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Conference to Explore Compassionate Use of Non-FDA Approved Medications

Experts from multiple fields will come together in New York to discuss the challenges of providing pre-approval access to investigational medications for patients in life-threatening situations.

Published October 21, 2015

NEW YORK, October 21, 2015 - On October 28-29 the conference Pre-Approval Access: Can Compassion, Business, and Medicine Coexist? will address a critical and timely issue: challenges surrounding pre-approval access to investigational medicines. The event, presented by NYU School of Medicine and the New York Academy of Sciences, and supported by Johnson & Johnson, will feature discussions with prominent representatives of governments, pharmaceutical companies, patient groups, NGOs, and foundations.

 "Imagine that someone you love was a matter of days from succumbing to a fatal infection, but the drug of last resort - and your only hope-had not yet emerged from the trials and approvals needed to reach the marketplace. I don't think there's much disagreement about what the right thing to do is. The important question is how do we as a society accommodate such 'compassionate use' cases?" says Fred Guterl, Executive Editor, Scientific American, who will moderate a panel on "New Directions for Pre-Approval Access."

 "I'm thrilled to be participating in this critical and timely dialogue, because the FDA's drug approval system is broken, and the conversation is no longer theoretical. Real lives are at stake. That's why 24 states and counting are now protecting patients' 'Right to Try' with investigational medicines that have passed basic FDA safety testing, but have not yet received full FDA approval," says conference speaker Christina Sandefur, Vice President for Policy, Goldwater Institute, who will participate in a panel discussion titled "Can Legislative or Regulatory Changes Ameliorate the Challenges of Compassionate Use and Expanded Access?"

"This isn't about questioning the FDA's role in ensuring drug safety, but the current options for expanded access don't make sense for our friends and loved ones who are facing imminent death," adds Sandefur.

 Strong viewpoints, which range from total support for compassionate use to those not in favor of it, are a hallmark of this issue-the entire spectrum of which will be represented at the conference. As with any controversial issue, a scientific approach demands that all aspects of the issue be identified and considered.

 In addition to discussions of the ethical, logistical, and safety aspects of pre-approval access, speakers will discuss specific, recent cases, including the successful social media advocacy campaign for Josh Hardy, for which Brooke Gladstone, WNYC Studios; On the Media, will moderate a panel discussion.  

Linda Mobula, MD, MPH, Deputy Team Leader, West Africa Ebola Disaster Assistance Response Team, USAID, will discuss another recent, high-profile case involving pre-approval access:

"In July 2014, under compassionate care, Dr. Kent Brantly and Nancy Writebol were administered ZMapp, an experimental drug for the treatment of Ebola virus disease. This likely contributed to their full recovery, enabling them to both see their families again. With over 11,000 Ebola related deaths and extremely high case fatality rates in West Africa, it is paramount that we discuss the administration of experimental therapies under compassionate care to Ebola patients, especially if this can improve their chances of survival," says Mobula.

"It is crucial to bring together a plurality of voices from the United States and around the world if we are to take advantage of the opportunity to achieve policy reform in this area that is so important to patients and families," says scientific conference organizer and opening speaker Arthur Caplan, PhD, Founding Director, Division of Medical Ethics at NYU School of Medicine.

For more information about the event, including a full agenda and speaker bios, please visit

 For press inquiries, including press passes to the event, please contact Diana Friedman (; 212-298-8645).


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