Appendix A

Workshop Agenda

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



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Appendix A Workshop Agenda Maximizing the Goals of the Cures Acceleration Network to Accelerate the Development of New Drugs and Diagnostics: An Institute of Medicine Workshop June 45, 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 discover- ing 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 73

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74 MAXIMIZING THE IMPACT OF THE CURES ACCELERATION NETWORK 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 estab- lished 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 refer- ence existing efforts in which such authority is currently applied across other federal agencies (e.g., DARPA, DTRA, BARDA). Explore promising models for publicprivate collaborations that could be strengthened or facilitated by activities under CAN. -- Discuss barriers to such collaborations and identify opportu- nities 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.

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APPENDIX A 75 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

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76 MAXIMIZING THE IMPACT OF THE CURES ACCELERATION NETWORK 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: Relationship of CAN to NCATS, other NIH Institutes/ Centers Identification of types of activities that are likely to be undertaken under CAN Discussion of role of CAN Board 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

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APPENDIX A 77 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: What are the translational science needs? What approaches have failed with existing authorities? What can we learn from these failures? What approaches/efforts could potentially benefit from new CAN authorities? 12:05 p.m. LUNCH

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78 MAXIMIZING THE IMPACT OF THE CURES ACCELERATION NETWORK 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

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APPENDIX A 79 Michael Gutch Managing Director MedImmune Ventures Issue for Discussion: What are the necessary conditions for the matching program to encourage and successfully de-risk investment decisions on part of matching funders? 2:00 p.m. Discussion with Speakers and Audience Issues for Discussion: What are the lessons learned from previous experiences? What are the respective roles of the various sectors (e.g., biopharma/biotech, venture capital/private equity, and patient/disease advocacy)? What are models for publicprivate collaborations that could be strengthened or facilitated by the matching authority? What are the barriers and opportunities and potential solutions for moving past those barriers? 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

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80 MAXIMIZING THE IMPACT OF THE CURES ACCELERATION NETWORK 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 AgencyEnergy (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: How can the other transaction authority (OTA) be most effectively applied in the biomedical/life sciences space?

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APPENDIX A 81 W hat kind of science/projects should be funded under exercise of OTA? What are the attributes of a promising project or science? How should the research needs be defined and executed? What are potential barriers that could impede the successful exercise of the OTA by NCATS/CAN? How can these barriers be overcome? What are the conditions for success? What are the respective roles of the various sectors (e.g., biopharma/biotech, venture capital/private equity, and patient/disease advocacy)? What are models for publicprivate collaborations that could be strengthened or facilitated by the flexible research authority? What are the barriers and opportunities and potential solutions for moving past those barriers? 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

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82 MAXIMIZING THE IMPACT OF THE CURES ACCELERATION NETWORK 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 publicprivate 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 8:35 a.m.FDA Presentations: Intersection of CAN with FDA Regulatory Science Initiatives and Activities Jesse Goodman Chief Scientist Food and Drug Administration ShaAvhre Buckman-Garner Director, Office of Translational Sciences Center for Drug Evaluation and Research Food and Drug Administration 9:05 a.m.Roundtable Discussion: Identification and Discussion of Regulatory Science Priorities That Are Important for Drug Development Panelists ShaAvhre Buckman-Garner Director, Office of Translational Sciences Center for Drug Evaluation and Research Food and Drug Administration

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APPENDIX A 83 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

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84 MAXIMIZING THE IMPACT OF THE CURES ACCELERATION NETWORK 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: Coordination of efforts across funding agencies/sources (e.g., HHS, DoD, NGOs, industry) and with other established partners in translational research activities (e.g., FNIH, Reagan-Udall Foundation, C-Path), and reduction of duplication. Implications of CAN for academic translational science-- career paths, funding opportunities, etc. 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

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APPENDIX A 85 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

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86 MAXIMIZING THE IMPACT OF THE CURES ACCELERATION NETWORK 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