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The IOM Forum on Drug Discovery, Development, and Translation is hosting a workshop on
Breakthrough Business Models:
Drug Development for Rare and Neglected Diseases and Individualized Therapies
June 23, 2008
NAS Keck Center, Room 100
500 5th St NW, Washington, DC 20001
AGENDA
8:30 AM Welcome, Background, and Introduction of Workshop Objectives
NANCY SUNG (Workshop Chair, Drug Forum Member)
Senior Program Officer
Burroughs-Wellcome Fund
AUDIO | SLIDES
8:45 AM Keynote – Changes in the translational research (discovery – phase II) process
- What are the scientific and regulatory elements/requirements of the translational process?
- Are the current approaches for fulfilling these requirements adequate to advance development of 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/CEO
University of Texas HSC at Houston
AUDIO | SLIDES
Q&A
9:05 AM Session 1 – Diverse Funding Organizations - Business Cases
- Are 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 an era of market fragmentation?
- Are federal regulatory policies, like the Orphan Drug Act and its antecedents, adequate for helping speed development and approval of drugs for rare diseases?
Moderator: TIMOTHY COETZEE
Executive Director
Fast Forward, LLC
AUDIO | (NO SLIDES)
9:10 AM OneWorld Health—A Not-for-profit Pharmaceutical Company10:10 AM Reaction Panel
VICTORIA HALE
Founder & Chair of the Board of Directors
Institute for OneWorld Health
AUDIO | SLIDES
9:25 AM Cystic Fibrosis Foundation Therapeutics’ Pipeline Approach to CFTR Drug Discovery and Development
DIANA WETMORE
Vice President of Alliance Management
Cystic Fibrosis Foundation Therapeutics
AUDIO | SLIDES
9:40 AM Surviving as a For-Profit Company in the Rare Disease World
DAVID MEEKER
President, Lysosomal Storage Disorder Therapeutics
Genzyme
AUDIO | SLIDES
9:55 AM New Business Models Addressing Global Health - A Framework for Private Equity
PETER CORR (Drug Forum Member)
Co-Founder and General Partner
Celtic Therapeutics
AUDIO | SLIDES (coming soon)
10:10 AM Reaction Panel
AUDIO | (NO SLIDES)
GAIL CASSELL (Drug Forum Co-chair)
Vice President, Scientific Affairs and Distinguished Lilly Research Scholar
for Infectious Diseases
Eli Lilly and Company
MARLENE HAFFNER
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
AUDIO | (NO SLIDES)
11:15 AM Break
11:30 AM Session 2 – Strategies for Facilitating Sharing of Research Materials and Data
- How can funding organizations corner the market on data to facilitate open access and sharing?
- Are open access databases making a difference? How might their use be broadened?
- What are the opportunities and challenges of managing these resources?
- Are federal regulatory policies, like the Orphan Drug Act and its antecedents, adequate for helping speed development and approval of drugs for rare diseases?
Moderator: MARGARET ANDERSON
Chief Operating Officer
FasterCures
AUDIO | (NO SLIDES)
11:35 AM Agreements for Research and Materials SharingQ&A
MICHAEL MOWATT
Director, Office of Technology Development
National Institute of Allergy and Infectious Diseases, NIH
AUDIO | SLIDES
11:50 AM 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
AUDIO | SLIDES
12:05 PM Genetic Alliance BioBank or Herding the Cats
SHARON TERRY
President and CEO
Genetic Alliance
AUDIO | SLIDES
Q&A
AUDIO | (NO SLIDES)
12:30 PM Lunch Break
12:50 PM Luncheon Keynote
TIM COTÉ
Director, Office of Orphan Products Development
U.S. Food and Drug Administration
AUDIO | SLIDES
1:30 PM Break
1:45 PM Session 3 – Strategies for Navigating Intellectual Property
- Are existing IP policies adequate to facilitate efficient management of IP throughout the development process when alternative funding models get involved in the process?
- Are there examples of novel strategies that have implemented in recent years to manage IP in light of new funding models?
- Are federal regulatory policies, like the Orphan Drug Act and its antecedents, adequate for helping speed development and approval of drugs for rare diseases?
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 PM Creating an Enabling IP Environment for Neglected and Rare Diseases
ANTHONY SO
AUDIO | SLIDES
2:05 PM Innovation in Alliances and Licensing: Transforming now for the future
CRAIG SORENSEN
Sr. Director, Strategic Research Alliances
Vertex Pharmaceuticals Incorporated
AUDIO | SLIDES
2:25 PM Accelerating Intellectual Property Sharing to Facilitate Translation
RUSTY BROMLEY
Chief Operating Officer
Myelin Repair Foundation
AUDIO | SLIDES
2:45 PM U.C. Berkeley's Approach to IP Management: Multiple Strategies are Required to Deploy Innovations for Maximal Impact
CAROL MIMURA
Assistant Vice Chancellor for Intellectual Property & Industry
Research Alliances (IPIRA)
University of California, Berkeley
AUDIO | SLIDES
Q&A
AUDIO | (NO SLIDES)
3:45 PM Break
4:00 PM Session 4 – Strategies for Facilitating Clinical Trials
- How are patient groups / disease foundations helping to facilitate trial launch?
- Are there innovative methods for conducting multi-center clinical trials with small numbers of patients?
Moderator: STEPHEN GROFT
Director, Office of Rare Disease Research
National Institutes of Health
AUDIO | (NO SLIDES)
4:05 PM 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
AUDIO | SLIDES
4:20 PM Accelerating Clinical Trials: The MMRF and MMRC Model
ANNE QUINN YOUNG
Program Director
Multiple Myeloma Research Foundation
AUDIO | SLIDES (coming soon)
4:35 PM Maximizing Your Assets in Clinical Trials: Economies of Scale and Standardization
SHARON HESTERLEE
Vice President Translational Research
Muscular Dystrophy Foundation
AUDIO | SLIDES
Q&A
AUDIO | (NO SLIDES)
5:15 PM Session 5 – Recap of key points made throughout the day
ENRIQUETA BOND
Burroughs Wellcome Fund
AUDIO | (NO SLIDES)
5:30 PM Adjourn
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