Pre-Approval Access: Can Compassion, Business, and Medicine Coexist?
Wednesday, October 28, 2015 - Thursday, October 29, 2015
The New York Academy of Sciences
Patients who have a serious or life threatening condition for which no satisfactory alternative therapy exists and who cannot enter a clinical trial may, under certain circumstances, gain access to new investigational medicines through what is known as "pre-approval access" or "compassionate use." The prospective benefits of non-regulatory approved medicines may outweigh the potential risks for many critically ill patients. However, a number of ethical and safety concerns have been raised related to ensuring that unapproved treatments are equitably distributed via systematic approaches, that society as a whole is best served through scientific studies on the risks and benefits of new medicines, and that individual patients are protected from undue harm.
To address these important issues, the NYU School of Medicine and the New York Academy of Sciences will present a two-day colloquium, Pre-Approval Access: Can Compassion, Business, and Medicine Coexist? International stakeholders — including representatives of patient-advocacy organizations, regulatory agencies, pharmaceutical companies, foundations, NGOs, and government — will come together to explore the ethical, legal, and social challenges surrounding compassionate use. This meeting aims to galvanize discussion through a series of debates and will open with a lecture by Arthur Caplan, PhD, Founding Director, Division of Medical Ethics at NYU School of Medicine.
*Reception to follow.
Livestream
The colloquium will be livestreamed during the two days of the colloquium. Registration for each day is separate, and if you would like register for Day 1 of the livestream, please click "Register for the 10/28/15 Livestream," if you would like to register for Day 2 of the livestream, please click "Register for the 10/29/15 Livestream," and if you would like to register for both days of the livestream, please register for each of the two days separately.
Registration Pricing
This event is free, but registration is required. To attend, please click on the "Register Now" button in the grey box at the top of this page. Your registration will be complete upon receipt of a confirmation email. If you do not receive a confirmation, please contact customerservice@nyas.org for assistance. Although on-site registration may be possible on the day of the event, pre-registration is highly encouraged due to space limitations.
This symposium is made possible with support from
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Agenda
* Presentation titles and times are subject to change.
The program is not certified for continuing education.
Day 1: Wednesday, October 28, 2015 | |||||||
7:00 AM | Registration and Breakfast | ||||||
8:00 AM | Welcome Remarks | ||||||
8:30 AM | Setting the Stage: Current Challenges and Complexities of Compassionate Use | ||||||
Session I. Patients' Perspectives: Compassionate Use and Expanded Access | |||||||
9:00 AM | Panel Discussion
| Marc Boutin, JD, National Health Council | |||||
10:30 AM
Networking Coffee Break
Session II. Case Study: Josh Hardy and the Impact of Social Media
11:00 AM
Panel Discussion
Moderator: | Brooke Gladstone, WNYC Studios; On the Media | |
Panelists: |
Debra Birnkrant, MD, U.S. Food and Drug Administration
Nancy Goodman, JD, Kids v Cancer
Kenneth I. Moch, MBA, Chimerix (2009-2014); Euclidean Life Science Advisors
Richard Plotkin, JD, The Max Cure Foundation
Meg Tirrell, MSJ, NBC Universal, CNBC
12:30 PM
Networking Lunch
Session III. Compassionate Use during a Public Health Crisis: Lessons Learned from the Ebola Epidemic
1:45 PM
Panel Discussion
Moderator: | Arthur Caplan, PhD, NYU School of Medicine | |
Panelists: |
Marion Danis, MD, U.S. National Institutes of Health
Linda M. Mobula, MD, MPH, Johns Hopkins University
Piero Olliaro, MD, PhD, World Health Organization
Annette Rid, MD, PhD, King's College, London
3:15 PM
Networking Coffee Break
Session IV. Stakeholder Perspectives: Key Issues When Considering a Compassionate Use Request
3:45 PM
Panel Discussion
Moderator: | Nancy Neveloff Dubler, LLB, New York City Health and Hospitals Corporation; NYU School of Medicine | |
Panelists: |
Richard Klein, U.S. Food and Drug Administration
Ronald Krall, MD, University of Pittsburgh
Sandy Macrae, MD, PhD, Takeda Pharmaceuticals
Jane Reese-Coulbourne, MS, ChE, Reagan-Udall Foundation for the U.S. Food and Drug Administration
Steve Usdin, BioCentury
4:45 PM
Closing Remarks
Jennifer E. Miller, PhD, NYU School of Medicine
Melanie Brickman Stynes, MSc, PhD, The New York Academy of Sciences
5:00 PM
Networking Reception
7:00 PM
Day 1 Adjourns
Day 2: Thursday, October 29, 2015
7:00 AM
Continental Breakfast
8:00 AM
Welcome Remarks
Future Vision and New Directions
Joanne Waldstreicher, MD, Johnson & Johnson
Session V. Can Legislative or Regulatory Changes Ameliorate the Challenges of Compassionate Use and Expanded Access?
8:30 AM
Panel Discussion
Moderator: | Alison Bateman-House, MA, MPH, PhD, NYU School of Medicine | |
Panelists: |
Peter C. Adamson, MD, The Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania
Fritz Bittenbender, MPA, Biotechnology Industry Organization
The Honorable Linda Rosenthal, New York State Assembly
Christina Sandefur, JD, Goldwater Institute
Carla Mann Woods, Mann Healthcare Partners
10:00 AM
Networking Coffee Break
Session VI. International Perspectives: How do Compassionate Use and Expanded Access Policies Differ Globally?
