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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."
I guess if you price your drug high enough and restrict its use enough nobody can prove it doesn't work?
So, treating 4000 people for one year would land them a billion dollars? Seriously?
What is the co-pay on that? :)
I've lost a friend to CF and even if this wouldn't have helped her, it is still good news. Anything that can help save lives. Already those with CF live longer and better lives as a whole. I hope some day CF can be treated enough to extend lives to normal ranges.
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?
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."
Early adopters can use it, and the price will be driven down for everyone, just like the cost of sequencing your own genome.
My question is, what drug is more expensive?
"First they came for the slanderers and i said nothing."
Obviously they're playing chicken with the insurance companies, trying to get them to pay out the nose. But would it be better to not publicize these expensive innovations, but only make them available to rich doctors that treat rich patients? Then after the R&D has been recouped, release the knowledge of them to patients of more limited means.
Otherwise, you're just dangling it in front of the poor/ uninsured. Hey, we could keep your kid alive, but neener, neener, neener.
All ideas^H^H^H^H^Hprocesses in this post are Patent Pending. (as well as the process of patenting all postings)
Most people suffering from Cystic Fibrosis do not live past their mid 30s. It's always doubly sad to see both a sick child and parents bankrupting themselves in an attempt to prolong their life.
My girlfriend's 25 year old son right this minute is in an ICU yet again due to CF, and the doctors don't think he's going to make it this time. And if he does there is no way in hell they can afford the drug even if he has the "right" mutation. This news is going to devastate her!
Polio is the ONLY named disease for which medicine has ever proceeded to effect a ' CURE'.
One the cusp of finding a cure for 4% of the population affected by a named disease, you can only see BAD news? My dear friend, finding gene therapy to cure and enable a quality of life you and others like you take for granted is a ' MIRACLE'. The GOOD NEWS is that one form of gene therapy works.
That offers promise that MAN in on the cusp of controlling his genetity if not his destiny, which holds the prospect humanity can cure diseases heretofore it could only suffer.
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...
At that price, it seems more like a 1% thing.
Slow down, cowboy! It has been 4 hours since you last posted. You must wait another few hours.
I bet they put a lot of effort into making sure they didn't cross the well-known psychological price-point of over $300K a year.
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.
Does anyone know how these pills are priced, and what kind of margin these companies make? Those prices seem INSANE.
I would think 294k would make it the MOST expensive drug... I have to imagine there is some chemotherapy that are expensive... but that seems insane... considering that it doesn't cure the problem, just mitigates it.
Yes Francis, the world has gone crazy.
Saddly it's about a decade too late for my college roomie with CF. He was brilliant and very funny, a good friend and a great programer. We're all a bit poorer every day this disease continues to kill.
-GiH
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.
The bad news: this drug will only affect 4% of patients with the disease in the U.S.
So that means this drug will affect 100% of the rest of the worlds population?! Sorry, U.S. citizens I guess there is a geolocator built into the new drug.
Mod parent up. With all the disinformation spattered at slashdot, someone needs to recognize it for what it is.
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.
it really is; glad they're around, but you ever notice they never have a cure? Not even if it was half a mil for a one-time cure; their business is repeat customers with literally life-long lock-in.
Big Pharma: drug dealers in every sense of the phrase.
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?
Human beings do not have cell walls. Cell walls are made of cellulose or chitin,and are typical of bacteria,fungi and plants. Human beings have cell membranes. Otherwise,a good informative summary.
I've got karma to burn so I'm going to ask this here.
The summary refers to the company planning on "developing another drug to try and address that problem." While I understand that most language, and particularly English, is ultimately defined by usage rather than by formal standards, as someone for whom English is not his first language, I find myself flabbergasted by the "try and" idiom. I can understand "try to" as it makes logical sense; I don't see, however, the logic behind "try and" as it implies two different activities, trying, and actually doing. That seems semantically inconsistent, I'd even say gratingly incongruous (to me at least, perhaps because I did not learn English as a child). I also doubt it's what the idiom is semantically implying anyhow. So, can a kind reader explain the logic behind "try and" replacing "try to" in most usage?
"Politicians and diapers must be changed often, and for the same reason."
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."
