7 year old fighting for his life - drug company REFUSES to allow access to life-saving medicine

I think it sucks they don't give it to him. I understand it's business but sometimes you've got to bend a bit. Hell, they could do it off the freakin' radar.

I agree with Kay on this. This drug was developed primary for the treatment of herpesvirus infections that are very serious infection in immunocompromised patients. It was not developed or adenovirus infections which the child has. I am also a bit suspicious on how this case had been reported by the press which seems to suggest that it is clearly an issue with money. This is a statement that was released by the company.

"This is one of hundreds of requests that Chimerix has received for the compassionate use of Brincidofovir. Providing Brincidofovir in any of these situations where there is currently limited evidence to suggest that Brincidofovir would be helpful, could very likely place the entire development program at risk."

It is completely possible that a "drug of last resort" can cause more harm than good, and may even kill the patient.

http://mobile.nytimes.com/2013/11/03...?from=magazine

Derek - I'm totally lost here. I was under the impression that adenovirus is an oncolytic virus, and kills cancer cells. So what's going on here? Would they have actually infected him as a cure and it got out of control or what? If he's got cancer, why would they try to kill adenovirus at all?

This is a no-win situation for all involved.

The child - well, that's obvious.

The FDA - if they approve a drug too fast or prematurely, people can die. When that happens they are demonized as corrupt or in it for the money. If it takes longer than people want they're ineffective and need reform. The FDA is far from perfect but I don't think either of those viewpoints are right. This isn't a matter of the drug companies handing them a stack of test results and 24 hours later they are approved or denied. It's a long and drawn out process.

The drug company - I've mentioned this before, but I have worked in this industry for 25 years. Right now, my biggest client is a medium sized drug company. I can tell you without hesitation that FDA approval is a very long and tedious process - and VERY expensive. In this case, the 72 million - while it helps - doesn't cover the cost. Well, not that AND the compassionate use program. Keep in mind that the drug in question has most likely been in development for the last 7 years or perhaps more.

Clinical studies of drugs are very complex. It's about testing the effectiveness of the drug, but also about the safety, the interactions with other drugs, shelf life and degradation, and many other things. And since all drugs effect all people differently, and use all types of other drugs, it takes a very long time.

So, like Kay, I completely understand both sides. If it was my kid, I'd be lobbying too. I tend to think that there isn't a "bad guy" here. Just an unfortunate set of circumstances.

Sal, adenoviruses are not yet associated with any cancers in humans. The patient's own immune system is destroyed in a bone marrow transplant in order to receive new bone marrow. This is why these patients are very susceptible to opportunistic infections, the most serious of which is cause by CMV, which is a herpesvirus.

It is very debatable on the role and severity of adenovirus infections in bone marrow transplant recipients. This is very specialized medical care and the experience varies greatly from one unit to another. Obviously, the unit looking after Josh is highly experienced as well. But somehow, I doubt the company would deny the medicine if they were absolutely sure it would work and cure the child within 2 weeks as the parents claimed.

I saw a number of experimental drugs used during the SARS crisis and it was impossible to prove one way or another whether they worked or not. They might even have caused more deaths because our death rate was much higher than China's whose medical care was not as advanced.

Why are the parents so sure it would work?

I wonder if ganoderma could help. http://carcin.oxfordjournals.org/con...ication_detail

If they gave the drug to everyone on compassionate grounds, NOBODY would get it! WHY? Because new companies(of which is was once one) would lose ALL money and be left wth something potentially WORTHLESS assuming they weren't sued, etc.... to the ground.

As MANY, even TIM and TL in a way, said "There is NO free lunch".

If they had an IRON CLAD WAIVER, that USED to exist, but no longer does, they could use THAT to kill HALF the equation! They could THEN appeal to all those so angry that the kid is not taken care of, and hopefully kill the OTHER part of the equation. THEN, I bet they would do it ASAP, and HAPPILY! It would be a win/win/win/win! ALAS, the waiver doesn't exist, and that charity is drying up. It STILL wouldn't help EVERYONE though!

BTW This can't be done without FDA approval for at least THIS case, that they APPARENTLY have. They STILL need doctors and nurses. They ALSO need to have INSURANCE approval, which they couldn't get because this isn't approved for this general circumstance. Failing that, they OBVIOUSLY need funds from SOMEPLACE ELSE.

BTW... It really is borderline illegal, and potentially could destroy careers, if you prescribe a drug for an offlabel use. Off-label use - Wikipedia, the free encyclopedia SO, from what derek is saying, this would likely not even have been covered under the compassionate use program! If it were to be passed for the circumstance here, a success would be a success and a failure would have been "well, we tried". If it were not for the circumstance, and it failed, it might have been another bad mark on the tests.

