Glomerular Filtration and determinants of glomerular filtration .pptx
Sharing Clinical Research Data: an IOM Workshop
1. 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
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. Sharing Clinical Research Data:
An Institute of Medicine Workshop
TABLE OF CONTENTS
October 4 - 5, 2012
Washington, DC
ITEMS TAB/PAGE
Agenda AGENDA
Sharing Clinical Research Data 1
COORDINATING ROUNDTABLE AND FORUMS
COORDINATING
ROUNDTABLE
Coordinating 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
WORKSHOP
Workshop, October 4-5 WORKSHOP
• Speaker Biographies 1
BACKGROUND ARTICLES
BACKGROUND
Background Reading for Workshop1 ARTICLES
Benefits
• 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 evidence
Models
• 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 57
1
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. ITEMS TAB/PAGE
Standardization 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 96
Culture 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
TRAVEL
Travel Information INFORMATION
• Logistics Memo 1
• Map of National Academies Building 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, DC
Background:
Pharmaceutical companies, academic institutions, advocacy organizations, and government
agencies such as FDA and NIH have large quantities of clinical research data. Increased data
sharing could facilitate scientific and public health advances, among other potential benefits to
patients and society. Much of this information, however, is never published or shared. More
specifically, study results are not always published and where results are published, they
typically only include summary-level data; participant-level data is privately held and rarely
shared or revealed publicly.
The workshop will explore the benefits of and barriers to the sharing of clinical research data
and will help identify strategies for enhancing sharing both within sectors and across sectors. To
make the workshop scope manageable, the workshop will focus on data resulting from
preplanned interventional studies of human subjects. While recognizing the importance of other
data sources such as observational studies and electronic health records, this focus was selected
to 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 the
workshop to the extent that these models provide lessons and best practices applicable to sharing
preplanned interventional clinical research data.
The workshop is being jointly organized by the Institute of Medicine’s Forum on Drug
Discovery, Development, and Translation; Forum on Neuroscience and Nervous System
Disorders; National Cancer Policy Forum; and Roundtable on Translating Genomic-Based
Research for Health. Participants will be invited from industry, academia, government agencies
such 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. 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 One
8:30 a.m. Opening Remarks
SHARON TERRY, Workshop Chair
President and Chief Executive Officer
Genetic Alliance
SESSION I: BENEFITS OF SHARING CLINICAL RESEARCH DATA
Session 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 Health
8:50 a.m. Fundamentals and Benefits of Sharing Participant-Level Clinical Trial Data
ELIZABETH LODER
Clinical Epidemiology Editor
British Medical Journal
9:10 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. 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
9:30 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 Development
10:30 a.m. BREAK
SESSION II: DATA SHARING MODELS: DESIGN, BEST PRACTICES, AND
LESSONS LEARNED
Session 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, LLC
10:55 a.m. The Limits of Summary Data Reporting: Lessons from ClinicalTrials.gov
3
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 Health
11:10 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 University
12:10 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. 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 Washington
12:40 p.m. Discussion among speakers, panelists, and audience
Discussant:
Sally Okun, Health Data Integrity & Patient Safety, PatientsLikeMe
1:00 p.m. LUNCH
KEYNOTE CASE STUDY: DISTRIBUTED SYSTEMS FOR CLINICAL RESEARCH
INFORMATION SHARING
1:30 p.m. RICHARD PLATT
Professor and Chair
Department of Population Medicine
Harvard Pilgrim Health Care Institute and Harvard Medical School
1:50 p.m. Discussion with speaker and audience
Moderator:
Sharon Terry, Genetic Alliance
SESSION III: STANDARDIZATION AND GOVERNANCE
Session 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. 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 Institute
2:10 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 Administration
2:25 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 audience
3:30 p.m. BREAK
3:45 p.m. Cost-Benefit Analysis of Retrospective vs. Prospective Data Standardization
6
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 Administration
4:00 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 Administration
4:30 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. 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. 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 Two
8:00 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 SHARING
Session 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 University
8:20 a.m. Clinical Trial Data Transparency: European Medicines Agency Perspective
HANS-GEORG EICHLER
Senior Medical Officer
European Medicines Agency
8:40 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. 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
8:55 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 Medicine
9:10 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 Health
10:10 a.m. Discussion with speakers and audience
10:30 a.m. BREAK
10:45 a.m. Facilitating Patient Ownership of Clinical Trial Data: Technical Challenges
and Opportunities
JOHN WILBANKS
Director
Sage Bionetworks
10
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 Technology
11:15 a.m. Discussion with speakers and audience
SESSION V: NEXT STEPS AND FUTURE DIRECTIONS
Session 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 Alliance
11:40 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. 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 University
12:00 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 Administration
12:45 p.m. Adjourn
12
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 Task
Pharmaceutical 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 sharing
tion and the National Institutes of Health have large of clinical research data. Participants will be invited
quantities of clinical research data. Data sharing within from industry, academia, government agencies such as
each sector and across sectors could facilitate scientific FDA and NIH, disease advocacy groups, and other
and public health advances and could enhance analysis stakeholder groups. The workshop will feature invited
of 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-
terprise
ed products.
