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Appendix A
Workshop Agenda
Envisioning a Transformed Clinical Trials Enterprise in the
United States: Establishing an Agenda for 2020
November 7-8, 2011
20 F Street NW Conference Center
20 F Street NW
Washington, DC 20001
Background:
There is increasing recognition that the clinical trials enterprise in the
United States faces substantial challenges impeding the efficient and effec-
tive conduct of clinical and translational research needed to support the
development of breakthrough medicines. A gap exists between the desired
state where medical care in the United States is provided solely based on
high-quality evidence and the reality of our limited ability to generate
timely and practicable evidence. Eighty-five percent of clinical decisions
in the United States are not supported by high-quality evidence. At the
same time, U.S. clinical trials that generate medical evidence are becom -
ing increasingly costly while experiencing greater setbacks. In addition,
the shifting “footprint” of clinical trials toward sites outside the United
States prompts questions about the generalizability and applicability of
the results of those clinical trials to the U.S. population and represents a
competitive challenge for the United States.
85
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86 ENVISIONING A TRANSFORMED CLINICAL TRIALS ENTERPRISE
The limited ability of the nation’s clinical trials system to support drug
development and evaluation exists within a broader context of a need for
a “learning health system,” where “knowledge generation is so embedded
into the core of the practice of medicine that it is a natural outgrowth
and product of the health care delivery process and leads to continual
improvement in care.” An essential component of such a learning health
system is a robust and well-working clinical trials enterprise to support
drug development; inform quality improvement; and support surveil-
lance, international, and comparative effectiveness research.
The IOM’s Forum on Drug Discovery, Development, and Translation has
established an initiative to address challenges facing the U.S. clinical trials
enterprise and to engage stakeholders in an open discussion of potentially
transformative strategies to improve the efficiency and effectiveness of
clinical trials.
Workshop Objectives:
• Frame the problem and discuss a vision for a clinical trials enter-
prise that is efficient and effective and fully integrated into the
health delivery system of 2020. Define how the envisioned clini -
cal trials enterprise differs from the current system and suggest
approaches to transform our current system into a learning system.
• Consider the following core themes in framing an agenda to effect
transformation of the U.S. clinical trials enterprise:
— Providing a vision for a clinical trials enterprise in the health
care system of 2020.
— Developing a robust clinical trials workforce.
— Aligning cultural and financial incentives.
— Building an infrastructure to support a transformed clinical trials
enterprise.
• Workshop presentations and panel discussions will be supported
and supplemented by discussion papers prepared by participants
in Forum activities. Each of the four workshop sessions will be
prefaced by presentations from discussion paper authors.
NOVEMBER 7, 2011
8:30 a.m. Breakfast available
9:00 a.m. Welcome and Introductions
Jeffrey Drazen, Workshop Chair
Editor-in-Chief
New England Journal of Medicine
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87
APPENDIX A
SESSION I:
FRAMING THE NEED FOR CHANGE:
ENVISIONING A CLINICAL TRIALS ENTERPRISE
IN THE HEALTH CARE SYSTEM OF 2020
Session Objectives:
• Set a framework within which to establish a vision for 2020 that
addresses the reinvention of the clinical trials enterprise around
partnership with the institutional health care delivery system of
2020.
• Given the anticipated changes in the structure of the U.S. health
care system, consider how an efficient, effective clinical trials enter-
prise can be integrated into the health care delivery system of 2020.
alastair J.J. WooD, Session Chair
Partner & Managing Director
Symphony Capital
9:10 a.m. Presentation of Discussion Paper: The Clinical Trials
Enterprise in the United States: A Call for Disruptive
Innovation
robert Califf
Director, Duke Translational Medicine Institute
Professor of Medicine
Vice Chancellor for Clinical and Translational Research
Duke University Medical Center
Gary filerman
President
Atlas Health Foundation
riCharD murray
Vice President, Global Center for Scientific Affairs
Office of the Chief Medical Officer
Merck & Co., Inc.
