This Child Is Dying. Is A Drug Maker Cold Hearted Or Practical?

(Memphis) A pharmaceutical company says it can’t slow down help for many patients to possibly save one.

Josh Hardy, 7, is in an intensive care unit at St. Jude in heart and kidney failure.

He vomits blood several times an hour as his family gathers in vigil.

Josh’s was diagnosed with a rare form of kidney cancer at 9 months old.

Over the years, cancer turned up in his thymus, lung, and bone marrow, and each time Josh beat it.

But a bone marrow transplant left Josh without much of an immune system, and in February doctors diagnosed him with an adenovirus that spread through his body.

They gave him an antiviral drug, an intravenous form of brincidofovir, but it ravaged his kidneys.

In a cabinet in Durham, North Carolina, there’s a drug that could likely help Josh Hardy, but the drug company won’t give it to him even after doctors at St. Jude requested it.

They’re adamant that spending the time to help Josh and others like him will slow down their efforts to get this drug on the market.

When asked how he will feel if Josh dies — and he’s in critical condition, so sadly that could happen soon — the president of the company that makes the drug doesn’t hesitate to answer.

“Horrible,” said Kenneth Moch. He would feel horrible and heartbroken.

But still, he said there’s no way he’s going to change his mind. There’s no way he’s going to give Josh this drug.

According to the Food and Drug Administration, if someone has a serious or immediately life-threatening disease and has tried and failed other available treatments, they can ask a drug company for an experimental drug, one that they’re still studying and has not yet been approved by the FDA.

Companies often say yes: The FDA approved 974 compassionate use arrangements in fiscal year 2013.

But pharmaceutical companies often say no, as they did to Josh Hardy.

“Our son will die without this drug,” said Todd Hardy, Josh’s father. “We’re begging them to give it to us.”

Countless members of “Josh’s army” have responded with angry tweets to @chimerix, telling them to “open their hearts,” asking the executives how they can sleep at night.

At its very simplest, this is it: Chimerix is going full speed ahead to get the drug on the market hopefully by the end of 2016, and if they spend time and money on compassionate use cases, it would greatly hinder their effort to get the drug, brincidofovir, on the market and available to everyone.

The company would have to dish out $50,000 per compassionate-use patient, since insurance doesn’t usually pay for experimental drugs, Moch said.

Perhaps even more important than the money, it would divert manpower in this 50-person company, since they’d have to handle the requests and then get the patient’s records and follow-up with them, as required by the FDA.

“If this were just one patient wanting this drug, then this would be a very different question,” he said. “But it’s yes to all or no to all.”

From 2009 to 2012, the company did give out the drug under compassionate use to 451 patients, Moch said, but at least at that time, the information gleaned from those 451 compassionate use patients was helpful to the Chimerix study and helped move the science along.

But currently doctors don’t really learn very much, if anything, from compassionate use patients, so the patients don’t help get the drug to market.

Art Caplan, a bioethicist at NYU Langone Medical Center, said he feels for both the Hardys and for Moch.

“We can’t ask the company to turn into a philanthropy or their investors will back out,” he said.

It’s not just the $50,000 per patient that might make investors squeamish, Caplan said, but compassionate cases can make a drug look bad. By definition, compassionate use patients are extremely sick, and might not do well with the drug. Companies have to report that poor outcome to the FDA in its application to market the drug.

Perhaps there’s another way to handle compassionate use requests, Caplan suggests. Perhaps a company like Chimerix could agree to give the drug only to the very most dire cases, and put a cap on the number of patients they help.

“They might want to open the door a little more broadly,” he said. “They might want to show a little compassion.”

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