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Cystic Fibrosis Gene Correction Drug Approved by the FDA
tguyton writes "The good news: the FDA just approved the distribution of the first drug to treat the underlying cause of Cystic Fibrosis, called Kalydeco by Vertex Pharmaceuticals. The bad news: this drug will only affect 4% of patients with the disease in the U.S. From the article: '[Affected patients] with the so-called G551D mutation have a defective protein that fails to balance the flow of chloride and water across the cell wall, leading to the buildup of internal mucus. The vast majority of cystic fibrosis patients have a different genetic defect, in which the protein does not reach the cell wall. Vertex is developing another drug to try and address that problem. Study data for that drug is expected later this year.' Hopefully the research involved will be applicable to finding treatments for other genetic diseases."
Further bad news: "...executives said Kalydeco would cost $294,000 for a year's supply, placing it among the most expensive prescription drugs sold in the U.S."
What is the co-pay on that? :)
Good lord, we are animals not plants. There is no such thing as a "cell wall" in our cells! Call it what it is: the cell membrane.
Pedantic? Yes, but the definitions are precise and are intended to be used precisely. Journalism like this makes me want to gouge my eyes out; a single high-school biology class teaches cell wall vs. cell membrane!
Just so no one gets confused, this molecule goes by 3 common names VX-770 and Ivacaftor and Kalydeco
http://en.wikipedia.org/wiki/Kalydeco
There are not three separate drugs for the same problem etc.
"Science flies us to the moon. Religion flies us into buildings." - Victor Stenger
Can anyone explain a bit about how this drug works? I understand CF is caused be a genetic "error", but is this an exon skipping drug (similar to what they're working on with muscular dystrophy) or is this something different?
Or given only 4% of 1 in 2000 need it, we could treat everyone if we all chipped in 2c a day.
This won't save lives. at $294k per year for treatment it will bankrupt people, leading to an increased stress in their entire family and the suicide and other health risks associated with that. It could actually cost more lives through these effects than it could ever save.
Communist!
Haida Manga
http://www.forbes.com/2010/02/19/expensive-drugs-cost-business-healthcare-rare-diseases.html
Alexion Pharmaceutical's Soliris, at $409,500 a year, is the world's single most expensive drug. This monoclonal antibody drug treats a rare disorder in which the immune system destroys red blood cells at night. The disorder, paroxysymal nocturnal hemoglobinuria (PNH), hits 8,000 Americans.
Elaprase ($375,000 per year) treats an ultra-rare metabolic disorder called Hunter's syndrome. Just 500 Americans suffer from the disease, which causes infections, breathing problems and brain damage.
Naglazyme from BioMarin Pharmaceuticals treats another rare metabolic disorder and costs $365,000 a year, according to investment bank Robert W. Baird. Viropharma predicts that sales of its Cinryze, a treatment to prevent a dangerous swelling of the face, will increase from $95 million last year to $350 million several years from now. The drug costs an estimated $350,000 a year.
From my understanding, those suffering from CF in California generally have their bills picked up by the state since no insurance plan could ever afford to treat CF patients. I believe this is under the state's Genetically Handicapped Persons Program.
But not having CF myself, I'm certainly no expert on the cost of care for it...
I don't know if it will be in time, but the cost might be as big an issue as you expect
"Wysenski said Vertex would provide the medicine for free to people with no insurance and household income of $150,000 or less. The company will also cover 30 percent of copay costs for select patients who have insurance."
I hope for the best.
I stole this Sig
That would seem to depend on how much of the price tag is production costs and how much is "Because we can, would you prefer to suffocate on your own mucus, sickie?"...
Pharmaceutical manufacturers certainly aren't known for their charitable pricing; but the economies of scale for a specialty drug with a few thousand users have got to be pretty lousy.
When the drug company provides some of your co-pay, they are in effect, jacking the cost of their product up, charging the insurance company, and giving some of that money back to the patient. The overall effect is mo money, mo money, mo money for the drug company.
