FDA Approves Most Expensive Drug Ever, a $3.5 Million-per-Dose Gene Therapy For Hemophilia B (cbsnews.com) 195
U.S. health regulators this week approved the first gene therapy for hemophilia, a $3.5 million one-time treatment for the blood-clotting disorder. From a report: The Food and Drug Administration cleared Hemgenix, an IV treatment for adults with hemophilia B, the less common form of the genetic disorder which primarily affects men. Currently, patients receive frequent, expensive IVs of a protein that helps blood clot and prevent bleeding. Drugmaker CSL Behring, based in Pennsylvania, announced the $3.5 million price tag shortly after the FDA approval, saying its drug would ultimately reduce health care costs because patients would have fewer bleeding incidents and need fewer clotting treatments.
According to a study cited by the National Library of Medicine, the price makes Hemgenix the most expensive medicine in the world, easily topping Novartis' Zolgensma gene therapy for spinal muscular atrophy (SMA), which costs right around $2 million per dose and is also a single-dose medicine. Like most medicines in the U.S., most of the cost of the new treatment will be paid by insurers, not patients, including private plans and government programs. After decades of research, gene therapies have begun reshaping the treatment of cancers and rare inheritable diseases with medicines that can modify or correct mutations embedded in people's genetic code. Hemgenix is the first such treatment for hemophilia and several other drugmakers are working on gene therapies for the more common form of the disorder, hemophilia A.
According to a study cited by the National Library of Medicine, the price makes Hemgenix the most expensive medicine in the world, easily topping Novartis' Zolgensma gene therapy for spinal muscular atrophy (SMA), which costs right around $2 million per dose and is also a single-dose medicine. Like most medicines in the U.S., most of the cost of the new treatment will be paid by insurers, not patients, including private plans and government programs. After decades of research, gene therapies have begun reshaping the treatment of cancers and rare inheritable diseases with medicines that can modify or correct mutations embedded in people's genetic code. Hemgenix is the first such treatment for hemophilia and several other drugmakers are working on gene therapies for the more common form of the disorder, hemophilia A.
NON USA COST $35 Dose (Score:3, Insightful)
NON USA COST $35 Dose
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More like $35,000. ...in a couple of decades, at best.
And most countries won't use it because it's "too expensive."
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More like $35,000. ...in a couple of decades, at best.
And most countries won't use it because it's "too expensive."
I'd have thought that. But checking Zolgensma in Australia: https://www.pbs.gov.au/medicin... [pbs.gov.au]
It is approved on the Pharmaceutical Benefits Scheme! Cost $2,527,773.87. Charge to patient: $42.50
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That will be AUD. 1 AUD = 0.672852 USD, so this is actually 1,700,558.64 USD.
Wonder what makes this stuff about 2x as expensive in the US? Some executive needs to finance another villa or maybe a yacht?
Re: NON USA COST $35 Dose (Score:5, Insightful)
When it comes to special care like this, those governments either pay the cost to have their citizens shipped out here for treatment, or they just plain refuse to cover it at all. Typically the latter, which is basically guaranteed in this case. When it comes to medical tourism, the US is one of the top destinations. The others at the top are mainly there because they're cheap, not necessarily that they're good.
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When it comes to special care like this, those governments either pay the cost to have their citizens shipped out here for treatment, or they just plain refuse to cover it at all.
You missed option 3, the (non-US) government tells the pharma company that if they want to do business here then they'll provide it at fixed price X, not $3.5M.
Oddly enough, no pharma company I know of has ever refused this offer, and they're all still making a fortune despite not being able to charge what they want.
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Indeed. Not all countries pray to the god of mammon. The US has let it take over though.
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Read the sentence after that one.
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You know that it's you that pay it anyway right?
Every time the government has to increase it's budget, it must increase the taxes for that, so you have to pay more taxes.
And if they just "tax the rich"? well, the rich mostly gets money from stuff you buy from their companies, so they will just increase the price of those things to offset the extra tax, and here it goes again, you're paying for it.
The government has also the option to print more money to pay for things with this extra magical money, but that
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It is paid for by the taxpayer, but that bill tends to be less than half of what it would be in the U.S. because countries with socialized medicine aren't afraid to use the buying power of their entire population as leverage to get a better deal.
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When you're not actively using the government power to help and enforce monopolies, the prices tend to be lower.
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Not even close. Not all taxes are regressive.
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Not even close. Not all taxes are regressive.
