Maximizing the Goals of the Cures Acceleration Network to Accelerate the Development of New Drugs and Diagnostics: An Institute of Medicine Workshop
June 4—5, 2012
National Academy of Sciences
Keck Building, Room 100
500 Fifth Street, NW
Washington, DC 20001
Background and Meeting Objectives:
Recent years have seen both extraordinary opportunity and complex challenges in pharmaceutical innovation. New biomedical technology platforms are creating novel avenues for research and new opportunities for the discovery and clinical development of innovative diagnostics and therapies. Yet despite these advances, the pathway from basic science to new therapeutics faces challenges on many fronts. The translational divide results in only a small fraction of investigational new drugs reaching FDA approval and the patients who need them. New paradigms for discovering and developing drugs are being sought to bridge the ever-widening gap between scientific discoveries and translation of those discoveries into life-changing medications. New collaborative approaches within the federal agencies, academia, and industry are directing focused attention
on the advancement of the drug development enterprise. Among these initiatives is the Cures Acceleration Network (CAN), which was originally authorized in the Patient Protection and Affordable Care Act (P.L. 111-148) and was subsequently amended by the Consolidated Appropriations Act, FY 2012 (P.L. 112-74), which moved CAN to the newly authorized National Center for Advancing Translational Sciences (NCATS).
This public workshop will consider options and opportunities to maximize the usefulness and impact of the CAN program in order to advance translational sciences. In addition to providing suggestions to NCATS, the workshop is, in part, in response to congressional interest in CAN expressed in the FY 2012 appropriations act conference report. The workshop will inform NIH/NCATS in its efforts to implement CAN and advance translational sciences, and will also inform the public, policy community, and other stakeholders as all of these parties continue to work to enhance the development and testing of therapies and diagnostics to patients. The summary will be provided to NCATS and the newly established CAN Board to help it identify ways to accelerate and expand the number of cures.
The workshop objectives are to:
- Identify and catalog potential tools, methods, and approaches that hold promise for accelerating translational science.
— Consideration of such promising approaches will draw from the experiences of existing activities at other federal agencies related to the goals of CAN (e.g., FDA, CDC, AHRQ).
- Discuss the authorities conferred to CAN and identify strategies for effectively using those authorities.
— Consideration of the CAN authorities will specifically explore the flexible research, or “other transactions,” authority and will reference existing efforts in which such authority is currently applied across other federal agencies (e.g., DARPA, DTRA, BARDA).
- Explore promising models for public—private collaborations that could be strengthened or facilitated by activities under CAN.
— Discuss barriers to such collaborations and identify opportunities and potential solutions for moving past the identified barriers.
— Discuss the respective roles of multiple sectors, including, e.g., biopharma, biotech, venture capital/private equity, and patient/disease advocacy.
- Identify barriers and potential solutions to facilitate coordination of activities under CAN with the FDA regulatory review process and timelines.
JUNE 4, 2012
DAY ONE
8:30 a.m. |
Opening Remarks |
Workshop Co-Chairs | |
CAROLYN COMPTON | |
President and Chief Executive Officer | |
Critical Path Institute | |
LOUIS DEGENNARO | |
Executive Vice President and Chief Mission Officer | |
The Leukemia & Lymphoma Society |
Session I: Overview of NIH Translational Sciences and the Cures Acceleration Network
Session Objectives:
- Provide an overview of the translational science initiatives at NIH.
- Provide an overview and description of the Cures Acceleration Network program goals and authorities.
8:40 a.m. | Background and Session Objectives |
SUDIP PARIKH, Session Chair | |
Vice President, Health Policy | |
Managing Director | |
Centers for Public Health Research and Evaluation | |
Battelle Health and Life Sciences | |
8:45 a.m. | Keynote Address: Introduction to NCATS and Overview of Translational Science Initiatives and CAN |
TOM INSEL | |
Acting Director | |
National Center for Advancing Translational Sciences | |
National Institutes of Health | |
9:05 a.m. | Plenary Discussion: Introduction to CAN Authorities |
LILI PORTILLA | |
Director, Office of Strategic Alliances | |
National Center for Advancing Translational Sciences | |
National Institutes of Health | |
BARBARA MCGAREY | |
Deputy Associate General Counsel for Public Health | |
Office of the General Counsel | |
National Institutes of Health | |
KATHY HUDSON | |
Acting Deputy Director, NCATS | |
Deputy Director for Science, Outreach, and Policy, | |
NIH Office of the Director | |
National Institutes of Health | |
9:25 a.m. | Discussion with Speakers and Audience: |
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Session II: Approaches to Accelerating Translational Science
Session Objectives:
- Through discussion of case examples and other mechanisms, identify potential approaches that hold promise for accelerating translational science, highlighting approaches that could potentially benefit from the new CAN authorities.
- Discuss and identify attributes of success stories and failures.