The insurance companies don't like this. They are already trying to stop drug company co-pay cards. Co-pays incentivize the patients into choosing cheaper drugs. Co-pay kickbacks from the drug companies mess with this. Expect the insurance companies to fight back. The simpliest way to do this is to declare the drug to be experimental.
All ideas^H^H^H^H^Hprocesses in this post are Patent Pending. (as well as the process of patenting all postings)
From a radio report, apparently they will also provide it free for uninsured people who earn less than $150k, so they'll be treating more than they'll be getting paid for.
As I noted above, the cost is only for those with insurance - they'll treat the uninsured free if they make less than $150k. So while it might cause insurance premiums to rise slightly, it shouldn't bankrupt anyone.
Polio is the ONLY named disease for which medicine has ever proceeded to effect a ' CURE'.
What? Polio is still around, while small pox has been eradicated in the wild. Plus, we have cures for Pellagra, Scurvy, and all sorts of other named disease...
Me thinks there be a troll here...
WARNING! This girl exceeds the MAXIMUM SAFE standards established by the FDA for BRATTINESS
Posting anon 'cause I've already modded.
My wife has MS and takes Tysabri (Natalizumab).
It started off costing us (and the insurance co.) $6000 a month. However Bio-gen began a copay/deductible assistance program after they saw how the price was keeping people from access to the drug. This has made our costs manageable. And I'm sure it's helped hundreds, if not thousands of people.
I bet (and hope) the same thing will happen to this drug.
It also isn't much good if you're not a plant or bacterium.
[Affected patients] with the so-called G551D mutation have a defective protein that fails to balance the flow of chloride and water across the cell wall
The vast majority of cystic fibrosis patients have a different genetic defect, in which the protein does not reach the cell wall.
May I oblige?
My brother lost his 18 year old son to cancer.
When your kid is that sick, there are no problems beyond their health. Call it a common human failing.
John McAfee 'It was like that time I hired that Bangkok prostitute; to do my taxes, while I fucked my accountant'
The fact that there's a market of only 4000 for it is why the per-unit cost is so high. It isn't about the cost of manufacturing the drug (at least not primarily). It's because they need to charge enough to recoup their expenses developing and testing the drug. It's a necessary part of a profit-driven medical research system. (A possible solution is left as an exercise for the reader.)
http://alternatives.rzero.com/
We spend ~$30billion a year on research in the U.S. on the NIH, so a partial solution is already in place.
The other thing to keep in mind is this drug is only highly priced for the next 20 years. After that the generic versions will be cheap, so future patients will benefit hugely. That's the beauty of the patent system. It hasn't been outrageously extended to hell like the copyright system has.
Oh, stop. The Party line here is to bash on big evil pharma until all drugs are produced for free by unicorns from fields of biodiverse flora.
Turn in your geek badge. You're going all mainstream with your facts and reality and pragmatism.
Just be careful that the "market" doesn't turn out to be a whole lot of people tired of being poor, sick and with no prospects of moving up taking out their rage on the rich and healthy people who keep talking about how the market will fix everything.
You people ought to investigate why we have a social safety net. Only half of the reason is to protect the poor and the sick. The other half is to protect the rich from the poor getting their pitchforks out.
Those who can, do. Those who can't, sue.
The best way to deal with Cystic Fibrosis is for Carriers to go the IVF route so they don't pass the gene on to their kids. All this "CURE" will do is make it so that CF isn't as lethal making it propagate out as a benign disease when in truth it will just be making a population dependent on drugs to live. A real Cure would be a gene therapy that removes the defect from every cell in the patients body so they don't have to continue taking it for the rest of their life, and have no risk of passing it on to their kids.
Interesting. It seems the most expensive drugs are expensive because they treat diseases that are rare, thus they don't get the advantage of spreading the research cost out over many sales.
"First they came for the slanderers and i said nothing."