o Benefits to the analysis of safety and efficacy
A number of efforts are currently underway to enhance Identify barriers and challenges to sharing clinical
public/private partnerships and foster collaboration research data
within the pharmaceutical sector to advance research Explore strategies to address these barriers and
and enhance the discovery and development of drugs, challenges, including identifying priority actions
devices, and diagnostic tools. Examples include the and “low-hanging fruit” opportunities
Analgesic Clinical Trials Innovation, Opportunities, Discuss strategies for using these potentially large
and Networks (ACTION) Initiative; the Foundation data sets to facilitate scientific and public health
for the National Institute of Health’s Biomarkers Con- advances.
sortium; the Innovative Medicines Initiative (Europe);
Critical Path Institute; Sage Bionetworks’ Clinical Tri- Planning Committee
al Comparator Arm Partnership; and the Life Sciences Sharon Terry, chair, Genetic Alliance
Consortium’s MetaPharm project. Planned within the Josephine P. Briggs, National Institutes of Health
context of these various efforts, this workshop will Timothy Coetzee, National Multiple Sclerosis Society
focus specifically on data sharing, which is one com- Steven Goodman, Stanford University
ponent of larger efforts to build partnerships and en- Robert A. Harrington, Stanford University
hance collaboration within and among sectors on Lynn Hudson, Critical Path Institute
research, development, and assessment of pharmaceu- Charles Hugh-Jones, Sanofi Oncology
tical products. Jan Johannessen, Food and Drug Administration
This project will be a coordinated effort of the IOM's Jeffrey S. Nye, Johnson and Johnson
Forum on Drug Discovery, Development and Transla- Richard Platt, Harvard Medical School
tion; Forum on Neuroscience and Nervous System William Z. Potter, FNIH Biomarkers Consortium
Disorders; National Cancer Policy Forum; and Frank W. Rockhold, GlaxoSmithKline
Roundtable on Translating Genomic-Based Research Michael Rosenblatt, Merck
for Health. All four Forums/Roundtables have done Vicki Seyfert-Margolis, Food and Drug Administration
previous work on this topic and this issue cuts across Deborah A. Zarin, National Institutes of Health
the 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 appropriate
professions in the examination of policy matters pertaining to the health of the public. In this, the Institute acts under both the Academy’s
1863 congressional charter 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 about this workshop, contact Rebecca English at renglish@nas.edu.
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 Collaborative
Discovery, Development, and Translation was created in Research
2005 by the IOM’s Board on Health Sciences Policy to
provide a unique platform for dialogue and collaboration The Forum has established an initiative to examine
among thought leaders and stakeholders in government, the state of clinical trials in the U.S., identify areas of
academia, industry, foundations and patient advocacy. strength and weakness in our current clinical trial
The Forum brings together leaders from private sector enterprise, and consider transformative strategies for
sponsors of biomedical and clinical research, federal enhancing the ways in which clinical research is
agencies sponsoring and regulating biomedical and organized and conducted. Workshops and meetings held
clinical research, the academic community, and in 2009 and 2010 considered case studies in four disease
consumers, and in doing so serves to educate the policy areas; and included discussions around issues of
community about issues where science and policy management of conflict of interest, and addressing
intersect. regulatory and administrative impediments to the conduct
The Forum convenes several times each year to of clinical trials. Meetings in 2011 will address how to
identify and discuss key problems and strategies in the move toward greater public engagement in and
discovery, development, and translation of drugs. To understanding of the clinical trial enterprise, and
supplement the perspectives and expertise of its members, establishing a framework for a transformed national
the 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 and
policy community in discussing areas of concern in the Addressing Urgent Global Health Problems
science and policy of drug development. The Forum’s
public meetings focus substantial public attention on The Forum is sponsoring a series of workshops on the
critical areas of drug development, focusing on the major global problem of MDR TB. The Forum held a
themes 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 (anticipated
from basic science to new therapeutics faces challenges 2011). Also in 2011, the Forum will convene a focused
on many fronts. New paradigms for discovering and initiative to consider the global drug supply chain for
developing drugs are being sought to bridge the ever- quality-assured second-line drugs for tuberculosis.
