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Cystic Fibrosis Gene Correction Drug Approved by the FDA

Unknown Lamer posted about 2 years ago | from the living-is-expensive dept.

Medicine 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."

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So that's not much good (0)

Anonymous Coward | about 2 years ago | (#38892393)

I guess if you price your drug high enough and restrict its use enough nobody can prove it doesn't work?

Re:So that's not much good (5, Insightful)

Anonymous Coward | about 2 years ago | (#38892999)

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] ?

So, treating 4000 people (0)

Anonymous Coward | about 2 years ago | (#38892415)

So, treating 4000 people for one year would land them a billion dollars? Seriously?

Re:So, treating 4000 people (3)

bWareiWare.co.uk (660144) | about 2 years ago | (#38892533)

Or given only 4% of 1 in 2000 need it, we could treat everyone if we all chipped in 2c a day.

Re:So, treating 4000 people (2)

LearnToSpell (694184) | about 2 years ago | (#38892621)

Communist!

Re:So, treating 4000 people (1)

Anonymous Coward | about 2 years ago | (#38892681)

That sounds like universal health care.

Re:So, treating 4000 people (0, Flamebait)

tmosley (996283) | about 2 years ago | (#38892973)

Let's do that with all disease, including the diseases of age, and those caused by poor lifestyle choices!

We could treat them all if we only chipped in $700 a day!

Re:So, treating 4000 people (0)

Anonymous Coward | about 2 years ago | (#38893349)

It's a GENETIC disease, not old age or poor lifestyle choice.

I'd hate to have to tell my kid: Well, honey I'm sorry but you'll have to die, daddy is too poor to pay 300k a year or get a tranplant... On the bright side, they have free counseling for acceptation and you'll get to see grandpa again... Not!

Thank the Flying Spaghetti Monster and his noodly appendages, I live in Canada.

Re:So, treating 4000 people (0)

tmosley (996283) | about 2 years ago | (#38893387)

Yeah, but we can't not help those other poor people as well! You are a cruel, terrible person if you think we shouldn't help everyone that we can until our entire civilization is ground into dust under the ever accumulating weight of those who find ways to exploit any and all coercive systems.

Re:So, treating 4000 people (5, Insightful)

fuzzyfuzzyfungus (1223518) | about 2 years ago | (#38892871)

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.

Re:So, treating 4000 people (4, Informative)

b0bby (201198) | about 2 years ago | (#38892911)

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.

Re:So, treating 4000 people (2, Funny)

Anonymous Coward | about 2 years ago | (#38893375)

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.

Re:So, treating 4000 people (3, Insightful)

tverbeek (457094) | about 2 years ago | (#38893069)

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 (2, Insightful)

Anonymous Coward | about 2 years ago | (#38893363)

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:So, treating 4000 people (1)

Rockoon (1252108) | about 2 years ago | (#38893537)

We spend ~$30billion a year on research in the U.S. on the NIH, so a partial solution is already in place.

Thats fine, except for covering testing costs as well. How many millions does it take just to get the FDA to allow? Well lets see what I found out from Google:

$802 million [healthcare-economist.com]

Re:So, treating 4000 people (0)

Anonymous Coward | about 2 years ago | (#38893257)

Posting Anonymously because I've already modded another post up in this discussion.

You have to understand how much it costs to make these drugs in the first place. Scientist salaries, lab equipment, specialized small-batch production, dozens of studies, FDA approval, etc., etc. Treating 4,000 people won't give the company 1 billion in profit (assuming they actually get paid). It will be 1 billion going toward paying the bills.

Cost (2)

mikehilly (653401) | about 2 years ago | (#38892453)

What is the co-pay on that? :)

Re:Cost (1)

gandhi_2 (1108023) | about 2 years ago | (#38892523)

The good news is you will meet your "out of pocket cap" in a few hours.

The bad news is your insurance won't approve of the drug until the generics come out... some time in 2064.