LOL. Yes they are, in practice. You can have most ridiculously progressive tax system in the world, and the rich will just take their money overseas, hire enough lawyers and creative accountants to find enough holes in those huge volumes of legislation the leftards love so much to drive a truck through, or just bribe their way out of it. How many times, in how many places the leftards managed to lie their way into power, making promises on how they are going to "tax the rich", and invariably, in every one o
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You ... actually believe that? LOL!
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"...a US Tomahawk missile costs $2 million per shot."
So, one shot of the drug that demonstrably improves a chance at more life costs less than two Tomahawk shots that are designed to take the lives of several/many.
As a species, "What are we doing wrong?"
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As a species, "What are we doing wrong?"
The list is long and the list of those that think nothing is actually wrong and pain and suffering is just dandy as long as only people not in their in-group are affected is a lot longer.
Most people are egotistical and vicious morons. As a group, this installment of the human race certainly deserves to be wiped out. And it is hard at work to do that to itself.
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Just curios, where do you think the government gets the money to pay for your 'free healthcare'? Magic? Pixies?
You pay for 'free healthcare' via taxes.
Sure. The question is how you pay for healthcare otherwise. Where do you think the money comes from when you have private health insurance and you need an expensive procedure? Insurance is just a form of risk pooling. It's just another kind of taxation, only all the money goes through a private company that takes a hefty chunk as profit. For that matter, where do you think the money comes from if the insurance company goes bankrupt or needs a bailout? What about when someone is uninsured, but receives treat
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Non USA cost is "not available at any price".
Of course, the in-USA cost is effectively "not available at any price" also, for anyone who isn't either a billionaire or extraordinarily well-insured. Hopefully the prices for this sort of thing will come down at some point to where they can be used to help more than a token number of people.
Instead of hoping for that while people die (Score:5, Insightful)
Go google some of Katie Porter's bits on big pharma if you doubt me.
We don't need them. And they're parasites. Why keep them around? It's time to get over grade school propaganda we were taught in 10th grade economics.
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why not just make it happen? We don't need the pharma companies. They spend a fraction of their money on research and focus only on finishing up work the gov't started. There's no reason why we couldn't do that in public universities (like the rest of the civilized world).
If the rest of the world has this problem solved, great -- hemophiliacs can just fly to a foreign country and get their dose of this treatment (or something equivalent) there, for a much lower price. Or if that's not possible, then perhaps that is the answer to your question.
Re:Instead of hoping for that while people die (Score:4, Informative)
Wow, I think you jumped a bit on the crazy train there. The GP was getting at the fact that the university developed drug model seems not to work because sophisticated drugs like this don't get made, as evidenced by the fact that it's only available in a country that has a mega capitalist model instead. Whereas you trash talked someone you don't even know. Not a good look.
Re: Instead of hoping for that while people die (Score:2)
Oh for fuck sakes. No they can't do that. You still have to be in those countries healthcare systems and this is an ongoing treatment anyway
Oh for fuck sakes. You didn't even finish your third sentence before you made a false statement. In fact, TFS says exactly the opposite.
Honestly why are you still here instead of Cuba? They give free health care you know, which automatically means they're a better country to you, so just go already.
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I'm here instead of Cuba because my job is here.
Oh that's not a problem, in fact Cuba guarantees you a job.
https://www.radiohc.cu/en/noti... [radiohc.cu]
I'm so sick of you guys pointing at socialist Nations that the United States has sanctioned and embargoed and routinely sense the CIA to destabilize and saying lookie lookie socialism doesn't work.
So which one HAS worked?
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What's really funny is how when someone suggests the U.S. adopt some European or Scandanavian country's policy, they're Socialist. When someone points out that they are a prosperous nation with a standard of living as good or better than the U.S., they suddenly become Capitalist.
From now on, I'm tempted to refer to them as Schrodinger's economies.
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Well said. This is pretty much the core problem: People that have no respect for human life unless it is somebody in their in-group. These people are commonly called "evil scum" and we just saw a nice example of their thinking.
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When you look at the world pharma R&D budget, a big block like the EU or US or China could easily afford to take it all on and make the resulting treatments available royalty free. Ideally they could all get together and fund that work.
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We don't need the pharma companies. They spend a fraction of their money on research and focus only on finishing up work the gov't started.
Not really. Don't confuse what a few American pharma companies do with their role on the wider world stage. There's some very real cost from taking something a university spat out to actual production at any kind of usable scale.
The rest of the world is highly reliant on pharma companies too, the difference is they don't have this ludicrous pricing scheme from the USA. Fix that, but don't throw the baby out with the bathwater just because your understanding of the industry is very narrow.
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If I create some new incredibly useful invention, but keep it secret, by what right can you shake me down for it?
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Non USA cost is "not available at any price".
False [slashdot.org]
Oh, did that fact just destroy your absurd exceptionalist fantasy? Sorry!
