Sharing Clinical Research Data: an IOM Workshop

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Briefing book for IOM workshop on Sharing Clinical Research Data, Oct. 4-5, 2012

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Sharing Clinical Research Data: an IOM Workshop

  1. 1. Forum on Drug Discovery, Development, and Translation Forum on Neuroscience and Nervous System DisordersRoundtable on Translating Genomic-Based Research for Health National Cancer Policy Forum Present Sharing Clinical Research Data:An IOM Workshop October 4-5, 2012 National Academy of Sciences 2101 Constitution Avenue, N.W. Washington, D.C. 20418
  2. 2. Sharing Clinical Research Data: An Institute of Medicine Workshop TABLE OF CONTENTS October 4 - 5, 2012 Washington, DCITEMS TAB/PAGEAgenda AGENDASharing Clinical Research Data 1 COORDINATING ROUNDTABLE AND FORUMS COORDINATING ROUNDTABLECoordinating Roundtable and Forums AND FORUMS• Forum on Drug Discovery, Development, and Translation 1• Forum on Neuroscience and Nervous System Disorders 3• Roundtable on Translating Genomic-Based Research for Health 5• National Cancer Policy Forum 7 WORKSHOPWorkshop, October 4-5 WORKSHOP• Speaker Biographies 1 BACKGROUND ARTICLES BACKGROUNDBackground Reading for Workshop1 ARTICLESBenefits• Akil, H., M. E. Martone, et al. Challenges and opportunities in 1 mining neuroscience data• Brown, J. S., J. H. Holmes, et al. A practical and preferred approach to 6 multi-institutional evaluations of comparative effectiveness, safety, and quality of care• Krumholz, H. M. Open science and data sharing in clinical research : 13 Basing informed decisions on the totality of the evidenceModels• Califf, R. M., D. A. Zarin, et al. Characteristics of clinical trials registered in ClinicalTrials.gov, 2007-2010 15• Doshi, P., T. Jefferson, et al. The imperative to share clinical study reports: Recommendations from the Tamiflu experience 25• Piwowar, H.A. Who shares? Who doesn’t? Factors associated with openly archiving raw research data 31• Turner, E. H., A. M. Matthews, et. al. Selective publication of 44 antidepressant trials and its influence on apparent efficacy• Wagner, J. A., M. Prince, et al. The Biomarkers Consortium: 53 Practice and pitfalls of open-source precompetitive collaboration• List of Clinical Research Data Sharing Projects 571 Background materials have been gathered throughout the workshop planning committee process to inform the development of the meeting and understanding of key issues. The articles contained in this briefing book are a subset of background materials collected and inclusion of particular articles does not denote IOM endorsement.
  3. 3. ITEMS TAB/PAGEStandardization and Governance• CDISC press release. CDISC, C-Path and FDA collaborate to develop data 75 standards to streamline path to new therapies• CDISC press release. Public release of Alzheimer’s clinical trial data by pharmaceutical researchers 77• Davies, K. Get smart: Knowledge management 80• Davies, K. Running tranSMART for the drug development marathon 82• The Economist editorial. Genomic research: Consent 2.0 85• Ghersi, D., et al. Reporting the findings of clinical trials: a discussion paper 86• Hayden, E. C. Informed consent: A broken contract 88• Kolata, G. Genes now tell doctors secrets they can’t utter 91• Savage, C. J. and A. J. Vickers. Empirical study of data sharing by authors publishing in PLoS Journals 96Culture and Policy• Eichler, H.-G., E. Abadie, et al. Open clinical trial data for all? A view from regulators 99• Laine, C., S. N. Goodman, et al. Reproducible research: Moving toward research the public can really trust 101• Law, M. R., Y. Kawasumi, et al. Despite Law, Fewer Than One In Eight Completed Studies Of Drugs And Biologics Are Reported On Time On ClinicalTrials.gov 106• Mansi, B. A., J. Clark, et al. Ten recommendations for closing the credibility gap in reporting industry-sponsored clinical research: A joint journal and pharmaceutical industry perspective 114• NIH. Final NIH statement on sharing research data• Vickers, A. J. Whose data set is it anyway? Sharing raw data from randomized trials 120• Zarin, D. A. and T. Tse. Moving toward transparency of clinical trials 126 TRAVEL INFORMATION TRAVELTravel Information INFORMATION• Logistics Memo 1• Map of National Academies Building 5
  4. 4. AGENDA
  5. 5. Forum on Drug Discovery, Development, and Translation Forum on Neuroscience and Nervous System Disorders National Cancer Policy Forum Roundtable on Translating Genomic-Based Research for Health Sharing Clinical Research Data: An Institute of Medicine Workshop DRAFT AGENDA October 4 & 5, 2012 National Academy of Sciences Building, Room 125 2101 Constitution Avenue, N.W. Washington, DCBackground: Pharmaceutical companies, academic institutions, advocacy organizations, and governmentagencies such as FDA and NIH have large quantities of clinical research data. Increased datasharing could facilitate scientific and public health advances, among other potential benefits topatients and society. Much of this information, however, is never published or shared. Morespecifically, study results are not always published and where results are published, theytypically only include summary-level data; participant-level data is privately held and rarelyshared or revealed publicly. The workshop will explore the benefits of and barriers to the sharing of clinical research dataand will help identify strategies for enhancing sharing both within sectors and across sectors. Tomake the workshop scope manageable, the workshop will focus on data resulting frompreplanned interventional studies of human subjects. While recognizing the importance of otherdata sources such as observational studies and electronic health records, this focus was selectedto encourage concrete problem-solving discussions over the course of a day-and-a-half meeting.Models and projects that involve sharing other types of data will be considered during theworkshop to the extent that these models provide lessons and best practices applicable to sharingpreplanned interventional clinical research data. The workshop is being jointly organized by the Institute of Medicine’s Forum on DrugDiscovery, Development, and Translation; Forum on Neuroscience and Nervous SystemDisorders; National Cancer Policy Forum; and Roundtable on Translating Genomic-BasedResearch for Health. Participants will be invited from industry, academia, government agenciessuch as FDA and NIH, disease advocacy groups, and other stakeholder groups.Meeting Objectives: Examine the benefits of sharing of clinical research data, and specifically clinical trial data, from all sectors and among these sectors, including, for example: o Benefits to the research and development enterprise o Benefits to the analysis of safety and efficacy Identify barriers and challenges to sharing clinical research data Explore strategies to address these barriers and challenges, including identifying priority actions and “low-hanging fruit” opportunities Discuss strategies for using these potentially large data sets to facilitate scientific and public health advances. 1
  6. 6. Forum on Drug Discovery, Development, and Translation Forum on Neuroscience and Nervous System Disorders National Cancer Policy Forum Roundtable on Translating Genomic-Based Research for Health Day One8:30 a.m. Opening Remarks SHARON TERRY, Workshop Chair President and Chief Executive Officer Genetic Alliance SESSION I: BENEFITS OF SHARING CLINICAL RESEARCH DATASession Objectives: Provide an overview of the benefits of sharing clinical research data, specifically clinical trial data, and discuss advantages and disadvantages of sharing participant vs. summary level data from individual trials as well as pooling data across multiple studies. Outline examples of scientific success stories that illustrate what can be accomplished when clinical trial data is shared.8:40 a.m. Background and Session Objectives WILLIAM POTTER, Session Co-Chair Co-Chair Emeritus Neuroscience Steering Committee FNIH Biomarkers Consortium DEBORAH ZARIN, Session Co-Chair Director, ClinicalTrials.gov National Library of Medicine National Institutes of Health8:50 a.m. Fundamentals and Benefits of Sharing Participant-Level Clinical Trial Data ELIZABETH LODER Clinical Epidemiology Editor British Medical Journal9:10 a.m. Pooling Data from Multiple Clinical Trials to Answer Big Questions ROBERT CALIFF Director, Duke Translational Medicine Institute Professor of Medicine Vice Chancellor for Clinical and Translational Research Duke University Medical Center 2
