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.”

More from CNN on this story

25 comments

  • Skeptic

    Everyone says its the doctors and hospitals that are the culprits in rising costs. The drug companies are what’s causing the most problems. They are playing G-D with our lives for the sake of money.

    • Maximum

      Don’t forget about the government entities that have over regulated every aspect of drug development, production and sales! The FDA changes the rules daily and keeps these companies stalled for years in testing and development. I am not abdicating letting these companies run wild but the regulations have hampered drug development and made the cost so prohibitive that unless a drug is going to make high millions in profit they can’t afford to even start. Lets place the blame evenly across the guilty parties.

    • halloween3

      Well said, these drug companies do what they want and it is all about the Money. If people do a little research they would know how corrupt these drug companies are.

    • Thomas Offal

      The company is not denying Josh the drug because it’s evil or because of money – it’s denying because providing the drug would likely delay FDA approval by months or years. The federal government Chimirex $72M to run a CLINICAL TRIAL — which does NOT require it (or any other pharmaceutical company) to distribute an unapproved drug on a “compassionate use” basis.

      Do you know WHY there are stringent laws/regulations governing clinical trials? Because 1) it ensures that drugs are safe before doctors are allowed to prescribe them and 2) stops horrors like the infamous Tuskegee Experiment from EVER happening again. The laws/regs are NEEDED.

      Last but not least, there are multiple stages to a clinical trial – in this particular case, the FDA has deemed it safe (Phase 1), effective relative to placebo (Phase 2) and is in the process of confirming it to be safe/effective (Phase 3). The drug was NOT designed to fight the type of infection little Josh is battling, so if he got the drug and had an adverse reaction for any reason (e.g. so sick that he died anyways, reacts badly with the other drugs he’s taken, etc), Chimirex is required to report it to the FDA, which will delay FDA approval by months or years while the reason for the adverse effect is investigated.

      My heart goes out to Josh and his family – but the drug company is making the RIGHT call by refusing to give him the drug.

  • Tata

    Amen Kay I’m praying that they have a heart in this case if they stop putting all the extra poison on food for faster growth& giving them medicine with all these crazy effects all of these unknown illnesses wouldn’t be killing our young

  • BlueVelVet2002

    Karma is a son of gun. No one ever wants to walk in another person shoes. This poor child and his family is suffering and this is about as cold as it can get. Sleep well Kenneth Moch (you could fight harder) and the big boys and girls at the FDA because you never know what tomorrow can bring. Ridiculous!

  • Kay

    They should have to go watch him lie in that bed, slowly slipping away from his family…. then go through the complete process of the funeral and watch the parents grieve a child they know could’ve been helped. Just think what would happen if this drug helped save this little boy? That could only be a good thing!!!! God will have His hand on this baby… no matter what. May God give this baby and family comfort for all of the days to come.

  • Angela VM

    This is total BS from the drug company. The only thing that they are concerned about is $$$$$. If it works for this child that is great, if it doesn’t work then it has not cost him anything. The drug companies run the health system here. and it is all about $$$$.

    • Thomas Offal

      Money isn’t the issue here — it’s the fact that if this kid (or ANYBODY ELSE) had an adverse reaction to the un-FDA-approved drug they received under “compassionate use”, it would delay FDA approval of the drug by months or years.

      There are VERY strict laws/regulations in place for clinical trials — to ensure drugs are safe before doctors are permitted to prescribe them AND to ensure horrors like the infamous Tuskegee Experiment never EVER happen again, i.e. laws/regs are needed.

      No one is entitled to a drug that is NOT FDA approved, not even a dying little boy.

  • KT

    I need someone to clarify something for me. I agree with what everyone is saying for the most part, but in this specific case is it really all about money? What if someone or a group of individuals were willing to donate the $50K, would the company allow the use of their drug?

    • George Brown

      No. Someone offered to pay already. The company maintains giving the child the medicine means it must give it to others which would then prompt a delay in FDA approval and therefore delay making it available to everyone.

  • Memphis

    Sad!! that a sick child has to endure pain and suffering at the hands of a uncompassionate drug company. God so love the world that HE GAVE HIS ONLY BEGOTTEN SON THAT WHO SO EVER BELEIVE IN HIM SHALL NOT PARRISH BUT HAVE EVER LASTING LIFE. May God bless this family and touch the hearts of the CEO/ EXECUTIVES of Chimerix Drug Company.

    • Thomas Offal

      Money isn’t the issue — an adverse effect that would delay FDA approval of the drug by months or years is the issue.

      The drug is not yet FDA approved, the drugmaker is NOT required to provide it under “compassionate use”. My heart goes out to this kid and his family, but I 100% understand WHY the Chimirex will not give him (or anybody else not currently enrolled in their Phase 3 clinical trial) access to this drug.

