Pre-Approval Access: Can Compassion, Business, and Medicine Coexist?

Pre-Approval Access: Can Compassion, Business, and Medicine Coexist?

Wednesday, October 28, 2015 - Thursday, October 29, 2015

The New York Academy of Sciences

Presented By

Presented by the NYU School of Medicine and 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

  • Johnson&Johnson

Presented by

  • NYU School of Medicine

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
Ellis Rubinstein, The New York Academy of Sciences
Paul Stoffels, MD, Johnson & Johnson

8:30 AM

Setting the Stage: Current Challenges and Complexities of Compassionate Use
Arthur Caplan, PhD, NYU School of Medicine

Session I. Patients' Perspectives: Compassionate Use and Expanded Access

9:00 AM

Panel Discussion

Moderator:

Arthur Caplan, PhD, NYU School of Medicine

Panelists:

Marc Boutin, JD, National Health Council
Pat Furlong, Parent Project Muscular Dystrophy
Danielle Leach, MPA, St. Baldrick's Foundation
Musa Mayer, MS, MFA, AdvancedBC.org; National Breast Cancer Coalition
Steven T. Walker, MS, PG, Abigail Alliance

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

Travel & Lodging

Our Location

The New York Academy of Sciences

7 World Trade Center
250 Greenwich Street, 40th floor
New York, NY 10007-2157
212.298.8600

Directions to the Academy

Hotels Near 7 World Trade Center

Recommended partner hotel

Club Quarters, World Trade Center
140 Washington Street
New York, NY 10006
Phone: 212.577.1133

The New York Academy of Sciences is a member of the Club Quarters network, which offers significant savings on hotel reservations to member organizations. Located opposite Memorial Plaza on the south side of the World Trade Center, Club Quarters, World Trade Center is just a short walk to the Academy.

Use Club Quarters Reservation Password NYAS to reserve your discounted accommodations online.

Other nearby hotels

Conrad New York

212.945.0100

Millenium Hilton

212.693.2001

Marriott Financial Center

212.385.4900

Club Quarters, Wall Street

212.269.6400

Eurostars Wall Street Hotel

212.742.0003

Gild Hall, Financial District

212.232.7700

Wall Street Inn

212.747.1500

Ritz-Carlton New York, Battery Park

212.344.0800

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.