Under the Affordable Care Act, all health insurance plans are requried to have an out-of-pocket maximum of at most $5,950 for individuals and $11,900 for families.
Then we better just toss the drug away. Right? What is your point? That it is expensive? Ok. That's great. A lot of stuff is expensive. Are you saying we shouldn't pay for medical research and force people to research for free? I'm not exactly sure where you're coming from or if you're just complaining about progress because it's not yet enough to help everybody who needs it. If we socialize our medicine we still need people to research and we still need to feed those people and it still won't be enough to help everybody who needs it, so we will have to research more and more.... we will always lose the healthcare battle no matter what your economic system is. People get sick and die, and if we spend all day every day trying to avoid it, it will still happen. So again, what is your point?
If I can just reach out with my words and touch a butthole, just one, it will all be worth it.
So you'd rather live in the times of strife where the poor were abjectly poor (and rather permanently poor), where the rich feared the poor at every turn, and where revolutions were fairly regular? You should put down your rose-colored glasses. Those times sucked for everyone.
Those who can, do. Those who can't, sue.
Cystic Fibrosis is a genetic disease that is well characterised. Remember, an ounce of prevention is worth a pound of cure. We'd prevent more deaths if we attempted to remove the defective allele from the gene pool than trying to cure it after the fact.
What we need is a place where prospective breeders can go and test themselves for known problematic alleles. We need a place where people who have problematic alleles can go and have their potential offspring tested, and select against the defective alleles. We need these services, and we should provide them for free because of the high cost in both money and suffering of these genetic diseases.
Give me Classic Slashdot or give me death!
Vertex has a very generous patient assistance program for this drug. It's *free* for those without insurance and who make less than $150k/year, and for others there's a generous co-pay assistance program. This drug will be available for everyone who needs it. We've spent 10 years and I don't even know how many hundreds of millions of dollars developing this drug, and helping save lives is what keeps us motivated every day.
---
of course, I don't speak for Vertex.
Thank the Flying Spaghetti Monster and his noodly appendages, I live in Canada.
Hopefully the researchers in the evil ignorant US will share what they've learned with the enlightened Canadians.
This is why Congress passed the Patient Protection and Affordable Care Act in 2010. This law "[p]rohibits individual and group health plans from placing lifetime limits on the dollar value of coverage, rescinding coverage except in cases of fraud, and from denying children coverage based on pre-existing medical conditions or from including pre-existing condition exclusions for children. Restricts annual limits on the dollar value of coverage (and eliminates annual limits in 2014)[.]" It also "[c]reates a temporary program to provide health coverage to individuals with pre-existing medical conditions who have been uninsured for at least six months." The guaranteed availability of insurance provision will be implemented in 2014.
You should check to make sure that your insurer is not imposing an annual limit lower than what is allowed. As of 2014 they won't be allowed to have one at all.
Check it out.
The good news: the mistake was made by the summary author and does not appear in TFA.
I think it's worth pointing something out: it's obviously not that we're incapable of harnessing the resources to generate the necessary research and manufacturing to produce new drugs in quantities to help everybody. We've done the research, and we've got the industrial capacity to do absolutely incredible things.
It's the same with food and shelter. Our planet and the industries we've built on it are perfectly capable of providing every last one of us with a roof over our head and enough food to keep us from starving. Instead farms sell grain to big beef farms while their neighbors are hungry. It's absolutely insane.
If, as a species, we are capable of healing all the sick, housing all the homeless and feeding all the hungry...why don't we just fucking do it? It's because we're bogged down in the game of tending to our whole contrived economic machine, instead of the game of tending to our real standards of living.
It would probably even be more productive in the long run, because the economic contribution of a person who is healthy, housed and fed is 9 times out of 10 going to be significantly higher than a starving homeless person dying in the street of a treatable disease.