  7. 7. Forum on Drug Discovery, Development, and Translation Forum on Neuroscience and Nervous System Disorders National Cancer Policy Forum Roundtable on Translating Genomic-Based Research for Health9:30 a.m. Panel Discussion: Perspectives on the Benefits of Sharing Clinical Trial Data  Data sharing – what does it mean from your perspective?  Considering the benefits and risks of sharing clinical research data, how extensively should it be shared to maximize new knowledge and ultimately patient benefit? Panelists: HARLAN KRUMHOLZ Harold H. Hines, Jr. Professor of Medicine and Epidemiology and Public Health Yale University School of Medicine MYLES AXTON Editor Nature Genetics JESSE BERLIN Vice President of Epidemiology Janssen Research and Development10:30 a.m. BREAK SESSION II: DATA SHARING MODELS: DESIGN, BEST PRACTICES, AND LESSONS LEARNEDSession Objectives: Present examples, best practices, and lessons learned from projects across the continuum of data sharing opportunities (e.g., rapid publication of participant-level data, increased access to participant-level data for qualified researchers, or maximizing the use of clinical research data that is currently held in centralized locations by requiring sharing or access to subsets of data). Distill best practices and lessons learned that can be applied broadly to new projects to maximize the use of data from individual trials and/or data pooling initiatives.10:45 a.m. Background and Session Objectives JEFFREY NYE, Session Chair Vice President and Head Neuroscience External Innovation Johnson and Johnson Pharmaceutical R&D, LLC10:55 a.m. The Limits of Summary Data Reporting: Lessons from ClinicalTrials.gov 3
  8. 8. Forum on Drug Discovery, Development, and Translation Forum on Neuroscience and Nervous System Disorders National Cancer Policy Forum Roundtable on Translating Genomic-Based Research for Health DEBORAH ZARIN Director, ClinicalTrials.gov National Institutes of Health11:10 a.m. Models that Increase Access and Use of Data from Individual Clinical Trials The DataSphere Project CHARLES HUGH-JONES Vice President, Medical Affairs North America Sanofi Oncology Yale/Medtronic Experience RICHARD KUNTZ Senior Vice President Chief Scientific, Clinical and Regulatory Officer Medtronic, Inc.11:40 a.m. Models that Foster Pooling and Analysis of Data FNIH Biomarkers Consortium Adiponectin Project JOHN WAGNER Vice President, Clinical Pharmacology Merck & Co., Inc. Novel Methods Leading to New Medications in Depression and Schizophrenia (NEWMEDS) Consortium JONATHAN RABINOWITZ Academic Lead, NEWMEDS Bar Ilan University12:10 p.m. Series of Brief Presentations on Overcoming Challenges Facing Clinical Trial Data Sharing Challenge #1: Permissions JENNIFER GEETTER Partner McDermott Will & Emery Challenge #2: Techniques and Methodologies 4
  9. 9. Forum on Drug Discovery, Development, and Translation Forum on Neuroscience and Nervous System Disorders National Cancer Policy Forum Roundtable on Translating Genomic-Based Research for Health JOHN IOANNIDIS [via video conference] C.F. Rehnborg Chair in Disease Prevention Stanford University Challenge #3: Culture KELLY EDWARDS Acting Associate Dean, The Graduate School Associate Professor, Bioethics and Humanities University of Washington12:40 p.m. Discussion among speakers, panelists, and audience Discussant: Sally Okun, Health Data Integrity & Patient Safety, PatientsLikeMe1:00 p.m. LUNCH KEYNOTE CASE STUDY: DISTRIBUTED SYSTEMS FOR CLINICAL RESEARCH INFORMATION SHARING1:30 p.m. RICHARD PLATT Professor and Chair Department of Population Medicine Harvard Pilgrim Health Care Institute and Harvard Medical School1:50 p.m. Discussion with speaker and audience Moderator: Sharon Terry, Genetic Alliance SESSION III: STANDARDIZATION AND GOVERNANCESession Objectives: Receive an update on recent legislative and regulatory language regarding standardization of clinical research data and discuss how stakeholders are designing and implementing data standardization plans in response. Discuss the relative cost-benefit of data conversion of existing trial data versus building an infrastructure to improve data collection and sharing moving forward. Present case studies from data sharing projects using different data standardization and governance models and consider lessons learned or best practices for the future.2:00 p.m. Background and Session Objectives 5
  10. 10. Forum on Drug Discovery, Development, and Translation Forum on Neuroscience and Nervous System Disorders National Cancer Policy Forum Roundtable on Translating Genomic-Based Research for Health FRANK ROCKHOLD, Session Co-Chair Senior Vice President, Global Clinical Safety and Pharmacovigilance GlaxoSmithKline Pharmaceuticals Research and Development LYNN HUDSON, Session Co-Chair Chief Science Officer and Executive Director Coalition Against Major Diseases Critical Path Institute2:10 p.m. PDUFA Update on Data Standards MARY ANN SLACK Office of Planning and Informatics Center for Drug Evaluation and Research U.S. Food and Drug Administration2:25 p.m. Standardization to Facilitate Data Sharing: Opportunities and Limitations CDISC Efforts to Support Clinical Research Data REBECCA KUSH President and Chief Executive Officer Clinical Data Interchange Standards Consortium HL7 Efforts to Support Clinical Care Data CHARLES JAFFE Chief Executive Officer Health Level 7 International Health Information Technology Perspective on Clinical Research Data Standards SACHIN JAIN Chief Medical Information and Innovation Officer Merck & Co., Inc.3:10 p.m. Discussion with speakers and audience3:30 p.m. BREAK3:45 p.m. Cost-Benefit Analysis of Retrospective vs. Prospective Data Standardization 6
  11. 11. Forum on Drug Discovery, Development, and Translation Forum on Neuroscience and Nervous System Disorders National Cancer Policy Forum Roundtable on Translating Genomic-Based Research for Health VICKI SEYFERT-MARGOLIS Senior Advisor, Science Innovation and Policy Office of the Chief Scientist U.S. Food and Drug Administration4:00 p.m. Case Studies: Standardization and Governance Models in Data Sharing Critical Path Institute and Coalition Against Major Diseases (CAMD) Alzheimer’s Clinical Trial Database CAROLYN COMPTON President and CEO Critical Path Institute Translational Medicine Mart (tranSMART) ERIC PERAKSLIS Chief Information Officer and Chief Scientist, Informatics U.S. Food and Drug Administration4:30 p.m. Panel Discussion Catalog new data sharing challenges not yet discussed and provide suggestions for overcoming these challenges. Given the data standardization and governance models discussed, suggest a framework to guide the development of new data sharing projects based on their purpose (e.g., regulatory approval with FDA, detecting safety signals, testing secondary hypotheses, etc.) Panelists: LAURA LYMAN RODRIGUEZ Director Office of Policy, Communications and Education National Human Genome Research Institute MEREDITH NAHM Associate Director for Clinical Research Informatics Duke Translational Medicine Institute NEIL DE CRESCENZO Senior Vice President and General Manager Oracle Health Sciences 7
  12. 12. Forum on Drug Discovery, Development, and Translation Forum on Neuroscience and Nervous System Disorders National Cancer Policy Forum Roundtable on Translating Genomic-Based Research for Health MICHAEL CANTOR Senior Director Clinical Informatics and Innovation Pfizer, Inc.5:30 p.m. Adjourn day 1 8
  13. 13. Forum on Drug Discovery, Development, and Translation Forum on Neuroscience and Nervous System Disorders National Cancer Policy Forum Roundtable on Translating Genomic-Based Research for Health Day Two8:00 a.m. Opening Remarks SHARON TERRY, Workshop Chair President and Chief Executive Officer Genetic Alliance SESSION IV: INCENTIVIZING POLICY AND CULTURAL SHIFTS TO ENHANCE DATA SHARINGSession Objectives: Receive an update on clinical trial data transparency decisions in Europe. Explore current incentives for and against (i.e., benefits and risks of) data sharing within and across sectors and suggest mechanisms to encourage stakeholders to engage in a culture of data sharing. Identify existing and potential strategies, including technology-based approaches, for protecting patient privacy and confidentiality while facilitating data sharing.8:10 a.m. Background and Session Objectives ROBERT HARRINGTON, Session Chair Arthur L. Bloomfield Professor of Medicine Chair, Department of Medicine Stanford University8:20 a.m. Clinical Trial Data Transparency: European Medicines Agency Perspective HANS-GEORG EICHLER Senior Medical Officer European Medicines Agency8:40 a.m. Clinical Research Data Sharing Practices and Attitudes ANDREW VICKERS Attending Research Methodologist Department of Epidemiology and Biostatistics Memorial Sloan-Kettering Cancer Center 9
