UPDATE: 9:00 p.m. -- Chimerix announced in a press release that it's reached an agreement with the FDA to start a pilot trial of open-label brincidofovir. The release adds, "This study is expected to begin with Josh Hardy as the first patient enrolled on Wednesday, March 12, 2014." Read the full press release here.
Josh Hardy has fought hard to stay alive. Just 7 years old, he has already survived four bouts of kidney cancer, heart failure and a bone marrow transplant. Now a new obstacle stands in the way of his health: a pharmaceutical company denying him a potentially life-saving medication.
As a viral infection devastates Hardy’s body, doctors at St. Jude's Children Hospital recommended the family try the antiviral drug brincidofovir, which has not yet been approved by the Food and Drug Administration. But the medicine's manufacturer, pharmaceutical company Chimerix, won't administer the drug and says giving it to him would slow down efforts to get brincidofovir approved and bring it to market.
"Our son will die without this drug," Todd Hardy, Josh's father, told CNN. "We're begging them [Chimerix] to give it to us."
The FDA's "compassionate use" clause sometimes allows pharmaceutical companies to administer unapproved medications to treat a patient with a life-threatening condition. Chimerix has done so in the past for 451 patients; but Chimerix President Kenneth Moch told CNN that the $50,000 it would cost to make this exception for Josh Hardy would be too much of a financial setback, since the company would have to make the same exception for every patient who wants the drug.
Chimerix has received more than $72 million in federal funding to develop brincidofovir, FOX News reports.
Medical ethicist Arthur Caplan of the New York University Langone Medical Center says both viewpoints are understandable in this complex case.
“I have huge sympathy for the family,” he told USA Today. “I think they are right to try and see what they can get for their child.”
Caplan also noted that there’s no guarantee the drug will even work, and that there’s always a risk involved in using drugs that are not FDA-approved.
"It's always a long shot that it will help and not make things worse," he said, per USA Today.