10:00 a.m. Panel Discussion with Speakers: A Framework for the
Clinical Trials Enterprise in the Health Care System of 2020
Objectives:
• uggest and discuss the key components of a transformed
S
clinical trials enterprise incorporated into the integrated
health delivery system of 2020.
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88 ENVISIONING A TRANSFORMED CLINICAL TRIALS ENTERPRISE
• iscuss the research outputs needed from the clinical
D
trials enterprise to drive improved patient care and feed
into a learning health system.
• onsider broad transformational changes including the
C
changing shape/structure of the pharmaceutical industry
and the increasing globalization in clinical trials and drug
development.
alastair J.J. WooD, Panel Moderator
Partner & Managing Director
Symphony Capital
neil Weissman
President
MedStar Health Research Institute
ihor rak
Vice President, Clinical Neuroscience
AstraZeneca
10:45 a.m. BREAK
11:00 a.m. Keynote Address: The Learning Health System
riCharD Platt
Professor, Harvard Medical School
Co-Chair, Clinical Effectiveness Research Innovation
Collaboration
IOM Roundtable on Value and Science-Driven Health Care
11:30 a.m. Discussion with Keynote Speaker, Panelists, and
Workshop Participants:
• ow can the national clinical trials enterprise fit into the
H
envisioned learning health system?
• hat infrastructure is needed to embed randomized con-
W
trolled trials in a learning health system?
• hat are the key barriers impeding integration of clini-
W
cal trials into a learning health system (e.g., financial,
cultural, regulatory)?
• here are the opportunities to address these barriers
W
and build the needed infrastructure? What stakeholders/
organizations/sectors should take the lead and how can
their efforts best be coordinated?
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89
APPENDIX A
Peter yu, Moderator
Oncologist, Palo Alto Medical Foundation
Chair, Electronic Health Record Working Group
American Society of Clinical Oncology
bryan luCe
Senior Vice President, Science Policy
United BioSource Corporation
12:15 p.m. LUNCH
SESSION II:
DEVELOPING A ROBUST CLINICAL TRIALS WORKFORCE
Session Objectives:
• efine a clinical trials “workforce” and identify workforce needs
D
that will be necessary to support clinical trials.
• iven the defined workforce, discuss the needs for career paths
G
and career development opportunities.
sherine Gabriel, Session Chair
William J. and Charles H. Mayo Professor of Medicine &
Epidemiology Mayo Medical School
Co-Principal Investigator and Director of Education,
Mayo Clinical Center for Translational Scientific
Activities (CTSA)
1:15 p.m. Presentation of Discussion Paper: Developing a Robust
Clinical Trials Workforce
robert Califf
Director, Duke Translational Medicine Institute
Professor of Medicine
Vice Chancellor for Clinical and Translational Research
Duke University Medical Center
elaine Gallin
Principal
QE Philanthropic Advisors
miChael lauer
Director, Division of Cardiovascular Sciences
National Heart, Lung, and Blood Institute
National Institutes of Health
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90 ENVISIONING A TRANSFORMED CLINICAL TRIALS ENTERPRISE
2:15 p.m. Panel Discussion with Speakers: Developing and
Sustaining a Clinical Trials Workforce
Objectives:
• ist key skills, techniques, and areas of expertise needed
L
by the workforce.
• hat are the benchmarks and metrics of success for an
W
effective clinical trials workforce?
• ropose and discuss the core competencies of a clinical
P
trials workforce.
sherine Gabriel, Panel Moderator
William J. and Charles H. Mayo Professor of Medicine &
Epidemiology Mayo Medical School
Co-Principal Investigator and Director of Education,
Mayo Clinical Center for Translational Scientific
Activities (CTSA)
briGGs W. morrison
Senior Vice President, Worldwide Medical Excellence
Pfizer Inc.
rebeCCa JaCkson
Professor and Associate Dean for Clinical Research
Director and Principal Investigator,
OSU Center for Clinical and Translational Science
The Ohio State University
3:00 p.m. BREAK
3:15 p.m. Keynote Address: Sustaining Institutional Support and
Patient Engagement in Clinical Trials: Models and Mes-
sages from the NIH Clinical Center
John Gallin
Director
National Institutes of Health Clinical Center
3:45 p.m. Discussion with Keynote Speaker, Panelists, and
Workshop Participants:
• hat are the needs for patient and broader public engage-
W
ment in clinical trials?