  14. 14. Forum on Drug Discovery, Development, and Translation Forum on Neuroscience and Nervous System Disorders National Cancer Policy Forum Roundtable on Translating Genomic-Based Research for Health8:55 a.m. Overview of Data Sharing Policies: Research Funders and Publishers STEVEN GOODMAN Associate Dean for Clinical and Translational Research Professor of Medicine & Health Research and Policy Stanford University School of Medicine9:10 a.m. Series of Presentations: Incentives for Data Sharing Within and Across Sectors Academic Perspectives PETER DOSHI Post-Doctoral Fellow Johns Hopkins University School of Medicine BETH KOZEL Instructor of Pediatrics Division of Genetics and Genomic Medicine St. Louis Children’s Hospital and Washington University Pharmaceutical Company Perspective [Speaker TBA] Federal Research Funder Perspective JOSEPHINE BRIGGS Director, National Center for Complementary and Alternative Medicine Director, National Center for Advancing Translation Sciences, Division of Clinical Innovation National Institutes of Health10:10 a.m. Discussion with speakers and audience10:30 a.m. BREAK10:45 a.m. Facilitating Patient Ownership of Clinical Trial Data: Technical Challenges and Opportunities JOHN WILBANKS Director Sage Bionetworks 10
  15. 15. Forum on Drug Discovery, Development, and Translation Forum on Neuroscience and Nervous System Disorders National Cancer Policy Forum Roundtable on Translating Genomic-Based Research for Health DEVEN MCGRAW Director, Health Privacy Project Center for Democracy and Technology11:15 a.m. Discussion with speakers and audience SESSION V: NEXT STEPS AND FUTURE DIRECTIONSSession Objectives: Discuss key themes from the workshop. Based on workshop presentations and discussions, identify potential next steps and priority actions for data sharing stakeholders to take action. Highlight potential opportunities and challenges that are currently on the horizon but may become more salient as technology evolves and/or data sharing becomes more pervasive.11:30 a.m. Background and Session Objectives SHARON TERRY, Workshop Chair President and Chief Executive Officer Genetic Alliance11:40 a.m. Session Chair Reports [5 minutes per Session] WILLIAM POTTER, Session I Co-Chair Co-Chair Emeritus Neuroscience Steering Committee FNIH Biomarkers Consortium DEBORAH ZARIN, Session I Co-Chair Director, ClinicalTrials.gov National Library of Medicine National Institutes of Health JEFFREY NYE, Session II Chair Vice President and Head Neuroscience External Innovation Johnson and Johnson Pharmaceutical R&D, LLC FRANK ROCKHOLD, Session III Co-Chair Senior Vice President Global Clinical Safety and Pharmacovigilance GlaxoSmithKline Pharmaceuticals Research and Development 11
  16. 16. Forum on Drug Discovery, Development, and Translation Forum on Neuroscience and Nervous System Disorders National Cancer Policy Forum Roundtable on Translating Genomic-Based Research for Health LYNN HUDSON, Session III Co-Chair Chief Science Officer and Executive Director Coalition Against Major Diseases Critical Path Institute ROBERT HARRINGTON, Session IV Chair Arthur L. Bloomfield Professor of Medicine Chair, Department of Medicine Stanford University12:00 p.m. Closing Discussion with Session Chairs, Panelists, and Audience Led by Workshop Chair JOSEPHINE BRIGGS Director, National Center for Complementary and Alternative Medicine Director, National Center for Advancing Translation Sciences, Division of Clinical Innovation National Institutes of Health MICHAEL ROSENBLATT Executive Vice President and Chief Medical Officer Merck & Co., Inc. JAY “MARTY” TENENBAUM Founder and Chairman Cancer Commons JANET WOODCOCK Director, Center for Drug Evaluation and Research U.S. Food and Drug Administration12:45 p.m. Adjourn 12
  17. 17. Forum on Drug Discovery, Development, and Translation Forum on Neuroscience and Nervous System Disorders National Cancer Policy Forum Roundtable on Translating Genomic-Based Research for Health SHARING CLINICAL RESEARCH DATA: A WORKSHOP October 4-5, 2012 – Washington, D.C. Background Information Statement of TaskPharmaceutical companies, academics, and govern- The Institute of Medicine will conduct a public work-ment agencies such as the Food and Drug Administra- shop that will focus on strategies to facilitate sharingtion and the National Institutes of Health have large of clinical research data. Participants will be invitedquantities of clinical research data. Data sharing within from industry, academia, government agencies such aseach sector and across sectors could facilitate scientific FDA and NIH, disease advocacy groups, and otherand public health advances and could enhance analysis stakeholder groups. The workshop will feature invitedof safety and efficacy. Much of this information, how- presentations and discussions that will:ever, is never published. This workshop will explore Examine the benefits of sharing of clinical re-barriers to sharing of clinical research data, specifical- search data from all sectors and among these sec-ly clinical trial data, and strategies for enhancing shar- tors, including, for example:ing within sectors and among sectors to facilitate o Benefits to the research and development en-research and development of effective, safe, and need- terpriseed products. o Benefits to the analysis of safety and efficacyA number of efforts are currently underway to enhance Identify barriers and challenges to sharing clinicalpublic/private partnerships and foster collaboration research datawithin the pharmaceutical sector to advance research Explore strategies to address these barriers andand enhance the discovery and development of drugs, challenges, including identifying priority actionsdevices, and diagnostic tools. Examples include the and “low-hanging fruit” opportunitiesAnalgesic Clinical Trials Innovation, Opportunities, Discuss strategies for using these potentially largeand Networks (ACTION) Initiative; the Foundation data sets to facilitate scientific and public healthfor the National Institute of Health’s Biomarkers Con- advances.sortium; the Innovative Medicines Initiative (Europe);Critical Path Institute; Sage Bionetworks’ Clinical Tri- Planning Committeeal Comparator Arm Partnership; and the Life Sciences Sharon Terry, chair, Genetic AllianceConsortium’s MetaPharm project. Planned within the Josephine P. Briggs, National Institutes of Healthcontext of these various efforts, this workshop will Timothy Coetzee, National Multiple Sclerosis Societyfocus specifically on data sharing, which is one com- Steven Goodman, Stanford Universityponent of larger efforts to build partnerships and en- Robert A. Harrington, Stanford Universityhance collaboration within and among sectors on Lynn Hudson, Critical Path Instituteresearch, development, and assessment of pharmaceu- Charles Hugh-Jones, Sanofi Oncologytical products. Jan Johannessen, Food and Drug AdministrationThis project will be a coordinated effort of the IOMs Jeffrey S. Nye, Johnson and JohnsonForum on Drug Discovery, Development and Transla- Richard Platt, Harvard Medical Schooltion; Forum on Neuroscience and Nervous System William Z. Potter, FNIH Biomarkers ConsortiumDisorders; National Cancer Policy Forum; and Frank W. Rockhold, GlaxoSmithKlineRoundtable on Translating Genomic-Based Research Michael Rosenblatt, Merckfor Health. All four Forums/Roundtables have done Vicki Seyfert-Margolis, Food and Drug Administrationprevious work on this topic and this issue cuts across Deborah A. Zarin, National Institutes of Healththe focus of each activity.The IOM was chartered in 1970 as a component of the National Academy of Sciences to enlist distinguished members of the appropriateprofessions in the examination of policy matters pertaining to the health of the public. In this, the Institute acts under both the Academy’s1863 congressional charter responsibility to be an adviser to the federal government and its own initiative in identifying issues of medicalcare, research, and education. For additional information about this workshop, contact Rebecca English at renglish@nas.edu.
