Drug Development, Compassionate Use, and Adverse Events: A Cautionary Tale

by Alison, Bateman-House, Ph.D., MPH

In late September 2014, a man was admitted to the hospital due to vomiting, diarrhea, and fever. Suffering from a viral infection with no known cure, he was given an experimental antiviral medicine; unfortunately, he died. Maybe the experimental medicine did not work. Alternatively, maybe the man was too ill by the time the drug was administered for anything, no matter how effective, to work.

This patient’s name was Thomas Eric Duncan, and he captured media headlines as the first person to die of Ebola in the United States. The fact that his doctors tried an experimental drug called Brincidofovir on him also captured headlines, with his unfortunate fate directly impacting Chimerix, the small biotech company developing the antiviral drug. At the time that Duncan became ill, Brincidofovir was being studied in clinical trials for other types of viral infections, and it showed good efficacy and tolerability. This, plus the fact that no treatment for Ebola existed, made it reasonable for Duncan’s doctors to request that they be allowed to try treating him with Brincidofovir.

This single case can offer no insight into whether Brincidofovir is effective against the Ebola virus. If Duncan had survived, his survival may or may not have been due to Brincidofovir’s antiviral properties. Likewise, his death may, or may not, indicate that Brincidofovir is not effective against Ebola. What this case can do, however, is demonstrate how a public failure of an experimental drug can impact the company developing the drug.

Chimerix’s stock price had jumped after it was announced that Duncan’s doctors were going to try Brincidofovir.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.