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While I suspect this drug will also be available, you have again demonstrated your exceptional ability to not understand logical truths, as the non-USA cost is indeed "Not available at any price" (at this juncture)
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Not true, see reference by quenda. The US is deeply corrupted by greed, do not try to make that look ok by lying about the rest of the world.
Re:NON USA COST $35 Dose (Score:5, Interesting)
Doesn't the decision on if the UK healthcare system will cover a drug depend significantly on if the cost is below some threshold per Quality Adjusted Life Year (QALY)? This threshold used to be around $50K but I've not looked at it in a few years. There have been exceptions made for conditions that have particularly effective lobbying groups behind them (after all, the decision is as political as it is medical).
So, for example, if a drug increases the quality of life for a patient by 25% from 50% to 75% but neither extends or shortens their life, the drug would be "justified" if it cost ~$12.5K dollars per year. If a drug extends life from 1 year to 3 years but still at 50% quality (i.e., 1 year at 50% quality vs 3 years at 50% quality), it would be justified if the total cost over the next three years was $75K.
Anyone who thinks the UHC systems are not cost conscious are fooling themselves -- but somehow people accept it more when the government, being a near monopoly, says "You can't have this because it's not cost effective - your life is worth at most $50K/year" than when a private insurer, of which there are various options, says something similar. Odd that...
(Of course, if a drug is not approved due to QALY in your situation, your government doctor probably won't tell you about it and it might not even be available in your country while the US your private doctor is likely to tell you that the drug would be your most effective option but explain that your insurance likely wont cover it. Ignorance is bliss.)
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(Of course, if a drug is not approved due to QALY in your situation, your government doctor probably won't tell you about it and it might not even be available in your country while the US your private doctor is likely to tell you that the drug would be your most effective option but explain that your insurance likely wont cover it. Ignorance is bliss.)
What typically happens when a drug is not picked up in the UK because of cost, the manufacturer magically finds out that they could supply it cheaper and the price goes down. The ones that are entirely unavailable really are the ones that show very little actual provable benefit. In other systems, that downward price pressure doesn't happen and the cost is either met by increasing the cost of insurance or not paid by using loopholes and delay tactics to ensure the patient either never receives the treatment
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Fortunately, the UK has the benefit of indirect subsidies from America which end up paying for the drug development/trials through their high drug prices - mostly paid by American taxpayers and employers via insurance premiums. Thus the manufacturers can reduce prices once the drug is approved as long as someone else is paying for the R&D if they still make reasonable profit on just the incremental cost of production. In the US drug companies also offer programs for low income patients to give cheap acc
Re: NON USA COST $35 Dose (Score:2)
Exactly this. Like it or not, human life has a price. Someone, somewhere must decide what that price is, and I would far prefer that to be based on objective, documented guidelines.
Taking the parent comment's numbers: a life is worth 50k/year,which would put the upper limit on any medication at about 80x50k or 4 million. Now, you can debate the 50k - maybe it should be more. What we should *not* have, is an opaque system where some faceless paper-pusher can make an arbitrary decision.
Yep (Score:2)
The data in this chart is from 2018. We can only imagine the further divergence in 2022.
https://www.rand.org/blog/rand... [rand.org]
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Re: NON USA COST $35 Dose (Score:2)
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It's not available outside of the US.
I wonder why? I also wonder how the rest of the world treats this condition. In countries with universal healthcare, a visit to the doctor & a prescription without healthcare coverage won't bankrupt you. It ain't the USA!
Re: NON USA COST $35 Dose (Score:2)
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Fun fact (Score:5, Insightful)
Re:Fun fact (Score:4, Insightful)
Re:Fun fact (Score:5, Interesting)
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"Hey, it seems to produce one beneficial effect in isolation! That's all I need to consider! My queen took a pawn, therefore, I'm slightly ahead, therefore I win the whole game!"
Re:Fun fact (Score:4, Insightful)
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The technology that made this possible was developed at public universities using taxpayer dollars. Privatize the profits, socialize the expenses. Are we ever going to get tired of them doing this to us?
And what stopped the university commercialising it? It's about the same difference as theory vs a product on the shelf. There's more involved in producing drugs than just doing some R&D at a university.
That said there's no justification for this price tag at all, but man the "evil big pharma" crap is getting tiring. Yeah, there's some real fucks in the world, but let's not pretend that means we can just go at it without pharma companies.
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It shouldn't really be that tough to figure it out if you just look at the average salary of a pharmaceutical sales rep.
how much is too expensive? (Score:4, Insightful)
There is one little snag - the insurers are funded by "patients". 3.5M treatment is well beyond what a single person contributes back to the society. At what point the treatment becomes too expensive to justify?