Re:Cost (4, Informative)

es330td (964170) | about 2 years ago | (#38893117)

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.

Re:Cost (0)

Anonymous Coward | about 2 years ago | (#38892551)

I'm sure the insurance companies will brand it "experimental" for the next few years and refuse to cover it at all, then cover it at their new 50% copay tier that a ton of companies have been creating recently.

Good news, though, if your loved one is suffering from Cystic Fibrosis, you may (4%) be able to go through bankruptcy and keep them alive instead of going through bankruptcy while they slowly die.

Reminder: a large number of people going through medical bankruptcy had insurance. Turns out that your 20% share of millions of dollars is still more than most people can afford for medical care.

Re:Cost (5, Insightful)

fuzzyfuzzyfungus (1223518) | about 2 years ago | (#38892935)

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...

Re:Cost (5, Informative)

esrobinson (1028500) | about 2 years ago | (#38893487)

At least for new policies, the Affordable Care Act (Obamacare) made lifetime maximums illegal.

May be free in California (4, Informative)

rwade (131726) | about 2 years ago | (#38892771)

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 [ca.gov] .

But not having CF myself, I'm certainly no expert on the cost of care for it...

Re:May be free in California (1)

gsgriffin (1195771) | about 2 years ago | (#38893207)

Which States are bankrupt and why?

Nonetheless a good day (1, Insightful)

Anonymous Coward | about 2 years ago | (#38892471)

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 (3, Insightful)

Nadaka (224565) | about 2 years ago | (#38892585)

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:Nonetheless a good day (0)

Anonymous Coward | about 2 years ago | (#38892729)

Typically companies which make very high cost drugs for orphan indications, will give drugs to sufferers who have no insurance. Note, however, that the customers of the insurance companies make up the difference in higher premiums.

Re:Nonetheless a good day (1)

Nadaka (224565) | about 2 years ago | (#38892813)

Even with an insurance 80% co-pay, 1 year of treatment will cost the person nearly $59k, that is more than the average persons gross income for a year, every year.

Re:Nonetheless a good day (0)

Anonymous Coward | about 2 years ago | (#38892883)

Does your insurance have a $60,000 deductible?

Re:Nonetheless a good day (1)

Nadaka (224565) | about 2 years ago | (#38892977)

No, but the deductible is payed before the 80% co pay kicks in. Mine also has a 100% insurance paid level that kicks in after a while, but that isn't available in all insurance plans.

Re:Nonetheless a good day (1)

Anonymous Coward | about 2 years ago | (#38893091)

You win. New drugs are too expensive and we should just stick with the technologies we already have. Forget I said anything.

Re:Nonetheless a good day (4, Informative)

esrobinson (1028500) | about 2 years ago | (#38893527)

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.

Re:Nonetheless a good day (3, Informative)

b0bby (201198) | about 2 years ago | (#38892945)

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.

Re:Nonetheless a good day (1)

Nadaka (224565) | about 2 years ago | (#38893181)

As I said above, even with an 80% co-pay by the insurance company, this will consume more than 100% of the income of the average American. The only people this won't bankrupt are either fantastically wealthy or who have insurance that covers 100% of the cost for the rest of their life. Merely being upper middle class with standard insurance won't be enough to keep you from being ruined.

Re:Nonetheless a good day (3, Insightful)

smelch (1988698) | about 2 years ago | (#38893541)

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?

Re:Nonetheless a good day (3, Insightful)

Hatta (162192) | about 2 years ago | (#38893673)

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.

Cell MEMBRANE (5, Informative)

Anonymous Coward | about 2 years ago | (#38892477)

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!

Same thing as VX-770 and Ivacaftor (4, Informative)

vlm (69642) | about 2 years ago | (#38892491)

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 [wikipedia.org]

There are not three separate drugs for the same problem etc.

