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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."
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
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...
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.
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.
CF is caused by defects in the CFTR protein, an ion channel on the surface of many cells, including the cells that line the respiratory tract. Basically, it creates an osmotic potential by moving chloride and other ions from the cell to the outside, so water flows out of the cell into the mucous in the airways, making it less viscous. I'm an anaesthetist, not a respiratory physician, but as far as I understand, in the F508 mutation (most common ~70%), CFTR doesn't even make it onto the cell membrane. In the G551D mutation, CFTR reaches the cell membrane, but degrades more rapidly than normal. Ivacaftor acts to increase the length of time the faulty protein stays on the surface until it's degraded. Hence why it's of no benefit in the most common mutation.
Depending on your insurance, you can also hit a "lifetime cap"(it isn't a 'death panel', because it is privately administered) where the insurer (further) controls their risk by simply halting all payments above a certain cost. These tend to fall in the 1-5million range, so they'll buy you a fair few packets of penicillin and casts for your little-league injuries; but 300k/year + other medical bills could mount rather quickly...
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 cousin died two years ago after fighting CF for 32 years. My aunt hit her annual out of pocket cap no later than Jan 31st every year.
At least for new policies, the Affordable Care Act (Obamacare) made lifetime maximums illegal.
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.
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?
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."
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."