Clearly letting someone die when there's a drug that can save them is inhumane. But even with a public system there comes a point where a certain treatment is just too expensive.
If we're talking about "humane", perhaps we should look at overall outcomes. What makes more sense...$300K/yr to keep one person alive, or put the money into education and prevention and possibly save multiple lives?
Any info as to just how the price of this goodie was set? Is it really 1e4 times as expensive to produce as , say, Valium?
Am I excessively cynical to think the price was set as high as they thought they could convince insurance companies to pony up?
https://app.box.com/WitthoftResume Code: https://github.com/cellocgw
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?
Not unless you've lived and worked on four different continents. :-)
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."
Of course, because clearly money is the end-all, be-all of existence.
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."
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."
Well, it's not my first inclination, but if it would reduce government spending then OK, reluctantly.
Holy crap! That's less than the government spends killing brown children, paying for blowjobs for senators, and sending killer robots after US citizens!
It's even less than the corporate welfare handouts to mega-corporations that profiteer from selling poor lifestyle choices!
LET'S GO FOR IT!!!! Vote tmosley on the Socialist ticket!
Is because the unfit decided they wouldn't mind surviving either, you darwinist cunt.
Live by your own example and kill yourself.
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."
They then state:
"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"
Note: this is a drug with a limited market, and drugs are expensive to develop, test and produce. A huge number of drugs are developed and fail to win approval. would it be better if this drug didn't exist and the people who benefit from it died at a young age?
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."
"Enlightened self interest" is one of the best jokes I've ever heard.
That, and since this is Slashdot after all, the basis of Sith philosophy...
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."
Yeah, it's expensive. Are many people going to benefit from it over the next five years? Prolly not. But has anyone developed a potential cure for cystic fibrosis before? I kinda doubt it.
It's called innovation, and the people shouldn't whine about it.
But I'm afraid Team Reaper's been making a bit of a rally of late.
The advances you cite:
washing hands before surgery -- Joseph Lister, 1867
vaccinations -- Edward Jenner, 1796
antibiotics -- Alexander Fleming, 1928
Are from a while back, and I've had time to rally.
The US the highest infant mortality of any civilized nation. Fewer babies die in Croatia than the US. Tuberculosis is once again a major concern in American cities. Drug-resistant strains are becoming a real problem, and the doctors in charge are screaming panicky warnings that we may be approaching the end of the Age of Antibiotics. Life expectancies in the US are actually declining, mostly due to heart disease, diabetes and cancer from the industrialized crap we call food. We're eating beef rinsed in ammonia, product that is literally called "pink slime."
Sarah Palin and Megyn Kelly are actually convincing most Americans that healthcare is a frivolous luxury. I love those two!
Sad to say, Mortality Inc. looks like an "BUY" for the forseeable future.
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."
A quarter million plus a year for the drug.
We used to federally fund a lot of such research. Now it's done primarily for profit by corporations. So now it costs a quarter million a year per patient for a CF cure for quite a few people.
At some point even the libertarians have to notice that the actual free market means death for most people when so much research is necessary to discover treatments. The profit necessary to support the private research is too high for most to partake of the benefits.
This is what taxes are for. Go back to the public model, and fund the universities again with copious research grants - and freely distribute the results. Use companies to manufacture and distribute the drugs, no more.
The Age of Reason came about because knowledge was shared freely, and patrons granted money to scientists to discover. This in-house profit-only model is stifling us.
I would say that priority no 1 for a civilization is to make sure people don't just go around killing and looting randomly
And who do you think gets killed and stolen from? The strong? Conan the Barbarian doesn't need a strong police presence.
care about the economy enough that at least a majority wont have to starve
Again, who's in danger of starving? Oh, that's right, Oliver Twist and his little buddies.
When the state is there education and some basic negative rights are in order
And as Oliver Wendall Holmes observed more than once, the people who most need their rights are the people least able to assert them on their own.
So, we shouldn't be worried about caring for the weak, until we've finished caring for the weak? OK, got it.
The reason this upsets me is because of the crusades your likes want to go on in other countries.
OK, "my likes" are ice cream and romantic evenings. If you mean "the likes of me" because I'm arguing we should aid and defend the poor and sick, then I stand in such good company that I blush to be seen with them.