widening gap between scientific discoveries and
translation of those discoveries into life-changing Promoting Public Understanding of Drug
medications. The Forum has explored these issues from Development
many perspectives—emerging technology platforms,
regulatory efficiency, intellectual property concerns, the Successful introduction of new therapeutic entities
potential for precompetitive collaboration, and innovative requires testing in an informed and motivated public. The
business models that address the “valley of death.” Forum has spent concerted effort to understand what
limits public participation and how to enhance more
Strengthening 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 the
attention on the scientific basis for the regulation of drugs. spring and fall of 2010 addressed these issues. The Forum
In February 2010, the Forum held a workshop that plans to continue to work with multiple stakeholders to
examined the state of the science of drug regulation and improve public understanding of and participation in the
considered 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. 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 KAUFFMAN
FasterCures National Institute of Neurological Disorders and Stroke
HUGH AUCHINCLOSS JACK KEENE
National Institute of Allergy and Infectious Diseases Duke University Medical Center
LESLIE BENET RONALD KRALL
University of California, San Francisco University of Pennsylvania
ANN BONHAM FREDA LEWIS-HALL
Association of American Medical Colleges Pfizer Inc.
LINDA BRADY MARK MCCLELLAN
National Institute of Mental Health Brookings Institution
ROBERT CALIFF CAROL MIMURA
Duke University Medical Center University of California, Berkeley
THOMAS CASKEY ELIZABETH (BETSY) MYERS
Baylor College of Medicine Doris Duke Charitable Foundation
GAIL CASSELL JOHN ORLOFF
Harvard Medical School Novartis Pharmaceuticals Corporation
PETER CORR AMY PATTERSON
Celtic Therapeutics, LLLP NIH Office of the Director
ANDREW DAHLEM MICHAEL ROSENBLATT
Eli Lilly & Co. Merck & Co., Inc.
TAMARA (MARA) DARSOW JANET SHOEMAKER
American Diabetes Association American Society for Microbiology
JIM DOROSHOW ELLEN SIGAL
National Cancer Institute Friends of Cancer Research
GARY FILERMAN ELLIOTT SIGAL
Atlas Health Foundation Bristol-Myers Squibb
GARRET FITZGERALD ELLEN STRAHLMAN
University of Pennsylvania School of Medicine GlaxoSmithKline
MARK GOLDBERGER NANCY SUNG
Abbott Pharmaceuticals Burroughs Wellcome Fund
HARRY GREENBERG JANET TOBIAS
Stanford University School of Medicine Ikana Media
STEPHEN GROFT JOANNE WALDSTREICHER
Office of Rare Diseases Research, NIH NCATS Janssen Research & Development
LYNN HUDSON JANET WOODCOCK
The Critical Path Institute FDA Center for Drug Evaluation and Research
TOM INSEL PROJECT STAFF
National Center for Advancing Translational Sciences Anne Claiborne, J.D., M.P.H., Forum Director
Rebecca English, M.P.H., Associate Program Officer
MICHAEL KATZ Rita Guenther, Ph.D., Program Officer
March of Dimes Foundation Robin Guyse, Senior Program Assistant
Elizabeth Tyson, Research Associate
The IOM was chartered in 1970 as a component of the National Academy of Sciences to enlist distinguished members of the appropriate professions in the
examination of policy matters pertaining to the health of the public. In this, the Institute acts under both the Academy’s 1863 congressional charter
responsibility to be an adviser to the federal government and its own initiative in identifying issues of medical care, research, and education.
2
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 Disorders
and Nervous System Disorders focuses on building
partnerships to further understand the brain and nervous Understanding the roles of genes and their products
system, 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 understanding
The forum focuses on the six themes outlined below, the brain and nervous system. The role of genetics in
and serves to educate the public, press, and policy disease states needs to be more completely understood
makers regarding these issues. to develop appropriate treatments and interventions.
The Forum brings together leaders from private The forum discusses approaches to better
sector sponsors of biomedical and clinical research, understanding environmental and multigenetic factors
federal agencies sponsoring and regulating biomedical that influence susceptibility, progression, and severity
and clinical research, the academic community, and of disease.
consumers.