Re:Same thing as VX-770 and Ivacaftor (3, Interesting)

the gnat (153162) | about 2 years ago | (#38893155)

Just so no one gets confused, this molecule goes by 3 common names VX-770 and Ivacaftor and Kalydeco

I asked a scientist I know at a Big Pharma company why there are always multiple names. His response was (paraphrased): "There's an entire department at our company full of people who come up with names for drug candidates - one name that's recognizable and easily pronounced which becomes the trademarked brand name, and another that's hard to pronounce or remember that becomes the official compound name, which is what the generics will eventually use." (I think he was exaggerating about it being an entire department, but I could be wrong.)

If you pay any attention to biomedical literature, they always use the compound name - thus "Prozac" will always be referred to as "fluoxetine", "Gleevec" is "imatinib", and so on.

Exon Skipping? (2)

Nos. (179609) | about 2 years ago | (#38892505)

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?

Re:Exon Skipping? (2)

PenguinX (18932) | about 2 years ago | (#38892847)

According to NPR, Kalydeco, "works by helping to fix one defect in the protein that causes the disease." Unfortunately the way the drug works is also very specific, and won't work for all sufferers NPR also reports that it will "only work for about 1,200 patients in the U.S.". Now, being that a cursory Internet search says that there are about 30,000 sufferers in the USA, it's pretty clear that Kalydeco is just a step in the right direction, at least from a medical and research perspective.

http://www.npr.org/blogs/health/2012/02/01/146166743/cystic-fibrosis-drug-wins-fda-approval?ps=sh_sthdl [npr.org]

Re:Exon Skipping? (5, Informative)

Cyclizine (2558090) | about 2 years ago | (#38892923)

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.

That's not such bad news (1)

phantomfive (622387) | about 2 years ago | (#38892517)

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?

Re:That's not such bad news (1)

vlm (69642) | about 2 years ago | (#38892617)

My question is, what drug is more expensive?

Idursulfase for the win. Interesting from a biochemical production standpoint.

http://en.wikipedia.org/wiki/Idursulfase [wikipedia.org]

You can get into definition battles, whats more expensive to society, a quarter million for one dude who actually needs it, or 100K unneeded $10 lorazepam prescriptions for people who don't really need it ...

Re:That's not such bad news (1)

tmosley (996283) | about 2 years ago | (#38893103)

"Need" doesn't justify the collective burdening of others. Sure, "we" could easily absorb the cost of one of these types of things, but what happens when there are thousands, or millions of similar cases? Who gets to decide who gets access to everyone's money? It is a system that breeds corruption, and will cause eventual collapse, and greater harm to most of the participants.

As cruel as it sounds, it is the market that must decide. It is the only way to keep things anywhere close to fair for EVERYONE, rather than making things extremely "fair" for a chosen few.

Re:That's not such bad news (0)

Anonymous Coward | about 2 years ago | (#38893239)

It is the only way to keep things anywhere close to fair for EVERYONE, rather than making things extremely "fair" for a chosen few.

LOL, how long have you been asleep?

Re:That's not such bad news (1)

tmosley (996283) | about 2 years ago | (#38893351)

Don't take that to mean that I don't think that the current system isn't like that. I decry such things where ever I see them, and I see them everywhere these days. Looking back, I see them going back a long time too.

Re:That's not such bad news (2)

NeutronCowboy (896098) | about 2 years ago | (#38893379)

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.

Re:That's not such bad news (1)

tmosley (996283) | about 2 years ago | (#38893437)

But we need them to get their pitchforks out. The system is corrupt to the core.

I will not be party to the provision of the mob with bread and circuses to protect cruel and incompetent leaders.

Re:That's not such bad news (2)

NeutronCowboy (896098) | about 2 years ago | (#38893643)

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.

Re:That's not such bad news (1)

Quiet_Desperation (858215) | about 2 years ago | (#38893535)

It might sound better to just say that health care policy is best not driven by fringe cases like this. You base policy out to, say, 2 or 3 sigmas, and handle anything beyond that on a case by case basis.