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91
APPENDIX A
• iscuss broad as well as disease-specific efforts to part-
D
ner with patients in the development and conduct of
clinical trials. How might these partnerships help create
a national culture of clinical research in the United States?
• ow can models and best practices from the NIH Clinical
H
Center be adapted to the realities of other institutions and
community systems to advance and promote the conduct
of clinical trials in those systems?
Janet tobias, Moderator
Co-founder and Partner, Ikana Health
Adjunct Assistant Professor,
Mount Sinai School of Medicine
annetine GeliJns
Professor of Health Policy
Co-Chair, Department of Health Evidence and Policy
Mount Sinai School of Medicine
heather snyDer
Senior Associate Director, Scientific Grants
Medical and Scientific Relations
Alzheimer’s Association
4:30 p.m. ADJOURN
Envisioning a Transformed Clinical Trials Enterprise in the
United States: Establishing an Agenda for 2020
NOVEMBER 8, 2011
8:30 a.m. Breakfast available
9:00 a.m. Welcome and Introductions
Jeffrey Drazen, Workshop Chair
Editor-in-Chief
New England Journal of Medicine
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92 ENVISIONING A TRANSFORMED CLINICAL TRIALS ENTERPRISE
SESSION III:
ALIGNING CULTURAL AND FINANCIAL INCENTIVES
Session Objective:
• onsider institutional needs to support clinical trials including
C
addressing cultural issues and aligning financial incentives to sup-
port and sustain an efficient clinical trials enterprise.
arthur h. rubenstein, Session Chair
Professor, Department of Medicine,
Division of Endocrinology
Raymond and Ruth Perelman School of Medicine
University of Pennsylvania
9:10 a.m. Presentation of Discussion Paper: Transforming the
Economics of Clinical Trials
JuDith kramer
Associate Professor of Medicine,
Duke University Medical Center
Executive Director,
Clinical Trials Transformation Initiative (CTTI)
kevin sChulman
Professor of Medicine and Business Administration
Duke University School of Medicine and
The Fuqua School of Business
9:50 a.m. Panel Discussion with Speakers: Aligning Cultural and
Financial Incentives to Support Clinical Trials
Objectives:
• iscuss incentives, reward structures, and business
D
models impacting the conduct of clinical trials, and the
key needs and challenges for advancing and improving
upon these models.
• onsider regulatory, financial, and cultural changes that
C
are needed to improve the environment for clinical trials.
arthur h. rubenstein, Panel Moderator
Professor, Department of Medicine,
Division of Endocrinology
Raymond and Ruth Perelman School of Medicine
University of Pennsylvania
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93
APPENDIX A
riCharD ruDiCk
Hazel Prior Hostetler Chair of Neurology,
Cleveland Clinic
Professor of Medicine,
Cleveland Clinic Lerner College of Medicine
Case Western Reserve University
ChristoPher bearDmore
Chief Executive Officer
Translational Research Management
sCott steele
Director, Research Alliances
Adjunct Associate Professor,
Community and Preventive Medicine
University of Rochester
10:35 a.m. BREAK
SESSION IV:
BUILDING AN INFRASTRUCTURE TO SUPPORT A
TRANSFORMED CLINICAL TRIALS ENTERPRISE
Session Objectives:
• et out an organizational framework for an infrastructure to con-
S
duct clinical trials.
• iscuss:
D
— organizational frameworks for the infrastructure (i.e., network
models or other approaches);
— informatics needs;
— regulatory reform or harmonization both within the United States
and internationally.