We pay a lot of lip service to "freedom." We even spend an ungodly amount of money already, through our military, to fight for a few particular types of freedom. But what about the freedom to buy food at a price you can afford? Or the freedom to have a roof over your head at a price you can afford? Or the freedom to buy medicine at a price you can afford? If the freedom our military fights for is worth the fortunes we spend on it, aren't those simpler freedoms worth a little something too?
Porquoi?
Yaaay for eugenics! We'll have Übermenschen in just a few generations!
I for one welcome our well-bred genetically superior overlords!
Welcome to the Panopticon. Used to be a prison, now it's your home.
It starts with cystic fibrosis and ends with a tendency to vote for whatever party is in the minority at the time they mandate yet another test. You can't keep on weeding out "unwanted" genes from the gene pool, because sooner or later, the decision what genes are unwanted will be used to create an "Uebermensch".
It may be desirable to get yourself tested genetically if you want to have some insurance your child doesn't suffer from genetic disease. However, once you provide these services, it will be hard to get insurance, a job and what not if you or your parents don't use them. Even if they are legally not enforced, capitalism will enforce them anyhow. Discrimination of certain genes will make a paria out of probably over 50% of people, making it (practically) illegal for them to breed, making it harder to get loans, insurances and certain jobs. The USA economy has enough parias as it is, please don't make it worse by adding gene testing as a discriminator.
I was promised a flying car. Where is my flying car?
If by "now" you mean in the year 2014, you might have a point.
Indeed, this assumption may already be included in their stated list price. "We know that if 100% of the people who need it could afford it, we would have to charge x to recoup costs and make our desired profit margin. Since we'll be subsidizing y% of users, we will need to actually charge z% of x (where z is greater that 100)."
I could point out that California isn't bankrupt because of healthcare costs. I could do a whole excrutiatingly exhaustive review of just why California's revenues got strangled. I won't, because it won't matter to you.
Since you're doing your thinking emotionally -- the shrinks nickame it the "Just World Hypothesis" -- let me see if I can counterbalance the fear you're suffering from by adding a different one.
If a civilization has any obligations -- any at all -- then caring for the vulerable is the first among them. Any system that can't -- or won't -- care for the very young, the very old and the physically infirm doesn't deserve to continue. I know Sparta sounds cool, but I'm kinda glad a people who threw babies against rocks and committed murder for sport aren't around any more. In fact, I would argue that their brutality is precisely WHY they're not around any more. People who are working together tend to weather crises far better than a group of jackasses standing around screaming "Only the Strong Survive!" Teamwork, you know? Maybe your coach mentioned it? Remember John Wayne screaming back, "That's WHAT I got?" No? OK. didn't think so.
I'm old enough now to have watched a few of my friends and some of my family die. Heart disease is bad. Diabetes is worse. Cancer is flat-out evil. Real "We had to pick up a knife to save you" surgery is damn near the same thing as surviving a stabbing. I know, I know, those are just words to you. Let me put it in terms you may understand. You'd much rather be eaten by a vampire than succumb to cancer. You'd prefer any videgame death to what most fatal diseases have in store for you.
Here's where I'm going to pull back my hood and tap you on the shoulder with a long, bony finger. Eat a perfect diet. Exercise all you want. Revel in whatever gifts youth and good health can provide. I'll still be waiting. I got Steve Jobs. I got Feynman. I got Newton. You think I'm going to miss you? At 35, you'll notice you've lost a step. At 45, you and your doctor will have a little chat. At 55, those chats become discussions. At 65, you begin long talks about the options you have remaining.
Believe me when I say they'll dwindle.
And this is the best possible outcome, assuming some little patch of slippery ice has't gotten you first.
So while you're sitting there blithely saying we should kick the sick and the weak to the curb, I'm smiling. Because I know you'll be among them soon enough.
And people like you always whine the loudest when I come.
He put his boots up on the table and made a face. "The sig," he smirked. "You can waste your life in search of the sig."
