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March 8, 2016

The Ethical Challenges of Compassionate Use

Author Affiliations
  • 1Division of Medical Ethics, NYU Langone Medical Center, New York, New York
  • 2Janssen Research & Development LLC, Titusville, New Jersey
JAMA. 2016;315(10):979-980. doi:10.1001/jama.2016.0416

Granting access to drugs, vaccines, biologics, and devices that have not yet been approved by governmental regulatory authorities is a growing challenge for physicians, public officials, patient advocacy groups, institutional review boards (IRBs), and patients.1 Although the issue of rapid access to investigational agents is not new, tracing back to the early days of the human immunodeficiency virus pandemic, the pace of requests has increased. This is attributable to many factors, including greater awareness of compassionate use on the part of patients and their physicians; more information available through the Internet and websites describing clinical trials; an increase in promising interventions, including genetic markers, immunotherapies, and recombinant vaccines; threats from potential epidemics such as Ebola, cholera, and influenza; and an increased willingness to try novel agents by patients who are chronically ill or dying.

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