Appendix A
Agenda

Objectives: The broad objectives of this 1-day workshop will be to:

  • Outline changes in the translational research (discovery through Phase II) process that have taken place over the past 10 years and why.

  • Discuss new models for funding translational research and new technologies and consider their impact on the process.

  • Examine regulatory, legislative, and institutional policy tools currently in place to help advance therapeutic development for rare or neglected diseases and individualized therapies, and discuss whether these tools are adequate or whether new ones are needed in light of these new funding models.

8:30

Welcome, Background, and Introduction of Workshop Objectives

 

NANCY SUNG (Workshop Chair, Drug Forum Member)

Senior Program Officer

Burroughs Wellcome Fund

8:45

Keynote: Changes in the Translational Research (Discovery– Phase II) Process

  • What are the scientific and regulatory elements/requirements of the translational process?



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Appendix A Agenda Objectives: The broad objectives of this 1-day workshop will be to: • Outline changes in the translational research (discovery through Phase II) process that have taken place over the past 10 years and why. • Discuss new models for funding translational research and new technologies and consider their impact on the process. • Examine regulatory, legislative, and institutional policy tools cur- rently in place to help advance therapeutic development for rare or neglected diseases and individualized therapies, and discuss whether these tools are adequate or whether new ones are needed in light of these new funding models. 8:30 Welcome, Background, and Introduction of Workshop Objectives Nancy Sung (Workshop Chair, Drug Forum Member) Senior Program Officer Burroughs Wellcome Fund 8:45 Keynote: Changes in the Translational Research (Discovery– Phase II) Process • What are the scientific and regulatory elements/requirements of the translational process? 0

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0 APPENDIX A • Are the current approaches for fulfilling these requirements ade- quate to advance development of drugs for rare or neglected dis- eases and individualized therapies? • Where along the continuum is investment needed? Innovative Safe New Drugs—Financial Sectoring Tom Caskey (Drug Forum Member) Chief Operating Officer and Director/Chief Executive Officer University of Texas Health Science Center at Houston q&A 9:05 Session 1: Diverse Funding Organizations—Business Cases • Why were new approaches or models needed, and what are their advantages? • Are these models effective at helping to speed development and how might their impact be measured? • Are these viable models for advancing products in the context of individualized therapies? • Are federal regulatory policies adequate for helping speed develop- ment and approval of drugs for rare diseases? Moderator: Timothy Coetzee Executive Director Fast Forward, LLC 9:10 OneWorld Health: A Not-for-Profit Pharmaceutical Company Victoria Hale Founder and Chair of the Board of Directors Institute for OneWorld Health 9:25 Cystic Fibrosis Foundation Therapeutics’ Pipeline Approach to CFTR Drug Discovery and Development Diana Wetmore Vice President of Alliance Management Cystic Fibrosis Foundation Therapeutics

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0 BREAKTHROUGH BUSINESS MODELS 9:40 Surviving as a For-Profit Company in the Rare Disease World David Meeker President, Lysosomal Storage Disorder Therapeutics Genzyme 9:55 New Business Models Addressing Global Health: A Framework for Private Equity Peter Corr (Drug Forum Member) Co-founder and General Partner Celtic Therapeutics 10:10 Reaction Panel Gail Cassell (Drug Forum Co-chair) Vice President, Scientific Affairs, and Distinguished Lilly Research Scholar for Infectious Diseases Eli Lilly and Company Marlene Haffner (Drug Forum Member) Executive Director, Global Regulatory Intelligence and Policy Amgen Mark Batshaw Chief Academic Officer Children’s National Medical Center Chaitan Khosla Professor, Departments of Chemistry, Chemical Engineering, and Biochemistry Stanford University Doug Onsi Venture Partner HealthCare Ventures q&A 11:15 Break

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0 APPENDIX A 11:30 Session 2: Strategies for Facilitating Sharing of Research Materials and Data • How have funding organizations controlled data and materials to facilitate open access and sharing? • Are open-access databases and repositories making a difference? How might their use be broadened? • What are the opportunities and challenges of managing these resources? • Are federal regulatory policies adequate for helping speed develop- ment and approval of drugs for rare diseases? Moderator: Margaret Anderson Chief Operating Officer FasterCures 11:35 Agreements for Research and Materials Sharing Michael Mowatt Director, Office of Technology Development National Institute of Allergy and Infectious Diseases, NIH 11:50 The Alzheimer’s Disease Neuroimaging Initiative (ADNI): A Public–Private Partnership Laurie Ryan Program Director, Alzheimer’s Disease Clinical Trials Division of Neuroscience, National Institute on Aging, NIH 12:05 Genetic Alliance BioBank or Herding the Cats Sharon Terry President and Chief Executive Officer Genetic Alliance q&A 12:30 Lunch Break

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0 BREAKTHROUGH BUSINESS MODELS 12:50 Luncheon Keynote The FDA Orphan Drug Program: A Proven Model Tim Coté Director, Office of Orphan Products Development U.S. Food and Drug Administration 1:30 Break 1:45 Session 3: Strategies for Navigating Intellectual Property • Are existing policies adequate to facilitate efficient management of intellectual property throughout the development process? • Which novel strategies have been implemented in recent years to manage intellectual property in light of new funding models? Moderator: Anthony So Professor of the Practice of Public Policy Studies Director, Program in Global Health and Technology Access Terry Sanford Institute of Public Policy, Duke University 1:45 Creating an Enabling Intellectual Property Environment for Neglected and Rare Diseases Anthony So 2:05 Innovation in Alliances and Licensing: Transforming Now for the Future Craig Sorensen Senior Director, Strategic Research Alliances Vertex Pharmaceuticals Incorporated 2:25 Accelerating Intellectual Property Sharing to Facilitate Translation Rusty Bromley Chief Operating Officer Myelin Repair Foundation

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 APPENDIX A 2:45 uC Berkeley’s Approach to Intellectual Property Management: Multiple Strategies Are Required to Deploy Innovations for Maximal Impact Carol Mimura Assistant Vice Chancellor for Intellectual Property and Industry Research Alliances (IPIRA) University of California at Berkeley q&A 3:45 Break 4:00 Session 4: Strategies for Facilitating Clinical Trials • What do patient groups and disease organizations need to know about working with FDA toward approval of new therapies? • How are patient groups and disease foundations helping to facili- tate trial launch? • Are there innovative methods for conducting efficient multicenter clinical trials with small numbers of patients? Moderator: Stephen Groft (Drug Forum Member) Director, Office of Rare Disease Research National Institutes of Health 4:05 FDA Review and Regulation of Small Clinical Trials: Successes, Barriers, and Directions for the Future Anne Pariser Medical Team Leader, Inborn Errors of Metabolism Team Division of Gastroenterology Products Center for Drug Evaluation and Research U.S. Food and Drug Administration 4:20 Accelerating Clinical Trials: The MMRF and MMRC Model Anne Quinn Young Program Director Multiple Myeloma Research Foundation

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 BREAKTHROUGH BUSINESS MODELS 4:35 Maximizing your Assets in Clinical Trials: Economies of Scale and Standardization Sharon Hesterlee Vice President, Translational Research Muscular Dystrophy Foundation q&A 5:15 Session 5: Recap of Key Points Made Throughout the Day Enriqueta Bond President Burroughs Wellcome Fund 5:30 Adjourn