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

A presentation at The New York Academy of Sciences

On October 28–29, 2015, the New York Academy of Sciences and NYU School of Medicine convened a multidisciplinary conference on pre-approval access to medicines outside a clinical trial, or what is often referred to as compassionate use. The process by which critically ill patients may request access to experimental therapies prior to regulatory approval poses complex ethical, legal, and social challenges that often leave patient advocacy groups, physicians, pharmaceutical companies, regulators, and patients at odds in the struggle to determine how far an individual can go to try to save his or her own life, and at what—and whose—cost.

The 2-day conference consisted of panel discussions designed to probe different aspects of compassionate use. Stakeholders came together to put the system under the microscope, examining how heightened public awareness of compassionate use, advances in drug development, the advent of social media, and the costliness of safely bringing a drug to market affect decisions about whether—and how—to provide experimental therapies on a compassionate-use basis.

Through spirited debates and networking opportunities, conference attendees worked to lay the foundation for better, more collaborative decision making regarding compassionate use policies and programs, and to answer the title question—Can Compassion, Business, and Medicine Coexist?

Tanya Scharton-Kersten was a featured panelist.