  18. 18. COORDINATINGROUNDTABLES and FORUMS
  19. 19. Board on Health Sciences Policy Forum on Drug Discovery, Development, and Translation FORUM ON DRUG DISCOVERY, DEVELOPMENT, AND TRANSLATION The Institute of Medicine’s Forum on Drug Transforming Research and Fostering CollaborativeDiscovery, Development, and Translation was created in Research2005 by the IOM’s Board on Health Sciences Policy toprovide a unique platform for dialogue and collaboration The Forum has established an initiative to examineamong thought leaders and stakeholders in government, the state of clinical trials in the U.S., identify areas ofacademia, industry, foundations and patient advocacy. strength and weakness in our current clinical trialThe Forum brings together leaders from private sector enterprise, and consider transformative strategies forsponsors of biomedical and clinical research, federal enhancing the ways in which clinical research isagencies sponsoring and regulating biomedical and organized and conducted. Workshops and meetings heldclinical research, the academic community, and in 2009 and 2010 considered case studies in four diseaseconsumers, and in doing so serves to educate the policy areas; and included discussions around issues ofcommunity about issues where science and policy management of conflict of interest, and addressingintersect. regulatory and administrative impediments to the conduct The Forum convenes several times each year to of clinical trials. Meetings in 2011 will address how toidentify and discuss key problems and strategies in the move toward greater public engagement in anddiscovery, development, and translation of drugs. To understanding of the clinical trial enterprise, andsupplement the perspectives and expertise of its members, establishing a framework for a transformed nationalthe Drug Forum also holds public workshops to engage a clinical trial enterprise.wide range of experts, members of the public, and the Developing Drugs for Rare and Neglected Diseases andpolicy community in discussing areas of concern in the Addressing Urgent Global Health Problemsscience and policy of drug development. The Forum’spublic meetings focus substantial public attention on The Forum is sponsoring a series of workshops on thecritical areas of drug development, focusing on the major global problem of MDR TB. The Forum held athemes outlined below. foundational workshop in Washington, DC in 2008, for which it commissioned a paper from Partners In Health.The Approach to Drug Development Additional workshops are being held in the four countries with the highest MDR TB burden—South Africa and Despite exciting scientific advances, the pathway Russia (held 2010), and India and China (anticipatedfrom basic science to new therapeutics faces challenges 2011). Also in 2011, the Forum will convene a focusedon many fronts. New paradigms for discovering and initiative to consider the global drug supply chain fordeveloping drugs are being sought to bridge the ever- quality-assured second-line drugs for tuberculosis.widening gap between scientific discoveries andtranslation of those discoveries into life-changing Promoting Public Understanding of Drugmedications. The Forum has explored these issues from Developmentmany perspectives—emerging technology platforms,regulatory efficiency, intellectual property concerns, the Successful introduction of new therapeutic entitiespotential for precompetitive collaboration, and innovative requires testing in an informed and motivated public. Thebusiness models that address the “valley of death.” Forum has spent concerted effort to understand what limits public participation and how to enhance moreStrengthening the Scientific Basis of Drug Regulation widespread acceptance of the importance of advancing therapeutic development through public participation in Over the past several years, the Forum has focused its the drug development process. Forum meetings held in theattention on the scientific basis for the regulation of drugs. spring and fall of 2010 addressed these issues. The ForumIn February 2010, the Forum held a workshop that plans to continue to work with multiple stakeholders toexamined the state of the science of drug regulation and improve public understanding of and participation in theconsidered approaches for enhancing the scientific basis drug development process.of regulatory decision making. 500 Fifth Street, NW Phone: 202 334-2715 Washington, DC 20001 Fax: 202 334-1329 E-mail: aclaiborne@nas.edu www.iom.edu/drug 1
  20. 20. FORUM ON DRUG DISCOVERY, DEVELOPMENT, AND TRANSLATION JEFFREY DRAZEN, (Co-Chair) New England Journal of Medicine STEVEN GALSON (Co-Chair) Amgen Inc.MARGARET ANDERSON PETRA KAUFFMANFasterCures National Institute of Neurological Disorders and StrokeHUGH AUCHINCLOSS JACK KEENENational Institute of Allergy and Infectious Diseases Duke University Medical CenterLESLIE BENET RONALD KRALLUniversity of California, San Francisco University of PennsylvaniaANN BONHAM FREDA LEWIS-HALLAssociation of American Medical Colleges Pfizer Inc.LINDA BRADY MARK MCCLELLANNational Institute of Mental Health Brookings InstitutionROBERT CALIFF CAROL MIMURADuke University Medical Center University of California, BerkeleyTHOMAS CASKEY ELIZABETH (BETSY) MYERSBaylor College of Medicine Doris Duke Charitable FoundationGAIL CASSELL JOHN ORLOFFHarvard Medical School Novartis Pharmaceuticals CorporationPETER CORR AMY PATTERSONCeltic Therapeutics, LLLP NIH Office of the DirectorANDREW DAHLEM MICHAEL ROSENBLATTEli Lilly & Co. Merck & Co., Inc.TAMARA (MARA) DARSOW JANET SHOEMAKERAmerican Diabetes Association American Society for MicrobiologyJIM DOROSHOW ELLEN SIGALNational Cancer Institute Friends of Cancer ResearchGARY FILERMAN ELLIOTT SIGALAtlas Health Foundation Bristol-Myers SquibbGARRET FITZGERALD ELLEN STRAHLMANUniversity of Pennsylvania School of Medicine GlaxoSmithKlineMARK GOLDBERGER NANCY SUNGAbbott Pharmaceuticals Burroughs Wellcome FundHARRY GREENBERG JANET TOBIASStanford University School of Medicine Ikana MediaSTEPHEN GROFT JOANNE WALDSTREICHEROffice of Rare Diseases Research, NIH NCATS Janssen Research & DevelopmentLYNN HUDSON JANET WOODCOCKThe Critical Path Institute FDA Center for Drug Evaluation and ResearchTOM INSEL PROJECT STAFFNational Center for Advancing Translational Sciences Anne Claiborne, J.D., M.P.H., Forum Director Rebecca English, M.P.H., Associate Program OfficerMICHAEL KATZ Rita Guenther, Ph.D., Program OfficerMarch of Dimes Foundation Robin Guyse, Senior Program Assistant Elizabeth Tyson, Research AssociateThe IOM was chartered in 1970 as a component of the National Academy of Sciences to enlist distinguished members of the appropriate professions in theexamination of policy matters pertaining to the health of the public. In this, the Institute acts under both the Academy’s 1863 congressional charterresponsibility to be an adviser to the federal government and its own initiative in identifying issues of medical care, research, and education. 2
  21. 21. Board on Health Sciences Policy Forum on Neuroscience and Nervous System Disorders FORUM ON NEUROSCIENCE AND NERVOUS SYSTEM DISORDERS The Institute of Medicine’s Forum on Neuroscience Genetics of Nervous System Disordersand Nervous System Disorders focuses on buildingpartnerships to further understand the brain and nervous Understanding the roles of genes and their productssystem, disorders in their structure and function, as well that control electrical activity, trophic factors,as effective clinical prevention and treatment strategies. transporters, and receptors is critical to understandingThe forum focuses on the six themes outlined below, the brain and nervous system. The role of genetics inand serves to educate the public, press, and policy disease states needs to be more completely understoodmakers regarding these issues. to develop appropriate treatments and interventions. The Forum brings together leaders from private The forum discusses approaches to bettersector sponsors of biomedical and clinical research, understanding environmental and multigenetic factorsfederal agencies sponsoring and regulating biomedical that influence susceptibility, progression, and severityand clinical research, the academic community, and of disease.consumers. The Forum will sponsor workshops for members Cognition and Behaviorand the public to discuss approaches to resolve keychallenges identified by Forum members. It strives to Higher brain functions that give rise to language,enhance understanding of research and clinical issues cognition, memory, and emotion depend on a complexassociated with the nervous system among the scientific and interdependent set of neuronal pathways.community and the general public, and provide a Understanding higher brain functions and theirmechanism to foster partnerships among stakeholders. impairment is critical to understanding effective treatments for disease.Nervous System Disorders Modeling and Imaging The forum promotes partnerships which will leadto a more complete understanding of the agents that Tools that assist in better understanding of the basicdamage