The Forum will sponsor workshops for members Cognition and Behavior
and the public to discuss approaches to resolve key
challenges 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 complex
associated 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 their
mechanism 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 lead
to a more complete understanding of the agents that Tools that assist in better understanding of the basic
damage cells and trigger cell death, as well as the and higher order functions of the brain aid in research
mechanisms that carry out the process. For example, and understanding of disorders of the brain. This area
protein aggregation has an important role in focuses on animal and computer models and
neurodegeneration 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 Issues
Mental 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 the
its role in mental illness and addictive behavior is engagement of these patient populations in research
critical to developing new treatments for these efforts. Clinical research is also examined that
conditions. Especially important is improving the elucidates environmental, genetic, social, and cultural
understanding of the neuronal mechanisms that give risk and protective factors which can guide the
rise to mental illnesses, increased risk behavior, and development of prevention and early intervention
resilience. The Forum discusses mechanisms that strategies.
might take advantage of the evolving nature of science
and the opportunities it brings to investigating the brain
and behavior using cross disciplinary approaches that
expand the understanding of how complex mental
illnesses and addictions arise from individual
components.
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. INSTITUTE OF MEDICINE
Forum on Neuroscience and Nervous System Disorders
STEVE HYMAN, (Chair)
The Broad Institute
SUSAN AMARA STORY LANDIS
Society for Neuroscience National Institute on Neurological Disease and Stroke
MARC BARLOW ALAN LESHNER
GE Healthcare, Inc. American Association for the Advancement of Science
MARK BEAR HUSSEINI MANJI
Massachusetts Institute of Technology Johnson and Johnson Pharmaceutical Research and Development,
Inc.
KATJA BROSE
Neuron DAVID MICHELSON
Merck Research Laboratories
DANIEL BURCH
CeNeRx Biopharma RICHARD MOHS
Lilly Research Laboratories
C. THOMAS CASKEY
Baylor College of Medicine JONATHAN MORENO
University of Pennsylvania School of Medicine
TIMOTHY COETZEE
Fast Forward, LLC ALEXANDER OMMAYA
U.S. Department of Veterans Affairs
EMMELINE EDWARDS
NIH Neuroscience Blueprint ATUL PANDE
GlaxoSmithKline, Inc
MARTHA FARAH
University of Pennsylvania STEVEN PAUL
Weill Cornell Medical College
RICHARD FRANK
GE Healthcare, Inc. TODD SHERER
Michael J. Fox Foundation for Parkinson’s Research
DANIEL GESCHWIND
University of California Los Angeles PAUL SIEVING
National Eye Institute
HANK GREELY
Stanford University JUDY SIUCIAK
Foundation for the National Institutes of Health
MYRON GUTMANN
National Science Foundation MARC TESSIER-LAVIGNE
The Rockefeller University
RICHARD HODES
National Institute on Aging WILLIAM THIES
Alzheimer’s Association
STEVEN HYMAN
The Broad Institute NORA VOLKOW
National Institute on Drug Abuse
THOMAS INSEL
National Institute on Mental Health KENNETH WARREN
National Institute on Alcohol Abuse and Alcoholism
PHILLIP IREDALE
Pfizer Global Research and Development JOHN WILLIAMS
Wellcome Trust
DANIEL JAVITT
Nathan S. Kline Institute for Psychiatric Research STEVIN H. ZORN
Lundbeck USA
FRANCES JENSEN
University 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 professions
in the examination of policy matters pertaining to the health of the public. In this, the Institute acts under both the Academy’s 1863 congressional
charter 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. ROUNDTABLE ON TRANSLATING GENOMIC-BASED RESEARCH FOR HEALTH
The sequencing of the human genome is rapidly and span a broad range of issues relevant to the
opening 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 coordination
issues to be addressed. of genomic information into health care and public
health including encompassing standards for genetic
The Institute of Medicine has convened a Roundtable screening and testing, improving information
on 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 using
government, 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 of
have a mutual concern and interest in addressing the genomic information for translation includes social
issues surrounding the translation of genome-based and behavioral issues for target populations. There
research for use in maintaining and improving health. are evolving requirements for the health professional
The mission of the Roundtable is to advance the field community, and the need to be able to understand and
of genomics and improve the translation of research responsibly apply genomics to medicine and public
findings 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 the
and 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 different
economic, social, and cultural contexts, necessitating Issues related to the developing science base are also
a need for increased communication and important in the translation process. Such issues
understanding across these fields. As a convening could include studies of gene-environment
mechanism for interested parties from diverse interactions, as well as the implications of genomics
perspectives to meet and discuss complex issues of for complex disorders such as addiction, mental
mutual 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/public
To achieve its objectives, the Roundtable conducts representatives. For more information about the
structured discussions, workshops, and symposia. Roundtable on Translating Genomic-Based Research
Workshop 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. 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 Alliance
Naomi Aronson, Ph.D. Debra Leonard, M.D., Ph.D.
