Family Fights For Compassionate Use of Non-FDA Approved Drug

A North Carolina company is developing an experimental drug that could save lives — including a 7-year-old boy named Josh Hardy.

So when Josh’s family heard about the possible cure – CMX001 (Brincidofovir) the life saving antiviral drug made by Chimerix – they pleaded to get it approved for their use.

Josh’s parents say that he needs this specific medication to fight off a viral infection he developed after a recent bone-marrow transplant.

“We’re trying to save our son,” Josh’s dad, Todd Hardy tells USA Today. He and his wife, Aimee, are urging supporters, on social media and in news stories, to pressure Chimerix to provide the drug, but the pharmaceutical company has already turned down multiple requests from Josh’s doctors.

This heartbreaking story outlines the ethical, medical and financial issues surrounding compassionate use – the use of medications that have not been approved by the Food and Drug Administration but are in earlier stages of development.

Due to the Hardys’ public outcry, thousands of people have e-mailed and called Chimerix pleading the company to make their drug available to Josh, company president and CEO Kenneth Moch said Monday.

“We have have great compassion for this family,” Moch says. “But this is not just about a single boy.”

But according to Josh’s parents, this is the only way to save their son’s life.  “Our doctor said ‘This drug will cure and save your son,’ ” the distraught father says.

Medical ethicist Arthur Caplan can see both sides of the story.

“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.

On the other hand, Caplan 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 decrease a push for a drug’s approval and expose a company to added risks if people end up harmed by their experimental drugs outside of formal trials.

As well, he says it’s important that any drug goes through the rigorous FDA approval process.

“It’s always a long shot that it will help and not make things worse,” Caplan adds.

In a statement via Facebook, the Hardy family pleads for public support on this controversial topic.

“Thank you to every member of Josh’s Army. The world has heard you and because of you Josh and many others will have the opportunity to receive CMX001 (Brincidofovir) the life saving antiviral drug made by Chimerix.

There are too many people to thank individually, everyone’s actions, thoughts and prayers have made a difference. Words cannot express how grateful the family is for your encouragement, conviction, and involvement. We would like to specifically acknowledge and thank the following groups and organizations; Coalition Against Childhood Cancer, PAC2, The Max Cure Foundation, Alex’s Lemonade Stand, CaringBridge, Boyko LLC, the FDA, and especially St. Jude Children’s Research Hospital. Please support these organizations in their fight against childhood cancer.

Finally, we would also like to thank the dedicated, gifted and talented employees, and Board Members, of Chimerix, especially Kenneth Moch, whose skill, dedication and expertise developed and made available this life saving drug. Thank you #SaveJosh.”

Our thoughts and best wishes go out to the Hardy family!

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