No drugs for dying child because $$$

[Ksandra]

(CNN) -- 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.
Kenneth Moch says he is also a father.
Kenneth Moch says he is also a father.

"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.
Josh Hardy beat cancer four times, his family says.
Josh Hardy beat cancer four times, his family says.

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 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."

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."

http://www.cnn.com/2014/03/10/health...html?hpt=hp_c2

Makes me want to punch something.

According to the comments (anyone can verify?) the company is lying in regards to compassion cases affecting approval as those are omitted from the decision making.

Here's the fb page:

https://www.facebook.com/SaveJoshHardy

Here is the change.org petition:

http://www.change.org/petitions/chim...ardy-right-now

White House petition:

https://petitions.whitehouse.gov/pet...-life/Jqg7ZMrR

[djzombie]

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.

This doesn't make sense to me. So for 3 years they were able to get valuable information from compassionate use cases but now all of the sudden they don't learn anything from them? How the fuck does that make any sense? Unless they've learned absolutely everything they can from those cases in 3 years, if so why isn't the drug on the market yet? I mean what takes 2 more years? Marketing? You really need to market something that saves lives? Sounds like some bullshit to me, but I don't know shit about pharmaceutical company's procedures.

And even if that is accurate, the bottom line is still money. They're either not going to give out the drug to patients this year because of the 50,000 per patient, or because their profits would be delayed from selling the drug on the market, probably both. Any talk of wanting to get the drug on the market faster to help people is clearly bullshit because they have the opportunity right here to help people via compassionate use cases and refuse.

[Ksandra]

This doesn't make sense to me. So for 3 years they were able to get valuable information from compassionate use cases but now all of the sudden they don't learn anything from them? How the fuck does that make any sense? Unless they've learned absolutely everything they can from those cases in 3 years, if so why isn't the drug on the market yet? Sounds like some bullshit to me, but I don't know shit about pharmaceutical company's procedures.

Ya, the whole thing is such shit. I don't know much about the whole procedures but this:

Chimerix is going full speed ahead to get the drug on the market hopefully by the end of 2016

Screams to me they want to save it so they can sell it, and don't want to waste any more money on it.

[hey]

They can't spare even one second to save the kids life, because it'll interfere with saving other lives. Ok, that makes sense, gotta focus on the important lives first.

But then how does the ceo himself have time to talk about how horrible it is?

Actually read more of it, and it makes sense. Can't have high profile cases that are most likely going to end in death either way. It sucks, but if the drug works, having something like this hold it back would suck even more.

[Myrrh_Quetz]

Shit like this pisses me off. It's the same thing for a kid with CF trying to get a new drug that costs 350k a year in Canada. Government is being massive dicks about it

[archibaldcrane]

That sucks, but if noone is going to cover the costs of the drug, I have a hard time raging against the drug company. If they kickstarted a fundraiser to hit a certain $ amount, and the company still refused, then that would be a shitty move. There's probably laws against paying for pre-market "compassionate use" drugs though.

[archibaldcrane]

According to the comments (anyone can verify?) the company is lying in regards to compassion cases affecting approval as those are omitted from the decision making.

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

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.

In the article the company isn't claiming that they'd have to report to the FDA - an NYU bioethicist is.

[Ksandra]

[strike]
Actually read more of it, and it makes sense. Can't have high profile cases that are most likely going to end in death either way. It sucks, but if the drug works, having something like this hold it back would suck even more.

Why would it hold it back?

In the article the company isn't claiming that they'd have to report to the FDA - an NYU bioethicist is.

Fair enough, but I still want to know their reason why this kid would hold them back but not the 451 other patients.

[archibaldcrane]

Because earlier in their development, it was worth the expense as it aided their research.

[Ksandra]

I wonder if the parents are refusing to pay for it or are compensation drugs not allowed to be paid for? I actually have seen no mention about this being an issue where the parents wouldn't pay for it just that it would "delay it getting released." Which sounded like a code word for "we want to be able to charge even more."

[archibaldcrane]

Or that any delay could prevent many more from receiving the drug.

[Blubbartron]

Why would it hold it back?



Fair enough, but I still want to know their reason why this kid would hold them back but not the 451 other patients.

The most plausible explanation I can think of is that 2-3 years ago they were still in the beginning of the human testing phase, and testing a drug which has had minimal human testing on people who are already out of options and likely to die anyway is the most ethical thing you can do while still being scientifically useful. After you establish the premise that the drug works on some humans (especially the worst cases), you can ramp up the clinical trials with patients that aren't so bad off as an "end phase" to make the data look good when presenting to the FDA. It's all about maintaining chronological consistency and the facade that the drug has been refined to increase efficacy while decreasing unintended consequences.