cells and trigger cell death, as well as the and higher order functions of the brain aid in researchmechanisms that carry out the process. For example, and understanding of disorders of the brain. This areaprotein aggregation has an important role in focuses on animal and computer models andneurodegeneration and is emerging as a common neuroimaging approaches.mechanism in several nervous system disorders (e.g.Parkinson’s and Alzheimer’s disease). Ethical and Social IssuesMental Illness and Addiction The Forum addresses ethical issues relating to the stigma associated with nervous system disorders, Improved understanding of the nervous system and mental illness, and addiction in society and theits role in mental illness and addictive behavior is engagement of these patient populations in researchcritical to developing new treatments for these efforts. Clinical research is also examined thatconditions. Especially important is improving the elucidates environmental, genetic, social, and culturalunderstanding of the neuronal mechanisms that give risk and protective factors which can guide therise to mental illnesses, increased risk behavior, and development of prevention and early interventionresilience. The Forum discusses mechanisms that strategies.might take advantage of the evolving nature of scienceand the opportunities it brings to investigating the brainand behavior using cross disciplinary approaches thatexpand the understanding of how complex mentalillnesses and addictions arise from individualcomponents. 500 Fifth Street, NW Phone: 202 334-3984 Washington, DC 20001 Fax: 202 334-1329 E-mail:baltevogt@nas.edu www.iom.edu/neuroforum 3
  22. 22. INSTITUTE OF MEDICINE Forum on Neuroscience and Nervous System Disorders STEVE HYMAN, (Chair) The Broad InstituteSUSAN AMARA STORY LANDISSociety for Neuroscience National Institute on Neurological Disease and StrokeMARC BARLOW ALAN LESHNERGE Healthcare, Inc. American Association for the Advancement of ScienceMARK BEAR HUSSEINI MANJIMassachusetts Institute of Technology Johnson and Johnson Pharmaceutical Research and Development, Inc.KATJA BROSENeuron DAVID MICHELSON Merck Research LaboratoriesDANIEL BURCHCeNeRx Biopharma RICHARD MOHS Lilly Research LaboratoriesC. THOMAS CASKEYBaylor College of Medicine JONATHAN MORENO University of Pennsylvania School of MedicineTIMOTHY COETZEEFast Forward, LLC ALEXANDER OMMAYA U.S. Department of Veterans AffairsEMMELINE EDWARDSNIH Neuroscience Blueprint ATUL PANDE GlaxoSmithKline, IncMARTHA FARAHUniversity of Pennsylvania STEVEN PAUL Weill Cornell Medical CollegeRICHARD FRANKGE Healthcare, Inc. TODD SHERER Michael J. Fox Foundation for Parkinson’s ResearchDANIEL GESCHWINDUniversity of California Los Angeles PAUL SIEVING National Eye InstituteHANK GREELYStanford University JUDY SIUCIAK Foundation for the National Institutes of HealthMYRON GUTMANNNational Science Foundation MARC TESSIER-LAVIGNE The Rockefeller UniversityRICHARD HODESNational Institute on Aging WILLIAM THIES Alzheimer’s AssociationSTEVEN HYMANThe Broad Institute NORA VOLKOW National Institute on Drug AbuseTHOMAS INSELNational Institute on Mental Health KENNETH WARREN National Institute on Alcohol Abuse and AlcoholismPHILLIP IREDALEPfizer Global Research and Development JOHN WILLIAMS Wellcome TrustDANIEL JAVITTNathan S. Kline Institute for Psychiatric Research STEVIN H. ZORN Lundbeck USAFRANCES JENSENUniversity of Pennsylvania, School of Medicine CHARLES ZORUMSKI Washington University School of Medicine PROJECT STAFF Bruce Altevogt, Ph.D., Forum Director Diana Pankevich, Ph.D., Associate Program Officer Elizabeth Thomas, Senior Project Assistant______________________________________________________________________________________________The IOM was chartered in 1970 as a component of the National Academy of Sciences to enlist distinguished members of the appropriate professionsin the examination of policy matters pertaining to the health of the public. In this, the Institute acts under both the Academy’s 1863 congressionalcharter responsibility to be an adviser to the federal government and its own initiative in identifying issues of medical care, research, and education.For additional information on the Forum on Neuroscience and Nervous System Disorders visit the project’s homepage at www.iom.edu/neuroforum,or call Bruce Altevogt at (202) 334-3984. 4
  23. 23. ROUNDTABLE ON TRANSLATING GENOMIC-BASED RESEARCH FOR HEALTHThe sequencing of the human genome is rapidly and span a broad range of issues relevant to theopening new doors to research and progress in translation process.biology, medicine, and health care. At the same time,these developments have produced a diversity of new Issues may include the integration and coordinationissues to be addressed. of genomic information into health care and public health including encompassing standards for geneticThe Institute of Medicine has convened a Roundtable screening and testing, improving informationon Translating Genomic-Based Research for Health technology for use in clinical decision making,that brings together leaders from academia, industry, ensuring access while protecting privacy, and usinggovernment, foundations and associations, and genomic information to reduce health disparities.representatives of patient and consumer interests who The patient and family perspective on the use ofhave a mutual concern and interest in addressing the genomic information for translation includes socialissues surrounding the translation of genome-based and behavioral issues for target populations. Thereresearch for use in maintaining and improving health. are evolving requirements for the health professionalThe mission of the Roundtable is to advance the field community, and the need to be able to understand andof genomics and improve the translation of research responsibly apply genomics to medicine and publicfindings to health care, education, and policy. The health.Roundtable will discuss the translation process,identify challenges at various points in the process, Of increasing importance is the need to identify theand discuss approaches to address those challenges. economic implications of using genome-based research for health. Such issues include incentives,The field of genomics and its translation involves cost-effectiveness, and sustainability.many disciplines, and takes place within differenteconomic, social, and cultural contexts, necessitating Issues related to the developing science base are alsoa need for increased communication and important in the translation process. Such issuesunderstanding across these fields. As a convening could include studies of gene-environmentmechanism for interested parties from diverse interactions, as well as the implications of genomicsperspectives to meet and discuss complex issues of for complex disorders such as addiction, mentalmutual concern in a neutral setting, the Roundtable: illness, and chronic diseases.fosters dialogue across sectors and institutions;illuminates issues, but does not necessarily resolve Roundtable sponsors include federal agencies,them; and fosters collaboration among stakeholders. pharmaceutical companies, medical and scientific associations, foundations, and patient/publicTo achieve its objectives, the Roundtable conducts representatives. For more information about thestructured discussions, workshops, and symposia. Roundtable on Translating Genomic-Based ResearchWorkshop summaries will be published and for Health, please visit our website at www.iom.edu/collaborative efforts among members are encouraged genomicroundtable or contact Adam Berger at 202-(e.g., journal articles). Specific issues and agenda 334-3756, or by e-mail at aberger2@nas.edu.topics are determined by the Roundtable membership, 500 Fifth Street, NW Phone: 202 334 3756 Washington, DC 20001-2721 Fax: 202 334 1329 www.iom.edu/genomicroundtable E-mail: aberger2@nas.edu 5