BlueCross/BlueShield Association College of American Pathologists
Euan Ashley, M.R.C.P., D.Phil., FACC, FAHA Elizabeth Mansfield, Ph.D.
American Heart Association Food & Drug Administration
Paul R. Billings, M.D., Ph.D. Garry Neil, M.D.
Life Technologies Corporation Johnson & Johnson
Bruce Blumberg, M.D. Robert L. Nussbaum, M.D.
Kaiser Permanente University of California San Francisco,
School of Medicine
Denise E. Bonds, M.D., M.P.H.
National Heart, Lung, and Blood Institute Michelle Ann Penny, Ph.D.
Eli Lilly and Company
Philip 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., FACP
Baylor College of Medicine Victoria M. Pratt, Ph.D., FACMG
Quest Diagnostics Nichols Institute
Sara Copeland, M.D.
Health Resources and Services Administration Ronald Przygodzki, M.D.
Department of Veterans Affairs
Victor Dzau, M.D.
Duke University Health System Allen D. Roses, M.D.
Duke University
W. Gregory Feero, M.D., Ph.D.
The Journal of the American Medical Association Kevin A. Schulman, M.D.
Duke University
Andrew N. Freedman, Ph.D.
National Cancer Institute Joan A. Scott, M.S., C.G.C.
National Coalition for Health Professional Education
Geoffrey Ginsburg, M.D., Ph.D. in Genetics
Duke University
David Veenstra, Pharm.D., Ph.D.
Jennifer Hobin, Ph.D. University of Washington
American Society of Human Genetics
Michael S. Watson, Ph.D.
Richard Hodes, M.D. American College of Medical Genetics and Genomics
National Institute on Aging
Daniel Wattendorf, M.D. (Lt. Col)
Sharon Kardia, Ph.D. Department of the Air Force
University of Michigan, School of Public Health
Catherine A. Wicklund, M.S., C.G.C.
Mohamed Khan, M.D., Ph.D. National Society of Genetic Counselors
American Medical Association
Project Staff
Muin Khoury, M.D., Ph.D. Adam C. Berger, Ph.D., Roundtable Director
Centers for Disease Control and Prevention Sean P. David, M.D., D.Phil., Puffer/ABFM Fellow
Claire Giammaria, M.P.H., Research Associate
Thomas Lehner, Ph.D., M.P.H. Tonia Dickerson, Senior Program Assistant
National Institute of Mental Health
The Institute of Medicine serves as adviser to the nation to improve health. Established in 1970 under the charter of the National
Academy of Sciences, the Institute of Medicine provides independent, objective, evidence-based advice to policy makers, health
professionals, the private sector, and the public. The mission of the Institute of Medicine embraces the health of people everywhere.
6
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. National Cancer Policy Forum Reports:
Developing Biomarker-based Tools for Cancer Screening, Diagnosis and Therapy (2006), Effect of
the HIPAA Privacy Rule on Health Research (2006), Implementing Cancer Survivorship Care
Planning (2007), Cancer in Elderly People (2007), Cancer-Related Genetic Counseling and Testing
(2007), Improving the Quality of Cancer Clinical Trials (2008), Implementing Colorectal Cancer
Screening (2008), Multi-center Phase III Clinical Trials and the NCI Cooperative Group Program
(2009), Ensuring Quality Cancer Care through the Oncology Workforce (2009), Assessing and
Improving Value in Cancer Care (2009), Policy Issues in the Development of Personalized Medicine
in Oncology (2010), A Foundation for Evidence-Driven Practice: A Rapid Learning System for
Cancer Care (2010), Extending the Spectrum of Precompetitive Collaboration in Oncology Research
(2010), Direct to Consumer Genetic Testing (with the NRC, 2010), Policy Issues in Nanotechnology
and Oncology (2011), National Cancer Policy Summit: Opportunities and Challenges in Cancer
Research and Care (2011), Patient-Centered Cancer Treatment Planning: Improving the Quality of
Oncology 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 and
Challenges 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 Cancer
Clinical Trials System for the 21st Century (2010), Evolution of Translational Omics (2012), Ensuring
the Quality of Cancer Care in an Aging Population (In preparation)
Membership of the Forum:
Chair - John Mendelsohn, MD, President, MD Anderson Cancer Center
Vice Chair - Patricia Ganz, MD, Professor of Medicine, Jonsson Comprehensive Cancer Center
Amy Abernethy, MD, Director, Duke University School of Medicine Cancer Care Research Program
Rafael Amado, MD, Senior Vice President and Head of GlaxoSmithKline Oncology R&D
