Should patients in need be given access to experimental drugs?

Expert Opin Pharmacother. 2015 Jun;16(9):1275-9. doi: 10.1517/14656566.2015.1046837.


Patient access to experimental drugs outside of clinical trials is called compassionate use or expanded access. Compassionate use/expanded access presents a powerful ethical dilemma in that it involves competing claims that both have moral weight: specifically, an individual patient's very understandable desire to try to extend his or her life versus the orderly and efficient functioning of a drug development and clinical trial system that benefits much larger numbers of patients. Patient advocates, the FDA, pharmaceutical trade groups, and state and national legislators in the US are all currently weighing in on patient access to experimental drugs, and new guidelines and rules are being introduced. In this editorial, we discuss the impulse to rescue individual patients facing dire diseases and underscore the ethical questions that such rescue efforts raise.

Keywords: compassionate use; expanded access; experimental drugs; investigational products; right to try.

Publication types

  • Editorial

MeSH terms

  • Compassionate Use Trials / ethics
  • Compassionate Use Trials / legislation & jurisprudence
  • Compassionate Use Trials / trends*
  • Drugs, Investigational / therapeutic use*
  • Health Policy
  • Humans
  • Investigational New Drug Application* / legislation & jurisprudence


  • Drugs, Investigational