9:45 a.m. | Background and Session Objectives |
BILL CHIN, Session Chair | |
Executive Dean for Research | |
Harvard Medical School | |
9:50 a.m. | Opening Plenary: Challenges and Needs in Translational Science—Industry Perspective |
JOSHUA BOGER | |
Founder, Vertex Pharmaceuticals | |
10:10 a.m. | Opening Plenary: Challenges and Needs in Translational Science—Academic Perspective |
R. SANDERS WILLIAMS | |
President | |
The Gladstone Institutes | |
University of California, San Francisco | |
10:30 a.m. | BREAK |
10:45 a.m. | Brief Presentations: Product Development/Commercialization Efforts |
JAMES BRADNER | |
Assistant Professor, Harvard Medical School | |
Instructor in Medicine, Dana-Farber Cancer Institute | |
STEPHEN SEILER | |
Founder and Chief Executive Officer | |
AesRx | |
LOUIS DEGENNARO | |
Executive Vice President and Chief Mission Officer | |
The Leukemia & Lymphoma Society | |
11:25 a.m. | Discussion with Speakers and Audience: |
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12:05 p.m. | LUNCH |
Session III: Application of Matching Authority
Session Objectives:
- Explore existing efforts in which the matching authority, or similar match-type requirement, is currently applied across other federal and state agencies.
- Examine benefits and advances that have been achieved through use of this authority.
- Discuss how barriers to application and use of those authorities have been overcome.
12:35 p.m. | Background and Session Objectives |
NANCY SUNG | |
Senior Program Officer | |
Burroughs Wellcome Fund | |
12:40 p.m. | Series of Brief Presentations: Application of “Matching” or Similar Authority |
MICHAEL WEINGARTEN | |
Director, SBIR Development Center | |
National Cancer Institute | |
National Institutes of Health | |
KRISTEN DOYLE | |
General Counsel | |
Cancer Prevention and Research Institute of Texas | |
ELLEN FEIGAL | |
Senior Vice President, Research & Development | |
California Institute for Regenerative Medicine | |
1:25 p.m. | Panel Discussion with “Matchers” |
JENS ECKSTEIN | |
President, SR One | |
GlaxoSmithKline | |
MARTIN LEHR | |
Osage University Partners | |
MICHAEL GUTCH | |
Managing Director | |
MedImmune Ventures | |
Issue for Discussion: | |
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2:00 p.m. | Discussion with Speakers and Audience |
Issues for Discussion: | |
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2:30 p.m. | BREAK |
Session IV: Application of Flexible Research Authority
Session Objectives:
- Explore existing efforts in which the flexible research (or similar) authority is currently applied across other federal agencies.
- Examine benefits and advances that have been achieved through use of these authorities.
- Discuss how barriers to application and use of those authorities have been overcome.
2:50 p.m. | Background and Session Objectives |
WILLIAM WARREN, Session Chair | |
Vice President, VaxDesign Campus | |
Sanofi Pasteur | |
2:55 p.m. | Series of Presentations: Agencies |
SCOTT ULREY | |
Deputy Director, Contracts Management Office | |
Defense Advanced Research Projects Agency | |
Department of Defense | |
JASON PARAGAS | |
Special Assistant to the Director | |
Defense Threat Reduction Agency | |
Department of Defense | |
GERALD KOVACS | |
Director, Division of Chemical, Biological, Radiological, | |
and Nuclear Countermeasures | |
Biomedical Advanced Research and Development Authority | |
Office of the Assistant Secretary for Preparedness and | |
Response | |
PEDER MAARBJERG | |
Assistant Director for External Coordination | |
Advanced Research Projects Agency—Energy (ARPA-E) | |
Department of Energy | |
4:15 p.m. | Panel Discussion with Speakers, Discussants, and Audience: Comparing Flexible Research Authority to Existing NIH Authorities |
Discussants: | |
DAN WATTENDORF | |
Program Manager, Defense Sciences Office | |
Defense Advanced Research Projects Agency | |
ROBI BLUMENSTEIN | |
President | |
CHDI Management | |
Issues for Discussion: | |
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5:30 p.m. | Adjourn Day One |
Maximizing the Goals of the Cures Acceleration Network to Accelerate the Development of New Drugs and Diagnostics
JUNE 5, 2012
DAY TWO
8:20 a.m. | Welcome and Introductions |
Workshop Co-Chairs | |
CAROLYN COMPTON | |
President and Chief Executive Officer | |
Critical Path Institute | |
LOUIS DEGENNARO | |
Executive Vice President and Chief Mission Officer The Leukemia & Lymphoma Society |
Session V: Situating CAN Within the Drug Development Ecosystem
Session Objectives:
- Identify potential approaches to facilitate coordination of activities under CAN with FDA regulatory science initiatives and activities.