Fred Appelbaum, MD, Director, Clinical Research, Fred Hutchinson Cancer Research Center
Peter Bach, MD, MAPP, Member, Memorial Sloan-Kettering Cancer Center
Edward Benz, MD, President, Dana-Farber Cancer Institute
Monica Bertagnolli, MD, Professor of Surgery, Harvard University Medical School
Otis Brawley, MD, Chief Medical Officer and Executive Vice President, American Cancer Society
Michael Caligiuri, MD, Director, Ohio State University Cancer Center, past President, AACI
Renzo Canetta, MD, Vice President, Oncology Global Clinical Research, Bristol-Myers Squibb
Michaele Chamblee Christian, MD, Retired
William Dalton, PhD, MD, CEO, M2Gen, H. Lee Moffitt Cancer Center
Wendy Demark-Wahnefried, PhD, RD, Professor and Chair, University of Alabama, Birmingham
Robert Erwin, MS, President, Marti Nelson Cancer Foundation
Roy Herbst, MD, PhD, Chief of Medical Oncology, Yale Cancer Center
Thomas Kean, MPH, President and CEO, C-Change
Douglas Lowy, MD, Deputy Director, Division of Basic Science, NCI
Daniel R. Masys, MD, Affiliate Professor, Biomedical Informatics, University of Washington
Martin Murphy, PhD, DMedSc, Chief Executive Officer, CEO Roundtable on Cancer
Brenda Nevidjon, RN, MSN, Professor, Duke University School of Nursing, past President, ONS
Steven Piantadosi, MD, PhD, Director, Samuel Oschin Comprehensive Cancer Institute
Lisa Richardson, MD, MPH, Associate Director, Division of Cancer Prevention and Control, CDC
Debasish Roychowdhury, MD, Senior Vice President- Global Oncology, Sanofi
Ya-Chen Tina Shih, PhD, Director, Program in the Economics of Cancer, University of Chicago
Ellen Sigal, PhD, Chairperson and Founder, Friends of Cancer Research
Steven Stein, MD, Senior Vice President, US Clinical Development & Medical Affairs, Novartis
John Wagner, MD, PhD, Vice President, Clinical Pharmacology, Merck Research Laboratories
Ralph Weichselbaum, MD, Chairman, Radiation and Cellular Oncology, University of Chicago
Janet Woodcock, MD, Director, Center for Drug Evaluation and Research, FDA
Forum Director: Sharyl Nass, PhD
8
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, 2012
Myles Axton, Ph.D.
Myles Axton is the 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, and between 1990 and 1995 did postdoctoral research at Dundee and at MIT’s
Whitehead Institute. Myles’s 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 biochemists, zoologists and medical students from primary research papers.
Helping to establish Oxford’s innovative research MSc. in Integrative Biosciences led Myles 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.
After spending 15 years as a faculty member at the University of Pennsylvania, in the Center
for Clinical Epidemiology and Biostatistics, under the direction of Dr. Brian Strom, Dr. Berlin left
the University of Pennsylvania to join Janssen Research & Development, where he is currently
Vice President of Epidemiology. He has authored or coauthored over 230 publications in a wide
variety of clinical and methodological areas, including papers on the study of meta-analytic
methods as applied to both randomized trials and epidemiology. He served on an Institute of
Medicine Committee that developed recently-released recommendations for the use of
systematic reviews in clinical effectiveness research, and currently serves on the Scientific
Advisory Committee to the Observational Medical Outcomes Partnership, a public-private
partnership 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 Translational
Sciences, 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 nephrology at the Mount Sinai School
of Medicine, followed by a fellowship at Yale, then work as a research scientist at the
Physiology 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 Medicine and
professorships in the Division of Nephrology, Department of Internal Medicine, and the
Department of Physiology. She joined the National Institutes of Health (NIH) in 1997 as director
of the Division of Kidney, Urologic, and Hematologic Diseases at the National Institute of
Diabetes and Digestive and Kidney Diseases. In 2006, Dr. Briggs accepted a position as senior
1
29. scientific officer at the Howard Hughes Medical Institute. In January 2008, she returned to NIH
as the Director of the National Center for Complementary and Alternative Medicine.