  24. 24. Institute of Medicine Roundtable on Translating Genomic-Based Research for Health Membership Wylie Burke, M.D., Ph.D. (Co-Chair) University of Washington Sharon Terry, M.A. (Co-Chair) Genetic AllianceNaomi Aronson, Ph.D. Debra Leonard, M.D., Ph.D.BlueCross/BlueShield Association College of American PathologistsEuan Ashley, M.R.C.P., D.Phil., FACC, FAHA Elizabeth Mansfield, Ph.D.American Heart Association Food & Drug AdministrationPaul R. Billings, M.D., Ph.D. Garry Neil, M.D.Life Technologies Corporation Johnson & JohnsonBruce Blumberg, M.D. Robert L. Nussbaum, M.D.Kaiser Permanente University of California San Francisco, School of MedicineDenise E. Bonds, M.D., M.P.H.National Heart, Lung, and Blood Institute Michelle Ann Penny, Ph.D. Eli Lilly and CompanyPhilip J. Brooks, Ph.D.Office of Rare Diseases Research Aidan Power, M.B., B.Ch., M.Sc., M.R.C.Psych. Pfizer Inc.C. Thomas Caskey, M.D., FACPBaylor College of Medicine Victoria M. Pratt, Ph.D., FACMG Quest Diagnostics Nichols InstituteSara Copeland, M.D.Health Resources and Services Administration Ronald Przygodzki, M.D. Department of Veterans AffairsVictor Dzau, M.D.Duke University Health System Allen D. Roses, M.D. Duke UniversityW. Gregory Feero, M.D., Ph.D.The Journal of the American Medical Association Kevin A. Schulman, M.D. Duke UniversityAndrew N. Freedman, Ph.D.National Cancer Institute Joan A. Scott, M.S., C.G.C. National Coalition for Health Professional EducationGeoffrey Ginsburg, M.D., Ph.D. in GeneticsDuke University David Veenstra, Pharm.D., Ph.D.Jennifer Hobin, Ph.D. University of WashingtonAmerican Society of Human Genetics Michael S. Watson, Ph.D.Richard Hodes, M.D. American College of Medical Genetics and GenomicsNational Institute on Aging Daniel Wattendorf, M.D. (Lt. Col)Sharon Kardia, Ph.D. Department of the Air ForceUniversity of Michigan, School of Public Health Catherine A. Wicklund, M.S., C.G.C.Mohamed Khan, M.D., Ph.D. National Society of Genetic CounselorsAmerican Medical Association Project StaffMuin Khoury, M.D., Ph.D. Adam C. Berger, Ph.D., Roundtable DirectorCenters for Disease Control and Prevention Sean P. David, M.D., D.Phil., Puffer/ABFM Fellow Claire Giammaria, M.P.H., Research AssociateThomas Lehner, Ph.D., M.P.H. Tonia Dickerson, Senior Program AssistantNational Institute of Mental HealthThe Institute of Medicine serves as adviser to the nation to improve health. Established in 1970 under the charter of the NationalAcademy of Sciences, the Institute of Medicine provides independent, objective, evidence-based advice to policy makers, healthprofessionals, the private sector, and the public. The mission of the Institute of Medicine embraces the health of people everywhere. 6
  25. 25. The National Cancer Policy Forum The National Cancer Policy Forum (NCPF) provides a continuous focus on cancer policy at the Institute of Medicine (IOM). IOM forums are designed to allow government, industry, academic, and other representatives to meet, confer, and plan on subject areas of mutual interest. The objectives of the NCPF are to identify emerging high priority policy issues in the nation’s effort to combat cancer and to examine those issues through convening activities that promote discussion about potential opportunities for action. These activities inform stakeholders about critical policy issues through published reports, and often provide input for planning formal IOM consensus committee studies. The IOM established the NCPF on May 1, 2005, to succeed the National Cancer Policy Board (1997-2005, the Board). The Board brought together leaders from the cancer community to identify and conduct studies and other activities contributing to cancer research, prevention, treatment, and public awareness. The combination of multi- disciplinary expertise (basic, clinical, and public health scientists, consumers, and advocates) and resources (grants from the National Cancer Institute (NCI) and Centers for Disease Control and Prevention (CDC), as well as smaller contributions from private sector organizations) allowed the Board to produce a remarkably original and diverse body of work contributing to improvements in knowledge and public policy. The NCPF continues to provide a focus within the National Academies for the consideration of issues in science, clinical medicine, public health, and public policy relevant to the goals of preventing, palliating, and curing cancer. The NCPF builds upon the work of the Board and enjoys a closer working relationship with its federal and non- federal sponsors. As a forum rather than a board, sponsors are full members with the academic, consumer, and policy community members. They bring ideas and requests to the deliberations and have the advantage of playing an active part in the discussions. Governmental sponsors represented on the NCPF include the NCI and CDC, and non- governmental sponsors include the American Association for Cancer Research, the American Cancer Society, the American Society of Clinical Oncology, the Association of American Cancer Institutes, Bristol-Myers Squibb, C-Change, the CEO Roundtable on Cancer, GlaxoSmithKline Oncology, Novartis, the Oncology Nursing Society, and Sanofi Oncology. Additional distinguished experts from the cancer community are also appointed as members to serve three-year terms. The NCPF operates under the aegis of the IOM Board on Health Care Services. The NCPF enables all members to be full participants in identifying and debating critical policy issues in cancer care and research, and in examining potential opportunities for actions. These convening activities result in published reports that are available to the public and may provide input to planning formal IOM consensus committee studies. Ideas for committee studies that emerge from the NCPF’s deliberations are handed off to an appropriate ad hoc committee appointed by IOM. The studies are conducted by NCPF staff and the committees often include one or more members of the NCPF. Forum sponsors often actively pursue activities to facilitate the implementation of recommendations made in these consensus reports, as well as suggestions put forth in NCPF workshops.500 Fifth Street, NW, Washington, DC 20001 Ph: 202-334-1233 Fax: 202-334-2862 7 www.iom.edu/Activities/Disease/NCPF
  26. 26. National Cancer Policy Forum Reports:Developing Biomarker-based Tools for Cancer Screening, Diagnosis and Therapy (2006), Effect ofthe HIPAA Privacy Rule on Health Research (2006), Implementing Cancer Survivorship CarePlanning (2007), Cancer in Elderly People (2007), Cancer-Related Genetic Counseling and Testing(2007), Improving the Quality of Cancer Clinical Trials (2008), Implementing Colorectal CancerScreening (2008), Multi-center Phase III Clinical Trials and the NCI Cooperative Group Program(2009), Ensuring Quality Cancer Care through the Oncology Workforce (2009), Assessing andImproving Value in Cancer Care (2009), Policy Issues in the Development of Personalized Medicinein Oncology (2010), A Foundation for Evidence-Driven Practice: A Rapid Learning System forCancer Care (2010), Extending the Spectrum of Precompetitive Collaboration in Oncology Research(2010), Direct to Consumer Genetic Testing (with the NRC, 2010), Policy Issues in Nanotechnologyand Oncology (2011), National Cancer Policy Summit: Opportunities and Challenges in CancerResearch and Care (2011), Patient-Centered Cancer Treatment Planning: Improving the Quality ofOncology Care (2011), Implementing a National Cancer Clinical Trials System for the 21st Century(2011), Facilitating Collaborations to Develop Combination Investigational Cancer Therapies(2011), The Role of Obesity in Cancer Survival and Recurrence (2012), Informatics Needs andChallenges in Cancer Research (2012), Reducing Tobacco-Related Cancer Incidence and Mortality(In Preparation)Spin-off IOM consensus committee reports:Cancer Biomarkers: The Promises and Challenges of Improving Detection and Treatment (2007),Beyond the HIPAA Privacy Rule: Enhancing Privacy, Improving Health through Research (2009),Evaluation of Biomarkers and Surrogate Endpoints in Chronic Disease (2010), A National CancerClinical Trials System for the 21st Century (2010), Evolution of Translational Omics (2012), Ensuringthe Quality of Cancer Care in an Aging Population (In preparation)Membership of the Forum:Chair - John Mendelsohn, MD, President, MD Anderson Cancer CenterVice Chair - Patricia Ganz, MD, Professor of Medicine, Jonsson Comprehensive Cancer CenterAmy Abernethy, MD, Director, Duke University School of Medicine Cancer Care Research ProgramRafael Amado, MD, Senior Vice President and Head of GlaxoSmithKline Oncology R&DFred Appelbaum, MD, Director, Clinical Research, Fred Hutchinson Cancer Research CenterPeter Bach, MD, MAPP, Member, Memorial Sloan-Kettering Cancer CenterEdward Benz, MD, President, Dana-Farber Cancer InstituteMonica Bertagnolli, MD, Professor of Surgery, Harvard University Medical SchoolOtis Brawley, MD, Chief Medical Officer and Executive Vice President, American Cancer SocietyMichael Caligiuri, MD, Director, Ohio State University Cancer Center, past President, AACIRenzo Canetta, MD, Vice President, Oncology Global Clinical Research, Bristol-Myers SquibbMichaele Chamblee Christian, MD, RetiredWilliam Dalton, PhD, MD, CEO, M2Gen, H. Lee Moffitt Cancer CenterWendy Demark-Wahnefried, PhD, RD, Professor and Chair, University of Alabama, BirminghamRobert Erwin, MS, President, Marti Nelson Cancer FoundationRoy Herbst, MD, PhD, Chief of Medical Oncology, Yale Cancer CenterThomas Kean, MPH, President and CEO, C-ChangeDouglas Lowy, MD, Deputy Director, Division of Basic Science, NCIDaniel R. Masys, MD, Affiliate Professor, Biomedical Informatics, University of WashingtonMartin Murphy, PhD, DMedSc, Chief Executive Officer, CEO Roundtable on CancerBrenda Nevidjon, RN, MSN, Professor, Duke University School of Nursing, past President, ONSSteven Piantadosi, MD, PhD, Director, Samuel Oschin Comprehensive Cancer InstituteLisa Richardson, MD, MPH, Associate Director, Division of Cancer Prevention and Control, CDCDebasish Roychowdhury, MD, Senior Vice President- Global Oncology, SanofiYa-Chen Tina Shih, PhD, Director, Program in the Economics of Cancer, University of ChicagoEllen Sigal, PhD, Chairperson and Founder, Friends of Cancer ResearchSteven Stein, MD, Senior Vice President, US Clinical Development & Medical Affairs, NovartisJohn Wagner, MD, PhD, Vice President, Clinical Pharmacology, Merck Research LaboratoriesRalph Weichselbaum, MD, Chairman, Radiation and Cellular Oncology, University of ChicagoJanet Woodcock, MD, Director, Center for Drug Evaluation and Research, FDAForum Director: Sharyl Nass, PhD 8
