Josh Hardy medication fight raises questions about drug approval - DC News FOX 5 DC WTTG

Josh Hardy medication fight raises questions about drug approval process

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WASHINGTON - After days of pleading with drug company executives, the parents of a Fredericksburg, Va., boy in danger of dying got what they have been praying for: a chance to get the medicine that could help their son survive.

Starting on Wednesday, 7-year-old Josh Hardy will be the first patient in a new trial launched by drug company, Chimerix. It means he will receive the experimental medication that doctors think will save his life.

His parents started hashtag #SaveJosh. It went viral on social media and was instrumental in helping the family secure this treatment.

“We got the great news last night and we're just looking forward to great success of the drug and the great success of the company to help other people,” said Josh’s mother, Aimee Hardy.

Right now, Josh remains in critical condition at St. Jude Children's Research Hospital in Memphis, Tenn., where doctors had also been pushing the company to provide the drug.

The #SaveJosh campaign wouldn't have been possible 20 years ago. Josh's story went around the world thanks to people who heard about his story and called for action.

But it is also causing some medical ethics experts to point out the campaign has pulled back the curtain on what they say is a drug approval process that is too slow.

Pharmaceutical company Chimerix says it is reversing its earlier decision and will give Josh him the experimental drug that could save his life.

“We are beginning a broader trial that will initially enroll 20 patients and will move to being what's called a pivotal Phase 3 study, so it will be designed to move potentially to the approval of brincidofovir by the FDA,” said Chimerix CEO Kenneth Moch. “The study has to be undertaken, but that is our hope and plan.”

The decision was fueled by an outpouring of public interest on Twitter, on Facebook and social networks like caringbridge.org. Tens of thousands people demanded Chimerix change its mind and give Josh the medication.

But while the #SaveJosh campaign has raised awareness, here in Washington D.C., it has also raised questions. For example: when it comes to experimental medicines being approved, what is right and what is wrong when science and emotion collide?

The Food and Drug Administration allows so called "compassionate use" of certain drugs -- in certain cases -- before they have been approved. But patient advocates like Frank Burroughs of the Abigail Alliance say the FDA should do more.

“These people are out of options,” he said. “Their only option is death. It doesn't have to be that way. The FDA is in the way.”

Art Caplan, a medical ethics expert and professor at the New York University Langone Medical Center, told us some scientists are concerned expanding the uses of unapproved medicines could alter clinical drugs trials.

"If all the Joshs get compassionate use, there's nobody in the trial and that's where the dilemma is,” he said.

We asked the FDA to speak with us about their compassionate use program. They instead sent us an email with information about their program and how FDA approves drugs, and how people can apply to the program.

View the links here:

Overview of the drug approval process:
http://www.fda.gov/Drugs/ResourcesForYou/Consumers/ucm143534.htm

Access to Investigational Drugs Outside of a Clinical Trial (Expanded Access):

http://www.fda.gov/ForConsumers/ByAudience/ForPatientAdvocates/AccesstoInvestigationalDrugs/ucm176098.htm

FDA Blog on Expanded Access:
http://blogs.fda.gov/fdavoice/index.php/2012/04/remembering-krebiozen-why-we-have-access-to-unapproved-drugs-and-why-we-need-scientific-evidence/

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