Would help to have an efficient government where office holders consult actual experts on complex topics, though, and that's where the whole idea sort of falls about, you see. :-\

Re:That's not such bad news (1)

fedos (150319) | about 2 years ago | (#38893555)

And, as usual with "the market will fix everything" crowd, "everyone" is a code word for "the rich", i.e. "a chosen few".

Re:That's not such bad news (0)

Anonymous Coward | about 2 years ago | (#38892647)

http://www.fiercepharmamanufacturing.com/pages/chart-worlds-most-expensive-drugs

GOOGLE IS YOUR FRIEND

Yes, I'm shouting

Re:That's not such bad news (5, Informative)

rgbrenner (317308) | about 2 years ago | (#38892657)

http://www.forbes.com/2010/02/19/expensive-drugs-cost-business-healthcare-rare-diseases.html [forbes.com]

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.

Re:That's not such bad news (2)

phantomfive (622387) | about 2 years ago | (#38893479)

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.

Ethical? (1)

Maximum Prophet (716608) | about 2 years ago | (#38892549)

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.

Re:Ethical? (1)

LearnToSpell (694184) | about 2 years ago | (#38892581)

Otherwise, you're just dangling it in front of the poor/ uninsured. Hey, we could keep your kid alive, but neener, neener, neener.

Isn't that how a for-profit medical system works?

Re:Ethical? (1)

Maximum Prophet (716608) | about 2 years ago | (#38892779)

Sure, but the US doesn't have one of those. There are laws that a hospital/doctor has to treat the patient in front of them. (If it's life or limb threatening)

Re:Ethical? (1)

sjames (1099) | about 2 years ago | (#38892971)

So they keep the kid alive then hound you through every legal means (and a few illegal ones thanks to debt collector ethics) for the rest of your (now) miserable existence,. Great.

Re:Ethical? (0)

tmosley (996283) | about 2 years ago | (#38893157)

No, it is how a FASCIST medical system works. And that is exactly what we have, and have had for a hundred years: http://mises.org/daily/4276 [mises.org]

Re:Ethical? (0)

Anonymous Coward | about 2 years ago | (#38892595)

Otherwise, you're just dangling it in front of the poor/ uninsured. Hey, we could keep your kid alive, but neener, neener, neener.

I heard that the drug company already announced that they were planning to cover the cost of this for patients who did not have insurance and made less than $150k /year?

Re:Ethical? (1)

dmacleod808 (729707) | about 2 years ago | (#38892653)

if so, that would be great. Most people make less than that.

Re:Ethical? (0)

Anonymous Coward | about 2 years ago | (#38893053)

70% of Americans have an AGI under $57,000/yr. Just saw it on the irs website. Which is funny, that's what Romney makes in a day.

Re:Ethical? (1)

Maximum Prophet (716608) | about 2 years ago | (#38892755)

Might happen. Insurance companies have already tried to block drug company copay cards, because by making the copay small or zero means reduces the incentive that the patient has to use the lower cost drugs.

Many insurance companies and Medicare have "We pay the lowest cost" rules. So if some patients are paying little to nothing, they want all their insurees to pay that cost.

The drug companies can get around this by offering the drug through doctors that don't take whichever insurance has the difficult rules. Sucks to be a patient that isn't close to one of those doctors.

There may be new rules or rules soon to take effect to cover this, but it's a mess right now. Anyway, soaking the rich always sounds good in practice, but eventually the rich get the better of the 99%.

Imagine there's a production or distribution shortage. Who do you think is going to get the drug, and who will wait? Do you think medical necessity will be the sole determination?

Re:Ethical? (1)

fedos (150319) | about 2 years ago | (#38893587)

Doctors don't sell drugs, pharmacists do.

Re:Ethical? (1)

Joce640k (829181) | about 2 years ago | (#38892765)

So by their logic: If you make $160k/year you can afford a drug which costs $294k/year...?