- Discuss existing activities in multiple sectors and address ways to maximize CAN impact in the drug development ecosystem.
- Explore promising models for public—private collaborations that could be strengthened or facilitated by activities under CAN. Discuss barriers to such collaborations and identify opportunities and potential solutions for moving past the identified barriers.
8:30 a.m. | Background and Session Objectives |
MARGARET ANDERSON, Session Chair | |
Executive Director FasterCures |
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8:35 a.m. | FDA Presentations: Intersection of CAN with FDA Regulatory Science Initiatives and Activities |
JESSE GOODMAN | |
Chief Scientist Food and Drug Administration |
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SHAAVHRéE BUCKMAN-GARNER | |
Director, Office of Translational Sciences Center for Drug Evaluation and Research Food and Drug Administration |
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9:05 a.m. | Roundtable Discussion: Identification and Discussion of Regulatory Science Priorities That Are Important for Drug Development |
Panelists | |
SHAAVHRéE BUCKMAN-GARNER | |
Director, Office of Translational Sciences Center for Drug Evaluation and Research Food and Drug Administration |
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ELIZABETH MANSFIELD | |
Director, Personalized Medicine, Office of In Vitro | |
Diagnostic Device Evaluation and Safety | |
Center for Devices and Radiological Health | |
Food and Drug Administration | |
THOMAS KALIL | |
Deputy Director for Policy | |
Office of Science and Technology Policy | |
Executive Office of the President | |
CAROLYN COMPTON | |
President and Chief Executive Officer | |
Critical Path Institute | |
GARRY NEIL | |
Corporate Vice President, Science and Technology | |
Johnson & Johnson | |
Panel Moderator: | |
MARGARET ANDERSON | |
Executive Director | |
FasterCures | |
9:45 a.m. | Roundtable Discussion: Role of CAN in Advancing Cross-Sector and Other Collaborative Translational Science Activities |
Panelists | |
ELLEN SIGAL | |
Founder and Chair | |
Friends of Cancer Research | |
DAVID WHOLLEY | |
Director, Biomarkers Consortium | |
Foundation for the NIH | |
JANE REESE-COULBOURNE | |
Executive Director | |
Reagan-Udall Foundation for the FDA | |
FREDA LEWIS-HALL | |
Chief Medical Officer | |
Pfizer Inc. | |
DOUG THROCKMORTON | |
Deputy Director | |
Center for Drug Evaluation and Research | |
Panel Moderator: | |
MYRL WEINBERG | |
President | |
National Health Council | |
Issues for Discussion: | |
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10:30 a.m. | BREAK |
Session VI: Concluding Panel Discussion: Principles for Deployment of CAN Authorities
Session Objectives:
- Discuss key themes from the workshop.
- Based on workshop presentations and discussions, identify principles for deployment of CAN authorities.
10:45 a.m. | Closing Discussion with Panelists and Audience: Led by Workshop Co-Chair(s) |
CAROLYN COMPTON | |
President and Chief Executive Officer | |
Critical Path Institute | |
LOUIS DEGENNARO | |
Executive Vice President and Chief Mission Officer | |
The Leukemia & Lymphoma Society | |
10:50 a.m. | Presentation of Key Themes/Suggested Paths from Workshop Session Chairs |
SUDIP PARIKH, Session I Chair | |
Vice President, Health Policy | |
Battelle Memorial Institute | |
BILL CHIN, Session II Chair | |
Executive Dean for Research | |
Harvard Medical School | |
NANCY SUNG, Session III Chair | |
Senior Program Officer | |
Burroughs Wellcome Fund | |
DAN WATTENDORF, Session IV Rapporteur | |
Program Manager, Defense Sciences Office | |
Defense Advanced Research Projects Agency | |
MARGARET ANDERSON, Session V Chair | |
Executive Director | |
FasterCures | |
11:40 a.m. | Reflecting on Potential Approaches to Maximize the Goals of CAN: Panel Discussion with Session Chairs, Panelists, and Audience |
Discussants | |
JOSHUA BOGER | |
Founder, Vertex Pharmaceuticals | |
KATHY HUDSON | |
Acting Deputy Director, NCATS | |
Deputy Director for Science, Outreach, and Policy, | |
NIH Office of the Director | |
National Institutes of Health | |
CAROL MIMURA | |
Assistant Vice Chancellor for Intellectual Property and | |
Industry Research Alliances | |
University of California, Berkeley | |
ROBERT O’NEILL | |
Senior Statistical Advisor | |
Center for Drug Evaluation and Research | |
Food and Drug Administration | |
12:20 p.m. | Closing Observations from NCATS |
KATHY HUDSON | |
Acting Deputy Director, NCATS | |
Deputy Director for Science, Outreach, and Policy, | |
NIH Office of the Director | |
National Institutes of Health | |
12:30 p.m. | ADJOURN |