The companies aren't required to report compassionate use (read the full article), but it could be concerning if the FDA finds out about it during review of the clinical trials. As the companies themselves put it:

The use of an investigational drug in less controlled setting, in patients with very advanced disease could lead to adverse reactions that might raise difficult to resolve but spurious safety concerns about the drug.

It's pretty stupid, and the FDA just needs to write up a bylaw or some shit to say that compassionate use cases will not have their outcomes count for or against the approval of drugs. Done and done.

[Tajin]

So, Didn't read LOL.jpg, but I wonder, if the company agreed to give the medicine and the kid died as a result of it, what then? People would hate on the company?

Edit: welp, this is what happens when you idle and press post 40 minutes later. I see my question has been somewhat addressed. :/

[Moss]

Based on the article I can't rage against the company - it's just a shitty situation. I'm sure there are a ton of hoops to jump through in the FDA approval process and don't doubt approving compassionate use cases would slow it down.

[Tagus]

problem is, if the kid takes the drug and still dies, the fda may not approve it to be sold ever in the future because there is now a statistical record of a child having died after taking it. any time somebody dies after taking a new medication it really hurts the company's chances of getting it approved. shit sucks man.

[djzombie]

The use of an investigational drug in less controlled setting, in patients with very advanced disease could lead to adverse reactions that might raise difficult to resolve but spurious safety concerns about the drug.

problem is, if the kid takes the drug and still dies, the fda may not approve it to be sold ever in the future because there is now a statistical record of a child having died after taking it. any time somebody dies after taking a new medication it really hurts the company's chances of getting it approved. shit sucks man.

what i'm hearing from these post is that its better for the company to wait until they are charging for the drug before giving it to critically ill patients whose life it may save, because that way if they still die at least the company has their money and can go on selling the fda approved drug that just killed a patient.

don't wanna raise those safety concerns til after the drug is approved and being sold. solid scientific process amirite?

[Ksandra]

http://www.cnn.com/2014/03/11/health...html?hpt=hp_c2

After days of pleading with drug company executives, Josh Hardy's parents got what they'd been praying for: a chance to get medicine that could help their son survive.

The Chimerix pharmaceutical company said Tuesday that the ailing 7-year-old will receive medicine that doctors hope will help him when he becomes the first patient in a new trial set to start Wednesday.
In the company's statement Tuesday, he said he hopes the new FDA-approved study will help other patients as well.

"This 20-patient open-label study underscores Chimerix's mission to develop innovative antiviral therapies in areas of high unmet need -- for everyone," he said. "Being unable to fulfill requests for compassionate use is excruciating, and not a decision any one of us ever wants to have to make. It is essential that each individual in a health crisis be treated with equal gravity and value, a principle we have upheld by pursuing further clinical study of brincidofovir that will inform its use in adenovirus and other serious DNA viral infections."

A Facebook page dedicated to the online campaign to help Josh get the medicine posted a thank-you message to supporters Tuesday night.

"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. "

I just hope that this getting the attention it did may help to make some ruling that the fda can't use compassionate cases in their approval decisions.

[hey]

what i'm hearing from these post is that its better for the company to wait until they are charging for the drug before giving it to critically ill patients whose life it may save, because that way if they still die at least the company has their money and can go on selling the fda approved drug that just killed a patient.

don't wanna raise those safety concerns til after the drug is approved and being sold. solid scientific process amirite?

Except the drug didn't kill the patient, they were going to die either way.

I get that big pharmaceutical companies suck, but ya know, sometimes the drugs actually do work. Just because they aren't miracle cures doesn't mean they aren't effective. Giving them to people you know aren't likely to be helped and will most likely die either way isn't really a solid scientific process. Even if they do get better, you've only given it to one patient in such a condition, so it means nothing.

[Serra]

So, can someone confirm or deny this -- I read that the company behind the drug is in the hole 200 million. Can't find any reputable sources though (didn't really try that hard to look into their finances).

[Meresgi]

So, can someone confirm or deny this -- I read that the company behind the drug is in the hole 200 million. Can't find any reputable sources though (didn't really try that hard to look into their finances).

Not unheard of, companies spend a FUCKTON of cash to research these things and that's why they are so Scrooge McDuck over giving them out to people, because they are basically betting the entire company while hoping the drug will recoup all their investments and make put them in the black.