Wizemann, Theresa, Robinson, Sally, Giffin, Robert. "Appendix A: Agenda." Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies: Workshop Summary. Washington, DC: The National Academies Press, 2008.
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Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies - Workshop Summary
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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Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies - Workshop Summary
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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Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies - Workshop Summary
Are the current approaches for fulfilling these requirements adequate to advance development of drugs for rare or neglected diseases 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 development 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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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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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 development 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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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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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 facilitate 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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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