ClyDe yanCy, Session Chair
Magerstadt Professor of Medicine
Chief, Division of Cardiology
Feinberg School of Medicine
Northwestern University
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94 ENVISIONING A TRANSFORMED CLINICAL TRIALS ENTERPRISE
10:55 a.m. Presentation of Discussion Paper: Developing a Clinical
Trials Infrastructure
Petra kaufmann
Director, Office of Clinical Research
National Institute of Neurological Disorders and Stroke
National Institutes of Health
Paul eisenberG
Senior Vice President
Global Regulatory Affairs and Safety
Amgen Inc.
Janet WooDCoCk
Director, Center for Drug Evaluation and Research
U.S. Food and Drug Administration
11:45 a.m. Panel Discussion with Speakers: Establishing an
Infrastructure to Support a Transformed Clinical Trials
Enterprise
Objectives:
• onsider the types of trials that should be done in the
C
United States and what the site/locus for the conduct
of these trials should be within the infrastructure model
proposed.
• hat are the roles and responsibilities of the various par-
W
ticipants and stakeholders in the proposed infrastructure
of the clinical trials enterprise?
ClyDe yanCy, Panel Moderator
Magerstadt Professor of Medicine
Chief, Division of Cardiology
Feinberg School of Medicine
Northwestern University
louis fiore
Principal Investigator, VA Cooperative Studies Program
Director, VA Cooperative Studies Program Coordinating
Center, Boston
Department of Veterans Affairs
miChael kinG Jolly
Senior Vice President
Quintiles Innovation
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95
APPENDIX A
karen marGolis
Professor of Medicine,
University of Minnesota Medical School
Director of Clinical Research
HealthPartners Research Foundation
12:30 p.m. LUNCH
SESSION V:
DEVELOPING AN AGENDA FOR THE CREATION OF A
TRANSFORMED CLINICAL TRIALS ENTERPRISE
Session Objectives:
• efine the key steps and responsible parties needed to achieve the
D
elements of a transformed clinical trials enterprise discussed dur-
ing the workshop.
• onsider the prioritization of transforming different elements of
C
the clinical trials enterprise—what is feasible and most desirable?
• onsider any unintended consequences of transforming the clini-
C
cal trials enterprise.
• uggest a transformative path forward for decision makers and
S
those implementing the work of clinical trials across the enterprise.
1:30 p.m. Jeffrey Drazen, Session Chair
Editor-in-Chief
New England Journal of Medicine
Presentation of Key Themes/Suggested Paths from Session
Chairs
alastair J.J. WooD, Session I Chair
Partner & Managing Director
Symphony Capital
sherine Gabriel, Session II Chair
William J. and Charles H. Mayo Professor of Medicine
& Epidemiology Mayo Medical School
Co-Principal Investigator and Director of Education,
Mayo Clinical Center for Translational Scientific
Activities (CTSA)
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96 ENVISIONING A TRANSFORMED CLINICAL TRIALS ENTERPRISE
arthur h. rubenstein, Session III Chair
Professor, Department of Medicine,
Division of Endocrinology
Raymond and Ruth Perelman School of Medicine
University of Pennsylvania
ClyDe yanCy, Session IV Chair
Magerstadt Professor of Medicine
Chief, Division of Cardiology
Feinberg School of Medicine
Northwestern University
2:30 p.m. Reflecting on Potential Paths Forward: Setting an Agenda
for a Transformed Clinical Trials Enterprise
Jean rouleau
Scientific Director
Institute for Circulatory and Respiratory Health
Canadian Institutes of Health Research (CIHR)
Jeffrey Drazen, Panel Moderator
Editor-in-Chief
New England Journal of Medicine
DouGlas CroPPer
President and Chief Executive Officer
Genesis Health System
lynn ethereDGe
Co-Founder
Health Insurance Reform Project and Rapid Learning Project
George Washington University
ronalD krall
Associate Fellow
University of Pennsylvania, Center for Bioethics
James DoroshoW
Director, Division of Cancer Treatment and Diagnosis
National Cancer Institute, National Institutes of Health
4:30 p.m. ADJOURN
