Dying Boy to Get Unapproved Drug After Family's Plea


"We all have great compassion for this child," Moch said. "We spent our lives trying to develop new medications for patients just like Josh… We need to make sure to get this drug available as soon as possible to as many people as possible."

He said in a statement Tuesday evening that the 20-patient trial in which Josh is involved underscore's Chimerex's mission.

"Being unable to fulfill requests for compassionate use is excruciating, and not a decision any one of us ever wants to have to make," he said in the statement. "It is essential that each individual in a health crisis be treated with equal gravity and value, a principle we have upheld by pursuing further clinical study of brincidofovir that will inform its use in adenovirus and other serious DNA viral infections."

New York University bioethicist Arthur Caplan said the issue is not merely about a family pleading with a pharmaceutical company. He said the tiny company likely has many things working against it that prevent it from offering its drug to patients outside of clinical trials.

The company likely didn't have the resources to develop to a broad compassionate use program, Caplan said, and if it did and patients died – even of unrelated causes – that could hurt the drug’s chances of getting approved by the Food and Drug Administration. Chimerex declined to comment on these issues.

“Month after month, these cases come up of families seeking drugs or medical devices in dire circumstances appealing for compassion,” Caplan said. “We’re treating it as if it’s standoff between a desperate family and a little company. It isn’t.”

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