Re:Ethical? (1)

Maximum Prophet (716608) | about 2 years ago | (#38892963)

Most people making 160K/year have very good insurance. If you don't and you have a kid with CF, hello bankruptcy, after you've been sucked dry by the medical/industrial establishment.

Re:Ethical? (0)

Anonymous Coward | about 2 years ago | (#38892989)

No, but you can afford the insurance that would pay for it.

Re:Ethical? (0)

Anonymous Coward | about 2 years ago | (#38893403)

Not if you have said disease, then they won't cover you. Let's say you are a contractor making $90/hr, doesn't matter if you can pay $2k/month to insurance they just won't cover you. I suffer from chronic pain and found this out the hard way...

Re:Ethical? (1)

tmosley (996283) | about 2 years ago | (#38893171)

Presumably you could afford the copay on your super-awesome insurance plan.

Re:Ethical? (1)

raddan (519638) | about 2 years ago | (#38893361)

Actually, Vertex is offering the drug free of charge to anyone who is uninsured and who makes less than $150,000/yr.

Given the cost of development, and the small merket, this sounds pretty reasonable to me.

Good news for the 1%ers with Cystic Fibrosis (0)

Anonymous Coward | about 2 years ago | (#38892607)

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.

Shit, shit, shit!!! (0)

Anonymous Coward | about 2 years ago | (#38892639)

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!

Re:Shit, shit, shit!!! (2)

quantaman (517394) | about 2 years ago | (#38892775)

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.

Re:Shit, shit, shit!!! (2)

Maximum Prophet (716608) | about 2 years ago | (#38892903)

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.

Re:Shit, shit, shit!!! (1)

Aryden (1872756) | about 2 years ago | (#38893287)

So ($294,000 * .20)*.70 = $41,160... That's what's left even after your co-pay and the drug company's assistance. That's still far out of reach for a hell of alot of people. We're talking about a single drug, this isn't including all the regular doctor's bills these people are paying as well.

For some reason, people in this country think the system works the way it is...I could pay out hundreds of thousands of dollars that i don't even have to keep someone alive, or i can go to jail for negligence or even murder if I just let them die...

Re:Shit, shit, shit!!! (1)

quantaman (517394) | about 2 years ago | (#38893529)

So their offer might not be as good as I thought.

Honestly I have no idea what a good system would be.

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. And there's still the question of how to price these things. It's entirely possible that it would not have been economical for them to develop the drug without charging $300,000 for a prescription, but when a drug is literally a lifesaver it's hard to come up with an appropriate number.

Re:Shit, shit, shit!!! (1)

Dog-Cow (21281) | about 2 years ago | (#38892815)

The news that some people with CF might be saved will devastate her? Sounds like she has problems beyond her son's health.

Re:Shit, shit, shit!!! (3, Insightful)

HornWumpus (783565) | about 2 years ago | (#38893039)

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:Shit, shit, shit!!! (0)

Anonymous Coward | about 2 years ago | (#38893289)

Seriously? You can't see how the death of a child from a disease at the very moment a cure for it is found can have a devastating effect on a parent? Perhaps you could benefit from a review of Psych 101 and the grief process.

Are U fucking NUTS? (0)

ElitistWhiner (79961) | about 2 years ago | (#38892689)

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.

Re:Are U fucking NUTS? (1)

Anonymous Coward | about 2 years ago | (#38892891)

There is an effective vaccine for polio, but a quick search reveals there is no cure for existing patients other than hoping the immune system deals with the virus in time (90-95% of people can do this)...

Re:Are U fucking NUTS? (2)

snowgirl (978879) | about 2 years ago | (#38892953)

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...

Re:Are U fucking NUTS? (1)

guises (2423402) | about 2 years ago | (#38893063)

We never and still don't have a cure for Small Pox, we have a vaccine. And, while I'm not an expert, saying that we have a "cure" for Scurvy is just silly. I'm not really supporting the GP here, I don't really know, but your examples aren't much better.

