Cystic Fibrosis Gene Correction Drug Approved by the FDA 264
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."
Nonetheless a good day (Score:1, Insightful)
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.
Re:Nonetheless a good day (Score:4, Insightful)
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.
Re:So, treating 4000 people (Score:5, Insightful)
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.
Re:Cost (Score:5, Insightful)
Re:So that's not much good (Score:5, Insightful)
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 [wikipedia.org]?
Re:Shit, shit, shit!!! (Score:4, Insightful)
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.
Re:So, treating 4000 people (Score:4, Insightful)
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.)
Re:So, treating 4000 people (Score:2, Insightful)
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.
Re:Nonetheless a good day (Score:4, Insightful)
Re:Nonetheless a good day (Score:4, Insightful)
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.
Re:Nonetheless a good day (Score:5, Insightful)
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?
Re:So, treating 4000 people (Score:5, Insightful)
TIme, GS, just give it a little time (Score:5, Insightful)
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.
I was wondering when Ayn Rand was going to show (Score:4, Insightful)
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.