10:30 AM
Panel Discussion
Moderator: | Arthur Caplan, PhD, NYU School of Medicine | ||
Panelists: |
Rob Camp, EUPATI; EURORDIS
Marcela Simões, MBA, Interfarma
Barbara von Tigerstrom, MA, LLB, PhD, University of Saskatchewan
Tom Watson, Idis Managed Access, Part of the Clinigen Group
Session VII. New Directions for "Pre-Approval" Access
11:30 PM
Panel Discussion
Moderator: | Fred Guterl, Scientific American | |
Panelists: |
Martha Donoghue, MD, U.S. Food and Drug Administration
Matthew Hepburn, MD, Defense Advanced Research Projects Agency
Amrit Ray, MD, Janssen Pharmaceutical Companies of Johnson & Johnson
Tanya Scharton-Kersten, PhD, International AIDS Vaccine Initiative (IAVI)
David I. Scheer, MS, Scheer and Company, Inc.
12:30 PM
Networking Lunch
Session VIII. Future Vision: Ethics and Policy Lens
1:30 PM
Panel Discussion
Moderator: | Alison Bateman-House, MA, MPH, PhD, NYU School of Medicine | |
Panelists: |
Daniel McIntyre, MPA, Biogen
Jerry Menikoff, MD, JD, U.S. Department of Health and Human Services
Franklin Miller, PhD, Weill Cornell Medical College
Nancy Neveloff Dubler, LLB, New York City Health and Hospitals Corporation; NYU School of Medicine
Beth Roxland, JD, MBioethics, Johnson & Johnson; NYU School of Medicine
Luann Van Campen, MA, MS, PhD, Eli Lilly and Company
3:00 PM
Closing Remarks
Valerie Cwik, MD, Muscular Dystrophy Association
Melanie Brickman Stynes, MSc, PhD, The New York Academy of Sciences
3:15 PM
Colloquium Concludes
Speakers
Scientific Organizing Committee
Alison Bateman-House, PhD, MPH, MA
NYU School of Medicine
Melanie Brickman Stynes, MSc, PhD
The New York Academy of Sciences
Arthur Caplan, PhD
NYU School of Medicine
website
Brooke Grindlinger, PhD
The New York Academy of Sciences
Erick T. Tatro, PhD
The New York Academy of Sciences
Speakers
Peter C. Adamson, MD
The Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania
website
Debra Birnkrant, MD
U.S. Food and Drug Administration
website
Fritz Bittenbender, MPA
Biotechnology Industry Organization
Marc Boutin, JD
National Health Council
website
Rob Camp
European Organization for Rare Diseases
website | website
Marion Danis, MD
U.S. National Institutes of Health
Martha Donoghue, MD
U.S. Food and Drug Administration
website
Pat Furlong
Parent Project Muscular Dystrophy
website
Brooke Gladstone
WNYC Studios; On the Media
website
Nancy Goodman, JD
Kids v Cancer
website
Fred Guterl
Scientific American
website
Matthew Hepburn, MD
Defense Advanced Research Projects Agency
Richard Klein
U.S. Food and Drug Administration
website
Ronald Krall, MD
University of Pittsburgh
Danielle Leach, MPA
St. Baldrick's Foundation
website
Sandy Macrae, MD, PhD
Takeda Pharmaceuticals
Carla Mann Woods
Mann Healthcare Partners
Musa Mayer, MS, MFA
AdvancedBC.org; National Breast Cancer Coalition
website
Daniel McIntyre, MPA
Biogen
website
Jerry Menikoff, MD, JD
U.S. Department of Health and Human Services
Franklin Miller, PhD
Weill Cornell Medical College
Jennifer E. Miller, PhD
NYU School of Medicine
website
Linda M. Mobula, MD, MPH
Johns Hopkins University
Kenneth I. Moch, MBA
Chimerix (2009-2014); Euclidean Life Science Advisors
Nancy Neveloff Dubler, LLB
New York City Health and Hospitals Corporation; NYU School of Medicine
Piero Olliaro, MD, PhD
World Health Organization
Richard Plotkin, JD
The Max Cure Foundation, Inc.
website
Amrit Ray, MD
Janssen Pharmaceutical Companies of Johnson & Johnson
website
Jane Reese-Coulbourne, MS, ChE
Reagan-Udall Foundation for the U.S. FDA
website
Annette Rid, MD
King's College, London
website
The Honorable Linda Rosenthal
New York State Assembly
Beth Roxland, JD, MBioethics
Johnson & Johnson; NYU School of Medicine
Christina Sandefur, JD
Goldwater Institute
website
David I. Scheer, MS
Scheer and Company, Inc.
Tanya Scharton-Kersten, PhD
International AIDS Vaccine Initiative (IAVI)
website
Marcela Simões, MBA
Interfarma
website
Paul Stoffels, MD
Johnson & Johnson
Steve Usdin
BioCentury
website
Luann Van Campen, MA, MS, PhD
Eli Lilly and Company
website
Barbara von Tigerstrom, MA, LLB, PhD
University of Saskatchewan
Joanne Waldstreicher, MD
Johnson & Johnson
Steven T. Walker, MS, PG
Abigail Alliance
website
Tom Watson
Idis Managed Access, Part of the Clinigen Group
website
Sponsors
This symposium is made possible with support from
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Special Needs
The New York Academy of Sciences complies with the public accommodation requirements of the Americans with Disabilities Act and the rules and regulations thereof. Please let us know if you require any assistance during the meeting. For any additional information and about special needs accommodations available to colloquium attendees, please e-mail jcurtin@nyas.org or call Jaclyn Curtin 212.298.8694.