  27. 27. WORKSHOP
  28. 28. Forum on Drug Discovery, Development, and Translation Forum on Neuroscience and Nervous System Disorders Roundtable on Translating Genomic-Based Research for Health National Cancer Policy Forum Speaker Biographies Sharing Clinical Research Data: An IOM Workshop October 4-5, 2012Myles Axton, Ph.D.Myles Axton is the editor of Nature Genetics. He was a university lecturer in molecular andcellular biology at the University of Oxford and a Fellow of Balliol College from 1995 to 2003. Heobtained his degree in genetics at Cambridge in 1985, and his doctorate at Imperial College in1990, and between 1990 and 1995 did postdoctoral research at Dundee and at MIT’sWhitehead Institute. Myles’s research made use of the advanced genetics of Drosophila tostudy genome stability by examining the roles of cell cycle regulators in life cycle transitions. Hisinterests broadened into human genetics, genomics and systems biology through lecturing andfrom tutoring biochemists, zoologists and medical students from primary research papers.Helping to establish Oxford’s innovative research MSc. in Integrative Biosciences led Myles torealize the importance of the integrative overview of biomedical research. As a full timeprofessional editor he is now in a position to use this perspective to help coordinate research ingenetics.Jesse A. Berlin, Sc.D.After spending 15 years as a faculty member at the University of Pennsylvania, in the Centerfor Clinical Epidemiology and Biostatistics, under the direction of Dr. Brian Strom, Dr. Berlin leftthe University of Pennsylvania to join Janssen Research & Development, where he is currentlyVice President of Epidemiology. He has authored or coauthored over 230 publications in a widevariety of clinical and methodological areas, including papers on the study of meta-analyticmethods as applied to both randomized trials and epidemiology. He served on an Institute ofMedicine Committee that developed recently-released recommendations for the use ofsystematic reviews in clinical effectiveness research, and currently serves on the ScientificAdvisory Committee to the Observational Medical Outcomes Partnership, a public-privatepartnership aimed at understanding methodology for assessing drug safety in large,administrative databases. He is also a fellow of the American Statistical Association.Josephine P. Briggs, M.D.Dr. Briggs is the Director of the National Center for Complementary and Alternative Medicine,and Acting Director, Division of Clinical Innovation, National Center for Advancing TranslationalSciences, NIH. An accomplished researcher and physician, Dr. Briggs received her A.B. inbiology from Harvard-Radcliffe College and her M.D. from Harvard Medical School. Shecompleted her residency training in internal medicine and nephrology at the Mount Sinai Schoolof Medicine, followed by a fellowship at Yale, then work as a research scientist at thePhysiology Institute at the University of Munich.In 1985, Dr. Briggs moved to the University of Michigan where she held several academicpositions, including associate chair for research in the Department of Internal Medicine andprofessorships in the Division of Nephrology, Department of Internal Medicine, and theDepartment of Physiology. She joined the National Institutes of Health (NIH) in 1997 as directorof the Division of Kidney, Urologic, and Hematologic Diseases at the National Institute ofDiabetes and Digestive and Kidney Diseases. In 2006, Dr. Briggs accepted a position as senior 1
  29. 29. scientific officer at the Howard Hughes Medical Institute. In January 2008, she returned to NIHas the Director of the National Center for Complementary and Alternative Medicine.Dr. Briggs has published more than 175 research articles, book chapter, and scholarlypublications and has served on the editorial boards of several journals, and was deputy editorfor the Journal of Clinical Investigation. Dr. Briggs is an elected member of the AmericanAssociation of Physicians and the American Society of Clinical Investigation and a fellow of theAmerican Association for the Advancement of Science. She is a recipient of many awards andprizes, including the Volhard Prize of the German Nephrological Society, the Alexander vonHumboldt Scientific Exchange Award, and NIH Directors Awards for her role in the developmentof the Trans-NIH Type I Diabetes Strategic Plan and her leadership of the Trans-NIH Zebrafishcommittee. Dr. Briggs is also a member of the NIH Steering Committee, the senior mostgoverning board at the NIH.Robert M. Califf, M.D.Dr. Califf is the Vice Chancellor for Clinical and Translational Research, Director of the DukeTranslational Medicine Institute (DTMI), and Professor of Medicine in the Division of Cardiologyat Duke University Medical Center in Durham, NC. He leads a multifaceted organization thatseeks to transform how scientific discoveries are translated into improved health outcomes.Prior to leading the DTMI, he was the founding director of the Duke Clinical Research Institute(DCRI), one of the nations premier academic research organizations. He is editor-in-chief of theAmerican Heart Journal, the oldest cardiovascular specialty journal, and a practicing cardiologistat Duke University Medical Center.Born in 1951 in Anderson, SC, Dr. Califf attended high school in Columbia, where he was amember of the 1969 AAAA SC Championship basketball team. After high school, Dr. Califfattended Duke University, graduating summa cum laude and Phi Beta Kappa in 1973. Heremained at Duke for medical school, where he was selected for the Alpha Omega Alphamedical honor society. After graduating from Duke University School of Medicine in 1978, hecompleted a residency in internal medicine at the University of California, San Francisco,returning to Duke for a cardiology fellowship.Dr. Califf is board certified in internal medicine (1984) and cardiology (1986), and was named aMaster of the American College of Cardiology in 2006. An international leader in the fields ofcardiovascular medicine, healthcare outcomes, quality of care, and medical economics, he hasauthored or coauthored more than 1,000 peer-reviewed articles and is among the mostfrequently cited authors in medicine. He is also a contributing editor for TheHeart.org, an onlineinformation resource for healthcare professionals working in the field of cardiovascularmedicine.As founder and for a decade director of the DCRI, Dr. Califf led many landmark clinical trials andhealth services research projects, and remains actively involved in designing, leading, andconducting multinational clinical trials. Under his guidance, DCRI grew into an organization withmore than 1000 employees and an annual budget of over $1 00 million; its umbrellaorganization, the DTMI, now has an annual budget of more than $300 million. Supported in partby a Clinical and Translational Science Award (CTSA) from the National Institutes of Health, theDTMI works with government agencies, academic partners, research foundations, and themedical products industry to conduct innovative research spanning multiple therapeutic arenasand scientific disciplines. 2
  30. 30. Forum on Drug Discovery, Development, and Translation Forum on Neuroscience and Nervous System Disorders Roundtable on Translating Genomic-Based Research for Health National Cancer Policy ForumDr. Califf serves as co-chair of the first Principal Investigators Steering Committee of the CTSAand has served on the FDAs Cardiorenal Advisory Panel, and on the Institute of Medicines(IOM) Pharmaceutical Roundtable, Committee on Identifying and Preventing Medication Errors,and Committee on Nutritional Biomarkers. In 2008, he was part of the subcommittee of theFDAs Science Board that recommended sweeping reform ofthe agencys science base. He wasalso a member of the IOM committees that recommended Medicare coverage of clinical trialsand the removal of ephedra from the market. Dr. Califf is currently a member ofthe IOM Forumin Drug Discovery, Development, and Translation and a member of the National AdvisoryCouncil on Aging.Reflecting his interests in healthcare quality, Dr. Califf was the founding director of thecoordinating center of the Centers for Education & Research on Therapeutics, a public-privatepartnership 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-privatepartnership focused on improving the clinical trials system. He is also chair of the ClinicalResearch Forum, an organization of academic health and science system leaders devoted toimproving the clinical research enterprise.Dr. Califf is married to Lydia Carpenter, and the couple has three children-Sharon Califf, agraduate of Elon College; Sam, a graduate student at the University of Colorado-Boulder; andTom, a graduate of Duke-and one grandchild. Dr. Califf