scientist and executive, Dr. Woodcock has received numerous awards, including a Presidential Rank Meritorious Executive Award, the American Medical Association’s Nathan Davis Award, and Special Cita- tions from FDA Commissioners. Dr. Woodcock received her M.D. from Northwestern Medical School, and completed further training and held teaching appointments at the Pennsylvania State University and the Uni- versity of California, San Francisco. Deborah A. Zarin, M.D., is director of ClinicalTrials.gov and assistant director of clinical research projects at the Lister Hill National Center for Biomedical Communications in the National Library of Medicine. In this capacity, Dr. Zarin oversees the development and operation of an inter- national registry of clinical trials. Previous positions include director of the Agency for Healthcare Research and Quality Technology Assessment Program and director of the American Psychiatric Association Practice Guidelines program. In these positions, Dr. Zarin conducted systematic reviews and related analyses in order to develop clinical and policy rec- ommendations. Her academic interests are in the area of evidence-based clinical and policy decision making, and she is the author of more than 70 peer-reviewed articles. Dr. Zarin graduated from Stanford University and received her M.D. from Harvard Medical School. She completed a clinical decision-making fellowship, and is board certified in general psychiatry as well as in child and adolescent psychiatry.