YEAH it stinks! ICSM. But this is the ONLY way the industries can survive. It goes on ALL THE TIME. Many have died because of it. Some may have lived because of it. I WOULD say "Blame the government", but with all the GARBAGE that is tried, there IS a legitimate reason for this. Let's say the insurance companies paid for non proven and non approved things, and they failed expectations. If they managed to STILL stay afloat the government, and or insured, would sue the pants off them. Insurance isn't supposed to be there to pay, but to pay for decent care. One thing they will do is actually reject charges that are improper.

Steve

It's a losing situation all around. There are apparently thousands of people who could benefit from this drug and one is being publicized. The rest are not. Their cases are most likely as tragic as the kids' case. Is any one of them more worthy for the drug than the other?

The company suspended the compassionate use program because they could no longer afford it. If this drug is as useful as these parents' think it is, it should be able to come to market, provided all goes well in clinical trials, without going bankrupt.

How could you choose to allow this child the medication and continue to refuse all others who need it? Is anyone suffering more special than anyone else?

I would like to point out another set of facts, just for another dimension
to the problem of "what is" and "what should be" while we think of what
we wish the solutions to this problem would be:


The FDA has 223 field offices and 13 laboratories.
It has less than 10,000 employees, many of them in administrative positions,
and they handle:

Food (everything from safety to advertisements and labels)
Dietary supplements (5-hour Energy and Cracker Jacks with Caffeine)
All human drugs
Vaccines
Medical devices that emit radiation
Cosmetics
Animal drugs
Tobacco products
Maggots
Inspection and regulation of everything they are responsible for
Legal battles
More than 100,000 new requests for clearance and approval every single month

And they are responsible for the health and safety of 330,000,000+ people.



Take a second to let that sink in if you didn't catch it:

225 offices, 13 labs.
<10,000 employees.
Literally millions of responsibilities to hundreds of millions of people,
all at a time when medical science is growing at unprecedented speed.

jenny,

One treatment te book I got suggested garlic!

BTW I am NOT talking about YOU in the next statement....

The utter MORONIC GARBAGE about "clotting problems" is just MORONIC!

By the SAME token, diabetics can't take sugar or insulin! EITHER could KILL them! Timeframes are generally in the seconds to minutes range, so hospitals ARE NOT AN OPTION!!!!!!!!! It needs to be done often within MINUTES! Diabetic people may have to plan out meals, take insulin just before a meal, may have to adjust, and provide for backup incase of hypoglycemia, etc....

So HOW do DIABETICS survive? Well, some people are BORN with diabetes and represent 10-13% of the US. Symptoms of sugar or insulin problems can in minutes range from going crazy to DEATH! Symptoms of sugar or insulin problems can, in weeks to years mean loss of limbs, vision, kidneys, etc....

So I guess the money grubbing people, rather than telling 30 MILLION+ people to go to HELL gave them LOW PRICE FREE ACCESS to devices that can test in SECONDS! By low cost, I mean MOST are in the $25-$35 range, and strips are like $0.50 or LESS!

For CLOTTING, the symptoms are much the same! You could get a clot that could cause a stroke, kidney damage, loss of a leg, etc... You can have a hemorage that could cause death or incapacity. Timeframes could be in the hours to week range, so they THINK hospitals are an option. NOT REALLY!!!!!!

They DO have devices that look and work similar to diabetic machines, but they require THREE WAY approval(Vs. NONE for diabetes), which is NOT transferable! They cost $1500-$2500. The strips cost about $7 each(vs about $0.50).

You CAN'T say "don't take garlic, etc... Suppose YOU slip up? Suppose a vendor does? Suppose you just have dumb luck? Garlic is only ONE thing to watch out for. MANY probably slip up there. Soy beans, cranberry, green vegetables, etc... are some other things. Exercise can ALSO affect it, as well as MANY vitamins and supplements. Suppose you use ON's whey protein isolate, and end up slipping up and taking ISOPUREs whey protein isolate. You're OK, RIGHT? RIGHT!?!?!? I mean ISOPURE is SO pure, practically NO soy, etc... NO PROBLEM, RIGHT? WELL, ISOPURE is now loaded with vitamins, INCLUDING K. So you could get a STROKE! It WOULD be nice to be able to check after such dietary changes. AGAIN, getting checked at a hospital could take DAYS!(Maybe a day to get approval, and they often use labs with a days turn around.) You could have had a few strokes by then. If the need is at work, you may lose pay!

heysal.