And "other countries?" You mean like Japan, Canada and Sweden? Please, by all means, send me to some more of those hellscapes.
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 cystic fibrosis. I'm 34 years old, and am in pretty good health: I can't see why I don't have at least 20 years left. That's the result of great new drugs coming out.
I'm sorta excited about this med, but it actually doesn't impact me. Why? I, like most CF patients, have the most common genetic variation in both alleles - delta F-508. This new medicine doesn't treat this variation (though the manufacturer actually has a variation of this drug that does treat delta F-508, and it's in the pipeline)
Cost? Yeah, it kinda freaks me out. However, CF is a small enough market that manufacturers typically subsidize treatment, for goodwill benefits. My current batch of meds - the ones I take when I'm healthy and doing good - probably costs over $100K retail. Even when I didn't have insurance, I never went without.
The best thing about a boolean is even if you are wrong, you are only off by a bit.
Do I remember the Reagan years? You mean Alexander "I'm in Charge" Haig and James "The Environment Doesn't Matter Because of the Rapture" Watt? You mean Ronald "We're Launching the Missies in Five Minutes" Reagan, the guy who once raised taxes on the rich?
Reaganomics worked great at inflating the debt and increasing poverty. Morning in America turned out to be overcast and hazy. You can draw a straight line from that infernal speech, go through the point at Gordon Gecko's "Greed is Good" speech, and draw it right through the ruins of New Orleans and Detroit. You can look at the other side and continue the line back through Nixon and Watergate and back through Eisenhower's farewell address, where he tried to warn us about this.
The only thing sadder than the Right's attempt at revisionist history and canonizing Reagan was their attempt to whitewash Nixon.
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."
Because without it, those of us with insurance pay for the care of those without. In this case, the uninsured pay none of the costs for this drug, increasing the price paid by those who are insured.
paintball
Oh, I absolutely agree with you there.
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."
...more and more lucrative "vanity" drugs, and fewer and fewer drugs for anything else.
Funny how that works out.
Come talk to me when the parents of autism and other children's diseases aren't being forced to hold bake sales for even the most cursory of research.
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."
You got my vote.
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."
Treatment... $300k/yr.
Prevention... $1500 for a vasectomy for the carriers.
Why would one need a year's supply?
If it's an actual cure, it changes all the faulty genes in all of the body, and then you are done.
If you need to take it for the rest of your life, it is NOT a cure, but drug, in the junkie sense.
If it is one of those gene therapy drugs, which TFS suggests, I highly doubt that it's the latter. But considering what a bunch of evil crooks the pharma industry is, I can hardly say I'd be surprised...
If you're against coercion then being the citizen of any country is going against that principle because government power of any sort is a coercive power, and you're submitting to it to reap the benefits of having the government exert its coercive force against the other, bigger, more-fit fellow who wants to cave your head in with a rock and take your stuff.
Just because the idea of civilization was perverted into a mechanism by which the haves can efficiently fuck the have-nots and be protected from rightful retribution (read: violent, painful death) doesn't change the original idea of civilization or the social contract.
But seriously, kill yourself, quit posting. In whichever order you prefer.
And that is an abomination.
We as a society decide we need fighter pilots, so we spend millions on their education. We decide we need law enforcement of every stripe, so we pay for thier training. We do this because, the argument goes, they contribute to public safety.
I would argue that the average cop and soldier saves fewer lives in their career than an ER doc does in a week. If we're willing to field armies of men with uniforms and guns to preserve life, why are we not willing to do the same for men with stethescopes and lab coats, when Hippocrates gives us such a better bang for our buck than Leonidas does?
When you are a physician holding actual lives in your hands, I think it's insane that we make you consider mundane bookkeeping while you're doing it. Anyone willing to wade into a storm of blood, screams and excrement to try to pull a life out of it shouldn't have to spend a minute of their lives thinking about how to pay their bills. The second you're willing to saw a human head in half in Gross Anatomy, OK, you're good. You've met your end of the social contract. We should take excellent care of you.
I don't think for one second you're overpaid. I think you're overworked, getting molested by insurance companies and undervalued. I want to get you help and support.
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."