The average cost to market for a new drug is now $1.4b (using the aggregate of all developments method) and 7 years from patent to commercially viable drug. This gives them 3/4 years to recoup their original investment so, as you mentioned, having such a small user group has a massive impact on price point.
We spend ~$30billion a year on research in the U.S. on the NIH, so a partial solution is already in place.
The other thing to keep in mind is this drug is only highly priced for the next 20 years. After that the generic versions will be cheap, so future patients will benefit hugely. That's the beauty of the patent system. It hasn't been outrageously extended to hell like the copyright system has.
It's worth pointing out that part of the calculus that goes into pricing a drug has to do with the fact that drugs rarely enjoy all 20 years of patent protection, due to the fact that the invention of the drug usually occurs in the R&D phase which predates the clinical trials, approvals, and manufacturing scale-up. The average effective patent life (i.e. the period during which a drug is actually for sale) is 7 to 12 years, so prices tweaked to compensate. The flip side is that it really discourages treatments for diseases that affect very small portions of the population,since you cannot count on recouping costs over long periods of time to compensate for the small patient pool. This is partially addressed by the Orphan Drug Act, but more often than not this is where charities funding disease-specific research really play a crucial role.
Technically the cure for malnourishment conditions also ends up being a lifelong treatment. You just can't win.
I read the internet for the articles.
I have heard of egregious prices for complex new drugs before. I had a roommate who received a tumor-necrosis-factor inhibiting drug for free, that cost thousands of dollars a month. I Do Not want to be inflammatory, nor to impugn the reputation of a hard-working company that sounds very nice and ethical, but I have been wondering about this huge-price+free-for-low-income bracket for a while now, and I have a question: Do the companies that offer these coupons get a tax write-off for the price of the medication they give away?
Well, at least it made an impact on one of your organs. :-)
Choices will be made.
Absolutely. And when we quit paying for billionaire tax cuts by skipping out on grandma's blood pressure medication, I'll be more amenable to hearing about them. Laffer and Stockman have both publicly recanted, and Buffet's with me on this one.
To expect a huge government organ could handle it without turning into a pit of corruption and graft is beyond delusional.
Yeah, because clearly the United States military has never accomplished anything of value. You're making a sophomore's argument for anarchy. Any government large enough to be effective is too large to work.
He put his boots up on the table and made a face. "The sig," he smirked. "You can waste your life in search of the sig."
OK, you're arguing that we need to do some form of triage because we don't have resources to go around.
I don't believe this is true. When Merck Pharmaceutical tells you it's going to cost tens of millions, you should think of that in the same way you hear cops talk about the street value of the drugs they've seized. It's a self-serving, nonsense number. The same companies that scream "It's horribly expensive to make!" scream "It's not fair to make us compete against the government!" when we threaten to make it for ourselves. Since this is Slashdot, compare the situation to when various municipalities have tried to set up their own ISP. The same telcos that scream they have to charge billions to serve a city suddenly begin screaming that it's not fair for us to find alternatives. I promise you, we'll find we can manufacture this drug for a sliver of what the drug company is claiming.
BUT, BUT, BUT RESEARCH COSTS! I hear you scream. In case you haven't been paying attention, research in this country is done with recycled tax dollars. We The People have already paid the research costs, and if Merck and GalaxoSmithKline want to argue that, then all they have to do is stop taking Federal dollars.
They won't, of course.
Secondly, you're arguing that it's immoral to take money by force from one person to pay for something for another.
You know, I kind of like this argument. I'd love to make sure not one more penny of mine went to finance Gitmo. But, OK, Death and Taxes. We set up governments, and we pay for them by taxes of some kind. You actually are free to opt out of paying these taxes if you wish. If you don't feel like paying taxes any more, all you have to do is leave, and then tell a representative of the US government that you are no longer interested in being part of the United States. It's easy. Of course, you'll find very shortly that it's cheaper to pay taxes than it is not to pay taxes, but maybe to can join all the other John Galts on that floating ocean platform they're trying to build -- you know the one that's not going to have any building codes, the one we're going to nickname "Rapture" when it finds the bottom of the ocean.