Re:Are U fucking NUTS? (1)

snowgirl (978879) | about 2 years ago | (#38893431)

We never and still don't have a cure for Small Pox, we have a vaccine. And, while I'm not an expert, saying that we have a "cure" for Scurvy is just silly. I'm not really supporting the GP here, I don't really know, but your examples aren't much better.

Oh, I totally agree that we don't have cures for Polio and Small Pox... but if the GP asserts that we have cured Polio, then we have cured Small Pox. (From a generic population as an organism sense, by vaccinating the population we have cured the population of that disease.)

However, while Pellagra and Scurvy are both malnourishment conditions, their cure is the missing nutrient. If anything, they're the best example of a cure. You have an disease, you are treated with the cure, and you are cured of the disease. The fact that the cure are simple nutrients instead of complicated drugs is basically moot.

In the same way, the antidote for 4-Hydroxycoumarins rat poison is just vitamin K... does that make it not an antidote? You know, because it's just a nutritional supplement instead of an actual opposing drug?

Re:Are U fucking NUTS? (1)

Cyclizine (2558090) | about 2 years ago | (#38893057)

Polio still exists, try going to a hospital in Sub-Saharan Africa. Eminently vaccine preventable, just politics unfortunately. Smallpox was declared eradicated in the late 70s.

Re:Are U fucking NUTS? (1)

sjames (1099) | about 2 years ago | (#38893093)

It's not a cure, it's a lifelong treatment. Too bad that life will be one of poverty.

Re:Are U fucking NUTS? (1)

montjoy0 (106031) | about 2 years ago | (#38893253)

Well, since patent lifetimes are 20 years, that's only $5,880,000 per person assuming that they live for that long. I wonder how much it cost to develop - it seems at nearly $6 million per person they easily stand to recoup their costs and make a sick profit.

Re:Are U fucking NUTS? (0)

Anonymous Coward | about 2 years ago | (#38893145)

Wait.. how is this a cure if you have to spend $300k for one year of treatment? Isn't the point of a treatment that it actually, you know, treats the underlying disease?

Why would you need to take this for a year or more then? This doesn't appear to be a cure. This seems like it modifies the defective protein rather than actually modifies the DNA. So calling it a "gene correction drug" if it doesn't actually fix the underlying genetic defect seems a bit of a stretch, does it not?

4%? (0)

Cro Magnon (467622) | about 2 years ago | (#38892781)

At that price, it seems more like a 1% thing.

$294,000 (0)

Anonymous Coward | about 2 years ago | (#38892823)

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.

Worth It (3, Interesting)

Anonymous Coward | about 2 years ago | (#38892995)

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.

Re:Worth It (1)

Aryden (1872756) | about 2 years ago | (#38893347)

One question, after the assistance program, how much is it costing you per month now?

Margin (1)

jpwilliams (2430348) | about 2 years ago | (#38893025)

Does anyone know how these pills are priced, and what kind of margin these companies make? Those prices seem INSANE.

Re:Margin (0)

Anonymous Coward | about 2 years ago | (#38893461)

The drug manufacturing process varies immensely based on the drug. For instance thyroid medication takes up to one year to create a single batch of drug. If anything goes wrong with production (loss of power), etc. The entire batch is ruined. They need to keep production one year ahead of packaging or risk the possibility of running out of pills.

among the most expensive... (1)

Mysticalfruit (533341) | about 2 years ago | (#38893217)

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.

Re:among the most expensive... (0)

Anonymous Coward | about 2 years ago | (#38893563)

That's nothing. 640k should be enough for almost anyone.

Too late for Honez. (1)

GodInHell (258915) | about 2 years ago | (#38893473)

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

Bad Policy (2)

medv4380 (1604309) | about 2 years ago | (#38893477)

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.

Only in the U.S.? (0)

Anonymous Coward | about 2 years ago | (#38893551)

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.

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