So the FDA is supposed to line up people, get doctors, follow the protocol, etc...? DREAM ON!

mike tucker,

Surprisingly, the FDA is *****NOT***** centralized!!!! I had to call like 4 FDA offices to find the ONE person, in Michigan of all places, that knew the details of a certain recall. So it seems more like a loose federation of STATE offices, than branches of a FEDERAL agency!

Midnight,

BE CAREFUL about those "charities"! I spoke to a CHARITY guy at a city council meeting and he was really just a person planning to get donations, FOR A CUT of the take! I told him I had all the stuff and would give it out for FREE, and he never called back!

Anyway, with the FDA, government, and doctors careers on the line, money ISN'T all that is needed. The whole thing for the trial may have been shut down, and who knows what restrictions there are.

Steve

Drug company will give ailing 7-year-old medicine that could save him'

Joe I love your posts man, but I have to comment.
As a Nurse I understand peoples frustration in times like this and wish it wouldn't happen.
We still need to look at the insurance companies point of view.
The drug costs are about 50g's for that med per patient.

They have okayed Company will provide medication for dying 7-year-old Josh Hardy | Fox News it I know after media hit them but you still have to understand there point. They are in a business and with people paying about 500 for a family a month for health insurance it would be impossible to for them to pay for a medication that has only been approved to help alleviate symptoms when morphine can do the same thing.

Yes some say the drug can cure him but it hasn't been proven.

It was sort of like a patient of mine, he had cancer and it hit him hard because he ignored his symptoms so by the time they caught it he was stage four and was given less than six months. A chemo drug could prolong his life for 5 years, he couldn't get Medicaid because you have a time frame from disability and poverty to get it.

He did go on the news and I think St. Jude did pick him up but think about it. If the insurance companies breaks rules to help people even children they can't stay in business.

The only thing we can do is health care reform and the Obama care won't do it. We have to change the pharmaceutical companies, the medical supply company's and the charges from the Doctors and Hospitals.

And then if we go social medicine were in more trouble cause they won't cover something like even with media attention.

So what do we do.

I am impressed the company and the FDA were able to come up with
what appears to be a good and creative solution.

I was wondering about clinical trials programs with similar drugs.
Or out of country.

With my dog that had a nose tumor, I looked at what Colorado State
University's renowned Vet School could do. At the time, what they
were doing did not seem significantly different from the palliative
care my vet was offering.

The ACA actually has clauses to INCREASE COSTS! It has OTHER clauses to DECREASE PROFITS! It really isn't the answer.

You can't REALLY decrease the profits, since many customers will get FAR MORE coverage than they pay for. This means THEY have a deficit, that has to be covered by profits from others that may TOMORROW need coverage. $50,000 IS a LOT! And this is apparently the fourth event this kid has been through. The treatment likely WON'T cure him, but MIGHT simply give him yet ANOTHER chance.

And increasing costs NEVER makes sense. But they do that to milk funds so politicians can pay their friends.

Starting a new trial is good. They can keep this from the old results, hopefully STILL have the other on track, and MAYBE will have a new class of conditions ON LABEL!

Still, I hope this doesn't cost the world a good drug or good company.

Steve

Well, there's a happy (for the family) resolution and a compromise for the company. Hope the clinical trial goes well for them.

Full Story Worth Reading
Company Makes Drug Available To Ailing Boy Following Public Outcry - Forbes

Chimerix maybe got some funding?
Good to see there are some good people at the FDA and companies like Chimerix.

If I were Emperor, I would take health care out of the hands of the politicians,
lawyers, and accountants. I would insist that the best medical brains come up
solutions- with the lawyers, accountants, and insurance types only providing
some needed guidance.

Dan

It's good they're trying to help the kid now but how many other people have to watch this news while a family member with the same problem doesn't get the drug?

I realize they can't save the world but this one person only gets the drug so pharma. can avoid bad publicity which they don't need right before the FDA approves the drug & money starts rolling in.

It's a crazy world we live in...

I hope it will work out for the boy but I suspect that there was another reason why they refused giving out the drug in the first place. It was that they thought that the drug might fail and the publicity generated could stifle further funding and development.

I have not seen a single case of Adenovirus disease in bone marrow transplant recipients while working as a virologist in the UK and Hong Kong. This must be an exceedingly rare disease and the demand for the drug in this situation must be extremely low. Therefore they could have easily accommodated the request without "opening the floodgates."

^^Exactly this, perfectly said.

Good news!
Josh Hardy moves out of ICU | The News Desk
"The deadly adenovirus he had been battling has all but disappeared from his bloodstream after multiple doses of brincidofovir, a drug being developed by Chimerix Inc."