enjoys time with his family, works at hisgolf game, listens to music, and remains an ardent supporter of the Duke mens and womensbasketball programs.Michael N. Cantor, M.D., M.A., FACPMichael Cantor is currently Senior Director, Information Strategy and Analytics, in Pfizer’sClinical Informatics and Innovation group. His work focuses on leveraging data reuse andintegration to support future horizons of scientific decision support for precision medicine. He iscurrently co-leading several initiatives around the secondary use of clinical data, includingPfizer’s ePlacebo/eControls database, as well as its comprehensive Clinical Lab Data Catalog.He created and co-leads the MEDIC (Multisite Electronic Data Infectious Disease Consortium)project, which aims to partner with academic medical centers to perform observational studiesusing data from electronic medical record (EMR) systems. He has served as an advisor toprograms across each of Pfizer’s Business Units, as well as the Worldwide Research andDevelopment organization, on the role of healthcare IT in advancing their strategic priorities.Dr. Cantor previously lead Pfizer Business Technology’s “Data Without Borders” strategy, withthe aim of advancing data sharing and reuse, both internally and externally, to advancePrecision Medicine. Outside of Pfizer, he has been a member of AMIA’s public policy committeefor six years, and led the committee’s initiative to update its positions around data stewardshipand reuse.Prior to joining Pfizer, Michael was the Chief Medical Information Officer for the SouthManhattan Healthcare Network of the New York City Health and Hospitals Corporation, basedat Bellevue Hospital in Manhattan. His work there focused on developing the network’s EMRsystem to improve patient safety and on using the network’s clinical data warehouse forresearch. He continues to see patients 1 day/week at Bellevue, and is a Clinical AssistantProfessor of Medicine at NYU School of Medicine. 3
  31. 31. Michael completed his residency in internal medicine and informatics training at Columbia, hasan M.D. from Emory University, and an A.B. from Princeton.Carolyn Compton, M.D., Ph.D.Dr. Carolyn Compton is the President and CEO of Critical Path Institute. She was most recentlythe Director of the Office of Biorepositories and Biospecimen Research (OBBR) and theExecutive Director of the Cancer Human Biobank (caHUB) project at the National CancerInstitute. In these capacities, she had leadership responsibility for strategic initiatives thatincluded the Innovative Molecular Analysis Technologies for Cancer program, the BiospecimenResearch Network program, and the NCI Community Cancer Centers project. She is an adjunctProfessor of Pathology at the Johns Hopkins School of Medicine.She received her M.D. and Ph.D. in degrees from Harvard Medical School and the HarvardGraduate School of Arts and Sciences. She trained in Pathology at Harvards Brigham andWomens Hospital and is boarded in both Anatomic Pathology and Clinical Pathology. Shecame to the NCI from McGill University where she had been the Strathcona Professor and Chairof Pathology and the Pathologist-in-Chief of McGill University Health Center from 2000-2005.Prior to this, she had been a Professor of Pathology Harvard Medical School, the Director ofGastrointestinal Pathology at the Massachusetts General Hospital, and the Pathologist-in-Chiefof the Shriners Hospital For Crippled Children, Boston Burns Unit for 15 years. During this timeshe served as the Chair of the Pathology Committee of the Cancer and Leukemia Group B for12 years. Her research interests are in colon and pancreatic cancer as well as epithelial biologyand wound healing.Dr. Compton has held many national and international leadership positions in pathology andcancer-related professional organizations. She is a Fellow of the College of AmericanPathologists and a Fellow of the Royal Society of Medicine. Currently, she is the Chair of theAmerican Joint Committee on Cancer (AJCC), serves on the Executive Committee of theCommission on Cancer of the American College of Surgeons, and serves as the PathologySection Editor for Cancer. She is a past Chair of the Cancer Committee of the College ofAmerican Pathologists and was Editor of the first edition of the CAP Cancer Protocols(Reporting on Cancer Specimens) used as standards for COC accreditation. Among her awardsare the ISBER Award for Outstanding Achievement in Biobanking, the NIH Directors Award, theNIH Award of Merit, and the CAP Frank W. Hartman Award. She has published more than 500original scientific papers, reports, review articles, books and abstracts.Neil de Crescenzo, M.B.A.Mr. de Crescenzo is Senior Vice President and General Manager for Health Sciences at Oracle.He is responsible for managing Oracles solution groups, strategic planning, productdevelopment, and sales, service, and support for the industry solutions sold into the healthcareand life sciences markets worldwide. Mr. de Crescenzo brings more than 20 years ofoperational and IT leadership across healthcare and life sciences to his work with customersand partners worldwide.Prior to joining Oracle, Mr. de Crescenzo held a number of leadership positions during hisdecade at IBM Corporation, working with healthcare and life sciences clients worldwide. Prior toentering the information technology industry, he held leadership positions in healthcareoperations at multiple medical centers and a major health insurer. Mr. de Crescenzo began hiscareer in investment banking, working with U.S. and European clients in the areas of corporatefinance and mergers and acquisitions. 4
  32. 32. Forum on Drug Discovery, Development, and Translation Forum on Neuroscience and Nervous System Disorders Roundtable on Translating Genomic-Based Research for Health National Cancer Policy ForumMr. de Crescenzo has been a keynote speaker at numerous industry conferences worldwideand is quoted frequently on industry issues. In 2005, he was named one of the "Top 25 MostInfluential Consultants" by Consulting Magazine.Mr. de Crescenzo has a BA in political science from Yale University and an MBA in hightechnology from Northeastern University.Peter Doshi, Ph.D.Dr. Doshi is a postdoctoral fellow in comparative effectiveness research at the Johns HopkinsUniversity School of Medicine.His overarching research interests are in improving the bases for credible evidence synthesis tosupport and improve the quality of evidence-based medical and health policy related decisionmaking.In 2009, he joined a Cochrane systematic review team evaluating neuraminidase inhibitors forthe treatment and prevention of influenza. Rather than focusing on publications, their reviewevaluates regulatory information including clinical study reports.He received his A.B. in anthropology from Brown University, A.M. in East Asian studies fromHarvard University, and Ph.D. in History, Anthropology, and Science, Technology and Societyfrom the Massachusetts Institute of Technology.Kelly Edwards, Ph.D.An associate professor in the School of Medicines Department of Bioethics and Humanities, Dr.Edwards also is a core faculty member for the UW Institute for Public Health Genetics. Shereceived both her Master of Arts degree in Medical Ethics and her Ph.D. in Philosophy ofEducation from the UW.Dr. Edwards work incorporates communication and public engagement as an ethical obligationfor clinicians and researchers. She is the director of the Ethics and Outreach Core for the UWCenter for Ecogenetics and Environmental Health, which is funded by the National Institute ofEnvironmental Health Sciences. She also is a co-director of the Regulatory Support andBioethics Core for the Institute for Translational Health Sciences (ITHS), a partnership of theUW, Fred Hutchinson Cancer Research Center, Seattle Childrens and other regionalinstitutions and community and tribal groups. Funded by the National Institutes of Health (NIH),the ITHS assists researchers with translating their scientific discoveries into practice.In addition, Dr. Edwards is a lead investigator with UW Center for Genomics and HealthcareEquality, funded by the NIHs National Human Genome Research Institute. Since 2004, she hasbeen the faculty advisor for the Forum on Science, Ethics and Policy, groups of graduate andprofessional students and postdoctoral fellows at the UW and University of Colorado thatpromote dialogue on issues concerning science and society.To further engage people in conversations about ethical dimensions of science and medicine,Dr. Edwards has facilitated Community Conversations and the Public Health Café, a series ofevents hosted in Seattle by the Northwest Association for Biomedical Research. Nationally, Dr.Edwards contributes to issues of ethical research practices with the Genetic Alliance, a healthadvocacy organization; Sage Bionetworks, a local non-profit; and the Institute of Medicine.Her courses include "Inquiry-Based Science Communication," "Applied Research Ethics,""Community-Based Participatory Research: A Model for Genetics Research with Native 5

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