Dr. Briggs has published more than 175 research articles, book chapter, and scholarly
publications and 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 senior most
governing board at the NIH.
Robert M. Califf, M.D.
Dr. Califf is the Vice Chancellor for Clinical and Translational 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, NC. He leads a multifaceted organization that
seeks 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 nation's premier academic research organizations. He is editor-in-chief of the
American Heart Journal, the oldest cardiovascular specialty journal, and a practicing cardiologist
at Duke University Medical Center.
Born in 1951 in Anderson, SC, Dr. Califf attended high school in Columbia, where he was a
member of the 1969 AAAA SC Championship basketball team. After high school, Dr. Califf
attended Duke University, graduating summa cum laude and Phi Beta Kappa in 1973. He
remained at Duke for medical school, where he was selected for the Alpha Omega Alpha
medical honor society. After graduating from Duke University School of Medicine in 1978, he
completed 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 a
Master of the American College of Cardiology in 2006. An international leader in the fields of
cardiovascular medicine, healthcare 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 contributing editor for TheHeart.org, an online
information resource for healthcare professionals working in the field of cardiovascular
medicine.
As founder and for a decade 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 1000 employees and an annual budget of over $1 00 million; 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 foundations, and the
medical products industry to conduct innovative research spanning multiple therapeutic arenas
and scientific disciplines.
2
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 Forum
Dr. Califf serves as co-chair of the first Principal Investigators Steering Committee of the CTSA
and has served on the FDA's Cardiorenal Advisory Panel, and on the Institute of Medicine's
(IOM) Pharmaceutical Roundtable, Committee on Identifying and Preventing Medication Errors,
and Committee on Nutritional Biomarkers. In 2008, he was part of the subcommittee of the
FDA's Science Board that recommended sweeping reform ofthe agency's science base. He was
also a member of the IOM committees that recommended Medicare coverage of clinical trials
and the removal of ephedra from the market. Dr. Califf is currently a member ofthe IOM Forum
in Drug Discovery, Development, and Translation and a member of the National Advisory
Council on Aging.
Reflecting his interests in healthcare quality, Dr. Califf was the founding director of the
coordinating center of the Centers for Education & 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 system. 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.
Dr. Califf is married to Lydia Carpenter, and the couple has three children-Sharon Califf, a
graduate of Elon College; Sam, a graduate student at the University of Colorado-Boulder; and
Tom, a graduate of Duke-and one grandchild. Dr. Califf enjoys time with his family, works at his
golf game, listens to music, and remains an ardent supporter of the Duke men's and women's
basketball programs.
Michael N. Cantor, M.D., M.A., FACP
Michael Cantor is currently Senior Director, Information Strategy and Analytics, in Pfizer’s
Clinical Informatics and Innovation group. His work focuses on leveraging data reuse and
integration to support future horizons of scientific decision support for precision medicine. He is
currently co-leading 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 co-leads the MEDIC (Multisite Electronic Data Infectious Disease Consortium)
project, 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 healthcare IT in advancing their strategic priorities.
Dr. Cantor previously lead 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. Outside of Pfizer, he has been a member of AMIA’s public policy committee
for six years, and led the committee’s initiative to update its positions around data stewardship
and reuse.
Prior to joining Pfizer, Michael was the Chief Medical Information Officer for the South
Manhattan Healthcare Network of the New York City Health and Hospitals Corporation, based
at Bellevue Hospital in Manhattan. His work there focused on developing the network’s EMR
system to improve patient safety and on using the network’s clinical data warehouse for
research. He continues to see patients 1 day/week at Bellevue, and is a Clinical Assistant
Professor of Medicine at NYU School of Medicine.
3
31. Michael completed his residency in internal medicine and informatics training at Columbia, has
an 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 recently
the Director of the Office of Biorepositories and Biospecimen Research (OBBR) and the
Executive Director of the Cancer Human Biobank (caHUB) project at the National Cancer
Institute. In these capacities, she had leadership responsibility for strategic initiatives that
included the Innovative Molecular Analysis Technologies for Cancer program, the Biospecimen
Research Network program, and the NCI Community Cancer Centers project. She is an adjunct
Professor 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 Harvard
Graduate School of Arts and Sciences. She trained in Pathology at Harvard's Brigham and
Women's Hospital and is boarded in both Anatomic 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 2000-2005.
