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 doesn’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.”