  • Theresa Orr

    Cold hearted, money driven, loss of priority, lack of ethics, morally desensitized, dirt bags…Make the medicine, give the medicine; if the demand is great, make more medicine; recruit others to continually make more medicine when you have a good product; explain challenges to the FDA…work harder, smarter…stronger…remember life-saving is the ultimate priority, monetary gains…secondary!

  • tjp

    I’ll be “devil’s” advocate: Experimental drugs are very touchy things. Consider the very real possibility that the drug fails. Not only does this child die, but more lives are impacted. The company goes under from the bad press and investors pull out. People lose their jobs that had nothing to do with the decisions. Caution and looking at the bigger picture are usually good traits, so we must not forget that. The reality of life is that not everyone can be saved. We all face our impending deaths whether we die later today or 20/30/40/50 years from now. Yes it is tragic, but if this drug fails with this specific case and gets mothballed, hundreds if not thousands of other patients could be effected in the future. Emotion gets the better of us. Issues like these aren’t black and white. Good or bad. Much like all of life. Keep that in mind and in perspective.

  • Jane

    Actually, in some respects, it’s the loved ones that don’t want to realize that it’s time to give up the ghost and let their sick child/parent/sibling go. It’s this reluctance to accept death that causes a lot of healthcare costs to skyrocket (bed costs in a hospital, physicians, as well as Big Pharma). This kid has no quality of life and hasn’t for a while. It’s time to let him go to a better place and stop his suffering.

    Before any of you decide that I’m a troll, I’ve been there having to make this decision, and I made sure to do the merciful thing and end my loved one’s suffering, And yes, I’m in the healthcare sector, so I also understand the costs involved as well.

  • Brian OR

    Marginal cost of delivering doses of existing drug: $1000.
    Cost of tracking patient outcomes: $49,000.
    Cost of bad publicity: Priceless.

    The company’s argument seems to be over cost of tracking. Knowing outcomes is highly useful (if for example a particular drug turned out to uniformly kill such dire-need patients). However, but it’s not self-evident that it should be Required under such last-chance cases. Alternatively, why not legally require industry group (1% surcharge on existing sales) or gov’t agency to set-aside funds to cover costs of approved compassionate use cases?

    • Thomas Offal

      Money isn’t the issue, nor is tracking — potential to delay FDA approval by months or years as a result of an adverse effect on a patient with “compassionate use” access to the drug is problem. The drug isn’t yet FDA-approved — so Josh isn’t entitled to access it, no matter how cute and young and sick he is.

      Bad publicity? I. Don’t. Think. So. Chimirex’s drug works GREAT — why else would Josh’s family (and hundreds of others) be begging for access to it?

      Clinical trials have several phases — to determine if a drug is safe (Phase 1), effective relative to placebo (Phase 2) and to confirm that the drug is indeed safe/effective before it is formally approved by the FDA (Phase 3). The drug in question is in Phase 3 — it’s safe and it works, or it would not have made it this far in the clinical trial.

  • EL Hoard

    This company is playing roulette with the life of this child. They choose to sacrifice this child for what might OR might not be for the greater good. You and I know that it’s about the money. My heart goes out to this family. This company has no heart, conscience, or compassion.

    • V. Cruz

      I believe both the company and parents are playing roulette with the child’s life. What wouldn’t i do to save my child in addition to selling my assets. As a parent it is my responsibility.
      Should i put my child in danger because the drug company won’t abide.
      That’s what refinancing, credit lines, equity lines, loans are for – emergencies. Fight one’s battles after – don’t endanger a life.
      Many take loans for cars, vacations, ect .. $50,000 to save a child …
      God forbid i would sell my home … material things are replaceable – my child, … priceless.

  • Amy Priesmeyer

    I would think it would look good for them to give this drug to Josh. Maybe after seeing it work with him, the FDA would approve it for others sooner.
    It is hard to see any child sick, hurting, and or dying and not be compassionate. My heart sinks at the thought it could be my own son in the same situation. God Bless the family and this little guy and the other children suffering.

  • V. Cruz

    I understand the problem very clearly. And i see the issues the family is dealing with. Also the drug company and their refusal to cooperate.

    WHAT I DON’T UNDERSTAND IS – I HEARD THE MOTHER ON THE CNN INTERVIEW SAYING TO THE EFFECT THAT SHOULD THIS END FATAL THE COMPANY IS TO BLAME.

    Why would any parent chance danger for their child. I mean if someone is in poverty and homeless i can see there are no options.

    I an not a wealthy person, barely middle class. I took an equity line of credit out for my husbands surgery and this was not chronic.
    For my child’s life – it is priceless. I would sell my car, jewelry, home, and any type of assets for his/her recovery/safety.

    What i am saying is why would one stand this gamble with the drug company is at stake.
    This is life … material things are replaceable.

    Many take out lines of credit and loans to buy a car … $50,000 to make a difference in a child’s life …

    I JUST DON’T UNDERSTAND …I do feel for Josh.

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