Prior to this, she had been a Professor of Pathology 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 research 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 professional organizations. She is a Fellow of the College of American
Pathologists and a Fellow of the Royal Society of Medicine. Currently, she is the Chair of the
American Joint Committee on Cancer (AJCC), serves on the Executive Committee of the
Commission on Cancer of the American College of Surgeons, and serves as the Pathology
Section Editor for Cancer. She is a past Chair of the Cancer Committee of the College of
American 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 awards
are the ISBER Award for Outstanding Achievement in Biobanking, the 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, 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 Oracle's solution groups, strategic planning, product
development, and sales, service, and support for the industry solutions sold into the healthcare
and life sciences markets worldwide. Mr. de Crescenzo brings more than 20 years of
operational and IT leadership across healthcare and life sciences to his work with customers
and partners worldwide.
Prior to joining Oracle, Mr. de Crescenzo held a number of leadership positions during his
decade at IBM Corporation, working with healthcare and life sciences clients worldwide. Prior to
entering the information technology industry, he held leadership positions in healthcare
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.
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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 Forum
Mr. de Crescenzo has been a keynote speaker at numerous industry conferences worldwide
and is quoted frequently on industry issues. In 2005, he was named one of the "Top 25 Most
Influential Consultants" by Consulting Magazine.
Mr. de Crescenzo has a BA in political science from Yale University and an MBA in high
technology from Northeastern University.
Peter Doshi, Ph.D.
Dr. Doshi is a postdoctoral fellow in comparative effectiveness research at the Johns Hopkins
University School of Medicine.
His overarching research interests are in improving the bases for credible evidence 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, their review
evaluates regulatory information including clinical 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.
An associate professor in the School of Medicine's Department of Bioethics and Humanities, Dr.
Edwards also is a core faculty member for the UW Institute for Public Health Genetics. She
received both her Master of Arts degree 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
Environmental Health Sciences. She also is a co-director of the Regulatory Support and
Bioethics Core for the Institute for Translational Health Sciences (ITHS), a partnership of the
UW, Fred Hutchinson Cancer Research Center, Seattle Children's and other regional
institutions 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 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 that
promote 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 of
events 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 health
advocacy 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
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33. American Communities?" and "Public Commentary 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 School of Medicine's Continuous Professional Improvement
Committee and is a former member of Medicine's Standing Committee on Issues of Women
Faculty, the Student Progress Committee and the Committee on Research and Graduate
Education. She is a current member of the UW Graduate School Committee on Interdisciplinary
Education.
Hans-Georg Eichler, M.D., M.Sc.
Dr. Eichler is the Senior Medical Officer at the European Medicines Agency in London, United
Kingdom, where he is responsible for coordinating activities between the Agency’s scientific
committees and giving advice on scientific and public health issues. From January until
December 2011, Dr. Eichler was the Robert E. Wilhelm fellow at the Massachusetts Institute of
Technology’s Center for International Studies, participating in a joint research project under the
MIT’s NEWDIGS initiative. He divided his time between the MIT and the EMA in London.
Prior to joining the European Medicines Agency, Dr. Eichler was at the Medical University of
Vienna in Austria for 15 years. He was vice-rector for Research and International Relations
since 2003, and professor and chair of the Department of Clinical Pharmacology since 1992.
His other previous positions include president of the Vienna School of Clinical Research and co-
chair of the Committee on Reimbursement of Drugs of the Austrian Social Security Association.
His industry experience includes time spent at Ciba-Geigy Research Labs, U.K., and Outcomes
Research at Merck & Co., in New Jersey.
Dr. Eichler graduated with an M.D. from Vienna University Medical School and a Master of
Science degree in Toxicology from the University of Surrey in Guildford, U.K. He trained in
internal medicine and clinical pharmacology at the Vienna University Hospital as well as at
Stanford University.
Jennifer S. Geetter, J.D.
Ms. Geetter is a partner in the law firm of McDermott Will & Emery LLP and is based in the
Firm's Washington, D.C., office. She focuses her practice on emerging biotechnology and
safety issues, advising 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 biological
samples and data warehousing. She also assists health care clients in implementing research
strategies, structuring research operational and compliance infrastructure, and in developing
guidelines for the appropriate relationships between providers and industry. She is a frequent
speaker on these topics.
Ms. Geetter a member of the Firm’s Life Sciences Affinity Group and Personalized Medicine
Team. She is also a co-chair of the Pro Bono and Community Service Committee for the
Washington, D.C., office. She sits on McDermott’s National Pro Bono Committee and the
American Health Lawyers Association Life Sciences Section Steering Committee.
Ms. Geetter is listed as a leading individual in health care in Washington, D.C., in Chambers
USA 2008: America’s Leading Lawyers for Business.
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