How Does Compassionate Use Work?
Compassionate Use, or Expanded Access, as it currently exists in the U.S. does not simply guarantee access to an experimental drug to any terminally ill patient who requests it.
Only a physician, on behalf of his/her patient, may apply for compassionate use access, and strict rules require that the patient must be suffering from an illness for which “there is reasonable likelihood that death will occur within a matter of months or in which premature death is likely without early treatment,” must have exhausted all other treatment options, and is ineligible to participate in this (or a similar) drug’s clinical trial.
There are additional safeguards (or roadblocks, depending one’s position) to ensure proper dispensing, application, and oversight of the drugs. The following is a brief description of how the Compassionate Use application process operates in the U.S.