Finally, you're arguing we can't fix everything. Maybe not.
But we can fix orders of magnitude more than we currently are.
He put his boots up on the table and made a face. "The sig," he smirked. "You can waste your life in search of the sig."
Laffer has been proven correct thus far
Yeah, except that not even Laffer agrees with that any more. He's been backpedaling furiously from a bad theory made 30 years ago that's been empirically, wondrously disproven over the past ten. Abondoning his little napkin sketch was the only way he could retain a shred of academic credibility.
You simply cannot provide everyone with all the healthcare they want
Sure, sure, sure, just answer me this.
Why do we give federal subsidies to Harvard Medical School?
Because they threaten to train more doctors if we don't. We grant them a federal subsidy to restrict admission because the American Medical Association says that too many doctors in the field will lead to a lower standard of living for doctors.
And it's not just Harvard. Every medical school is granted subsidies to restrict enrollment.
Hmm. Seems odd, doesn't it? We can't find enough resources to meet America's medical needs in much the same way that companies can't find enough American engineers to fill all the jobs.
But let's assume those nonsense numbers are true. Let's try this. How about we divert all of the resources from the current War on Drugs and War on Terrorism and redirect those trillions of dollars to a War on Illness? Surely we can agree that a few more Harvard-educated medical doctors would do more good then a few thousand more TSA agents.
How about we find all the kids bright enough to become doctors and sponsor them through medical school? How about we devote research dollars to more than just making sure rich guys can screw their trophy wives?
How about we agree with all the drugs companies that government is wasteful and inefficient, and that we welcome their competition when we start opening drug factories they same way we open utility companies. How about when they start whining about horrendous research costs, we tell them we couldn't agree more, which is why we're going to ask them to pay back all the money we gifted to them over the past six decades.
How about we take Manhattan and Apollo project resources for the next 20 years and apply them to healthcare? Then let's see if your nonsense about "We can't afford to take care of our own" falls apart into the same pile of bull that the Laffer Curve and Supply-Side economics did.
He put his boots up on the table and made a face. "The sig," he smirked. "You can waste your life in search of the sig."
I have CF. None of the meds I need I've ever done without, even when I had no insurance. Granted, they aren't quite as expensive as Kalydeco, but we're talking over $100K a year in meds. The pharmas tend to see to it that specialized drugs for small markets get to the patients: consider it a loss leader to benefit public perception and stock price.
The best thing about a boolean is even if you are wrong, you are only off by a bit.
From a radio report, apparently they will also provide it free for uninsured people who earn less than $150k, so they'll be treating more than they'll be getting paid for.
So if you earn $151,000/yr you're expected to pay for a medicine that costs almost twice your entire yearly income? Okaaay....
You know what's hilarious? We spend $700 billion/year on the "defense" budget vs. $30 billion/year on the NIH. I find that hilarious. All these stupid diseases could be cured in 15 years if we reversed those numbers.
And if you made all of those discoveries available patent-free, nobody will will want to attack you. You're providing $700 billion a year in free medical research. Why kill the goose that lays the golden eggs?
Of course it means you can't muck about in the afairs of other countries. That's no fun. Stupid idea...
My question is how many people with a salary of approximately $150k do not have health insurance?
Doesn't this only exemplify how very fucked up the entire healthcare system in this country is? Insurance isn't something you should have to have to be able to afford medical treatments, because everyone is not guaranteed to be able to get insurance to begin with. I don't believe any insurance companies would jump at the chance to sign up a new customer with a pre-existing CF diagnosis. Medical care should be at prices people can half-way afford without insurance. Otherwise all you really have is medical providers milking insurance companies for every dollar they can (raising premiums for everyone, healthy or not) and the uninsured are just SOL.