Company denies drug to dying child

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In an intensive care unit in Memphis, a virus ravages the body of a 7-year-old who’s in heart and kidney failure. He vomits blood several times an hour as his family gathers in vigil.

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. 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.

Helping Josh, they say, means hurting others.

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.

‘We’re begging them’

It’s called “compassionate use,” but sometimes it feels anything but compassionate.

Here’s the way it works: 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.”

So now, like many families, the Hardys have turned to the media, Facebook, and change.org to pressure the drug company to change its mind.

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.

“Everyone is watching,” one tweeter warned the company. Others have tweeted out the e-mail addresses of the company’s board members. Chimerix executives say they’ve received physical threats.

Moch, the company president, has read these tweets and said he is heartbroken, but the issue is complex and unsuitable for a 144-long character debate.

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. And 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.

Beat cancer four times

Josh’s journey began when he 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.

His doctors at St. Jude’s Children’s Research Hospital said not to give up hope. Since they’d been part of the brincidofovir studies, they’d seen how in sometimes just a week or two, the the oral form of the drug could get rid of an adenovirus without damaging the kidneys. Now all they had to do was ask the company that makes brincidofovir: Chimerix, Inc.

On February 12, the St. Jude’s doctors called a Chimerix executive, Dr. Marion Morrison, and asked for permission to use brincidofovir. She said no.

On March 5, the doctors asked again. Two days later they got an answer by e-mail from another executive, Dr. Herve Mommeja-Marin, who said the company was not “in a position to provide drug for this and other subjects in similar circumstances due to a limited inventory and our limited resources.”

‘He holds our son’s life in his hands’

Moch wants you to know that he has children of his own, and if his child had an aggressive adenovirus like Josh, he’d be doing the same thing as Todd and Aimee Hardy.

“There are no words to express our compassion for this young boy and his family and what they’re going through,” he said.

Art Caplan, a bioethicist, 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.”

But right now, Chimerix stands firm that their compassionate use program is almost completely over.

“We’ve had employees who ask for the drug for family members who are close to death, and the answer has been no,” said Mommeja-Marin, the Chimerix executive.

But that’s not good enough for the Hardys.

“He holds our son’s life in his hands,” Todd Hardy said. “This is just beyond belief to me.”

25 comments

  • ginger

    A child needs help to live; so another words… They are gonna let this helpless child suffer..( not only him but his family ). Children are a gift from god ; if there is a cure or something that could help him…. It should be given to him…not sitting on a shelf…. Don’t understand why they are letting him die…. For gods sake, he is only seven… ( it might help someone later, why not now…..

  • its my business

    Quoted = 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. And 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.”

  • Suzi

    What they are ACTUALLY saying is that they cannot SELL the drug yet & they darned sure won’t GIVE it away…What they mean by “it will hurt others” is that it will affect their profit margin…Where is the compassion in this country?

    • MiMi LLawsonn

      Compassion in this country……THERE IS NONE….there is almost very little standards, morals, values, respect and faith. It has gone to hell in a handbasket….and it is sad that our NC LAWMAKERS do not care what happens to the people here. THEY COULD MAKE SO MANY GOOD CHANGES, but don’t

      • Triple Aught

        This story beats all I have ever heard. The inhumanity of this.
        This company is not even persuaded by the boy’s terrible situation.
        There was a time not too long ago when a company who had this drug would have made this happen.
        Today, even peer pressure is unable to persuade these money grubbers from doing the right thing.
        It seems that Morals, Ethics and Principle have all been sold out to the bottom line.
        Will even one be found Faithful…?

  • Charlie Vickers

    This company states “it is yes to all, or no to all”. They have already said yes to 415 by their own admission. By their own stated standard they must help Josh. After saying yes 415 times they cannot now say no.

    • its my business

      Can you even imagine if you were the boy’s parents? Can any of us imagine that there could be a magic wonder drug out there that could help our child and we are forbidden to try it. (Even though 415+ have used it for this situation) I would jump through hoops of fire to try, I sure can tell you that!

  • its my business

    And in a reply to you MOCH is not MUCH -Quoted … “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.” These words from MOCH. Compassionate use cases at $50,000 per – – person. GIVE THE KIDS THE MEDS if family agrees. Or as you stated, we will let the Compassionate use cases die while because it would greatly hinder your effort to get your DRUG on the Market. I just wish that there was some balance between waiting for the whole time and team of 50 people to patent this and move on. If the kids need this med. MOCH better step u to the plate.

  • FaithC

    I wonder if the drug would be available to the child of one of the people who work for this company? If the CEO’s child was sick, would they give it to that child? I will assume the answer is yes. How can anybody stand by and do nothing knowing they have the ability to help the child? How do they sleep at night?

    • Jimmy

      Never assume… “We’ve had employees who ask for the drug for family members who are close to death,and the answer has been no,” said Mommeja-Marin,the Chimerixexecutive.

      Call your politicions and tell the to make the FDA back off and let the drug company simply hand the child the medication. That will never happe though, the goverment loves its bureaucracy. They have convinced you yo blame the drug company. Just a thought.

  • Jimmy

    Maybe we should go after the government run, out of control FDA. The mind boggling bureaucracy that comes from the government seems to be the hold up. I could be wrong though.

  • Philomena Marino

    If no one will help this boy, alternative is his only hope. If the drug company chooses to let a child die, that right there tells you what our countries corporate priorities are.
    II would get this boy on Chinese herbs immediately. Yunnan piao alone stops bleeding and is antiviral.
    Colloidal silver and things like rhemannia, astragalus, chaga mushroom.
    And before anyone says quackery, your precious conventional medical system just told this boy, go die, oh well.

  • Lex

    Maybe you guys should reread the story and stop picking out bits and pieces. This kid is so sick it’s highly unlikely the drug will help him.. not to mention they gave him an IV form of it which ravaged his kidneys. If they effects are BAD it has to be reported to FA which could hinder the mess from reaching more than just this ONE kid….. In science there will be sacrifices….. One child or MILLIONS of sick people…….
    And really let’s face it….. In God’s time it could just be this kid’s time. …..

    • Charlie Vickers

      Lex, maybe you should reread Josh’s story. The intravenous form of this med attacked his kidneys. As such they could not continue with that therapy. The difference is they are trying to get the oral form of this med from this company because it does not attack the kidneys. By this company’s own statement if they say “yes” to anyone they have to say “yes” to all. They have already said “yes” to 415 patients. Unless they are a lying, deceitful company they have to say “YES” to Josh!

  • Samantha

    Yeah they might be a possibly the drug won’t work but that don’t mean they can’t give it a try I mean really this child is 7 years old have a heart let them have this drug you might can save his life if it was your child you would give it to him regardless of what anybody said so why don’t you just let the doctors try it and see if it will work this should be able to life and have a full happy life and if you want to treat one of Gods angels like this I feel sorry for you bec God will bring you down then you can think about this little sweet child and what he had to go thru because you was to heartless to help him and his family.

    • Charlie Vickers

      Lex, the rest of us are arguing for the life of this child and you are arguing for all the “i” to be dotted and all the “t” to be crossed. Fact is their executive said it had to be yes to all or no to all and they have already said yes previously. Their facts not mine!

  • lorriegoose

    They didn’t give us the email addresses of the drug company executives to contact. If like to know how to let them know how I feel about this and maybe if enough of us make a plea to them they will change their minds. I bet if this were Mr Moch’s son the answer would be very different.

  • Charlie Vickers

    Lorriegoose, the drug company did give the med and after just 3 treatments he is practically normal according to a news release just yesterday. The right response from the drug company, and the right results from the treatment.

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