A company says it can't give an experimental drug to one child without giving it others, slowing attempts to get the drug approved for many more people in the future.
A Virginia family's plea to get an unapproved medication for a 7-year-old cancer survivor with a life-threatening illness has led thousands of people to contact the small North Carolina company that makes the drug.
The family of Josh Hardy says he needs a medication made by Chimerex, of Durham, N.C., to fight off a viral infection he developed after a recent bone-marrow transplant.
"We're trying to save our son," says Josh's dad, Todd Hardy, who is with him at St. Jude Children's Research Hospital in Memphis. He and his wife, Aimee, are urging supporters, on social media and in news stories, to pressure the company to provide an antiviral drug called brincidofovir. The company has already turned down a request from Josh's doctors.
The case illustrates the ethical, medical and financial quandaries that surround so-called compassionate use – the use of medications that have not been approved by the Food and Drug Administration but are in earlier stages of development. FDA rules allow companies to provide such drugs to some patients under certain circumstances.
Since Thursday, thousands of people have e-mailed, called or otherwise contacted Chimerex to ask the company to make their drug available to Josh, company president and CEO Kenneth Moch said Monday.
But Moch says the company can't say yes to them – because that would mean saying yes to many more similar patients and draining the company of resources it needs to complete a formal study of the drug, seek FDA approval and potentially help many more future patients.
"We have have great compassion for this family," Moch says. "But this is not just about a single boy."
The 54-employee company receives "many, many requests" for compassionate use of the only drug it has in clinical testing, he says. It can only say yes to people who meet a couple of strict criteria – those who have been on the drug in a previous trial and newborns with herpes simplex virus, he says. The company is about to start a trial in such babies. Moch says the company would also make the drug available to smallpox victims in a bioterror outbreak.
Josh doesn't meet any of those criteria, he says, and the company has no current study for children with his infection. Its only ongoing study is in adults with a different viral infection. A broader compassionate-use program ended two years ago, Moch says. The company says it has provided the drug to 430 compassionate-use patients in all.
Todd Hardy says doctors at St. Jude thought of brincidofovir because they tried it in children in a previous study. They are seeking it for Josh, he says, because the antiviral drug he is getting now to fight off an adenovirus infection has damaged his kidneys. "Our doctor said 'This drug will cure and save your son,' " he says.
St. Jude's press office did not immediately respond to a request for comment.
Medical ethicist Arthur Caplan, a professor at New York University Langone Medical Center, says he understands the viewpoint of both the family and the drug company.
"I have huge sympathy for the family. I think they are right to try and see what they can get for their child," he says.
But, he says, the company is in a position shared by many small pharmaceutical companies with limited resources and worried investors. Dedicating staff, time and money to compassionate-use requests can slow down a push for a drug's approval, he says, and expose a company to added risks if people end up harmed by their experimental drugs outside of formal trials.
He says it's also important to remember that doctors and patients can't know what they are getting with an experimental drug that has not gone through the FDA approval process.
"It's always a long shot that it will help and not make things worse," Caplan says.
But Todd Hardy, a custom home builder from Fredericksburg, Va., says he is convinced the drug remains his son's best shot at getting the three wishes he had his mother write on a board near his hospital bed in anticipation of his 8th birthday on March 31.
"He wants to drink whatever he wants, he wants to be able to walk by himself and he wants to get out of the hospital."