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Re:how much is too expensive? (Score:5, Interesting)
The treatment is justified as being cheaper than the alternative. So your real question is "why don't we dash hemophiliac babies on the rocks before they become a burden?"
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It's recessive. You might qualify for extermination.
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It's recessive. You might qualify for extermination.
It is only recessive for females.
Queen Victoria was a carrier, as was her granddaughter Czarina Alexandra of the Russian Empire.
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I'd go one step further and mandate sterilisation for anyone carrying the gene. In just one generation the problem is solved.
Not true. The blot clotting gene on the X chromosome contains several "kinks" that are highly susceptible to mutation. About a third of hemophilia cases are caused by spontaneous mutation.
So even with forced sterilization, the disease would continue to occur.
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Nice. That fuckup above you argues for fascist-style eugenics and it would not even work! Some people are just complete and utter failures.
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I'd go one step further and mandate sterilisation for anyone carrying the gene. In just one generation the problem is solved. Don't worry there's 8 billion humans already - we'll be fine.
Except for the fascist Eugenics you are advocating and that you _cannot_ limit to this one factor. Are you really this stupid or are you plain evil?
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Are clotting agents and blood transfusions effective compared to the alternatives? What side effects do they have? What is their effect on lifestyle? How does blood type availability affect their odds of effective treatment?
I saw somewhere else that the alternative treatment for hemophilia B has an MSRP of something like $30,000 per week. Based on some other clues, this appears to be Xyntha, which is, according to Wikipedia, "a recombinant antihemophilic factor genetically engineered from Chinese hamster ov
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how many people's lives could you save or improve if you used just half of this amount.
Tell all those people living in poverty, that you are worth so much more than they are. That your inconvenience needs to be alleviated and that you don't give a damn if they have enough to eat.
Now if some twit like Bezos wants to buy this, go ahead, but please do not make the rest of the population pay for it.
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You're really bad at this. Your buddies on 8chan or whatever might not know how taxes work, but the majority of people here do.
Take your bullshit elsewhere.
Re:how much is too expensive? (Score:5, Informative)
The insurance company isn't paying this amount.
I take Elliquis. Nominally, a month's supply costs about $500. But the manufacturer offers a coupon that brings the cost down to $10/month. It's $10/month for me even before I have met my deductible.
Clearly, the insurance company isn't paying $500/month, just as the insurance company isn't going to pay $3.5M for this treatment.
Re: how much is too expensive? (Score:2)
Re:how much is too expensive? (Score:5, Interesting)
So what is your proposal? Death panels?
Actuaries. Basically the same thing.
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Genetic screening programs for the at-risk. WAAAYYY cheaper, and a nose bleed won't kill you.
Alternatives for the at-risk of giving birth to a bleeder:
Or, since it's only the mother's x chromosome that carries a defective gene as one of the pair of xx, just screen the eggs (which only carry one copy of the x gene) so that you only use eggs with only a g
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The religious fuckups do not want that. For that US that means end of discussion, because they have taken over.
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Indeed.
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Yes.
For corporations.
Re: how much is too expensive? (Score:4, Informative)
So what is your proposal? Death panels?
Your insurance company already has death panels, you just never thought of that.
Re:how much is too expensive? (Score:5, Insightful)
So what is your proposal? Death panels?
Basically, yes. Whether the decision is made by insurance companies or government bureaucrats, there are practical limits in every country on how much can be spent.
You don't have a right to infinite amounts of other people's money.
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You don't have a right to infinite amounts of other people's money.
I don't want "infinite amounts," I'm sure we could do a lot of good if we just put 50% of the wealth of the top 0.1% into healthcare, education, and public transport. The super-rich would still be super-rich, they'd just have to share a bit more of it with the rest of us.
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So what is your proposal? Death panels?
At current pace it's looking like pitch forks is going to win by default.
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So what is your proposal? Death panels?
Every corporation that deals in something even slightly risky has a dollar sign on human life. Even when they go out of their way to hide it, you can compare their risk tolerance (e.g. tolerance to a fatality of a member of the public, to tolerance on loss of production from a commercial incident) to come at the number.
You can use the fancy politicised term "death panels" if you want, but the reality is that insurance companies do pull the plug on you. No one is worth unlimited funds, potentially even their
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Very rare disease (Score:2)
Feels good when you can redirect the resources of the entire globe to solve your own person problems before everyone else's.
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How do you know the research done for this drug won’t be of any future use?
Re:Very rare disease (Score:4, Informative)
I was surprised they did this for the very rare B first instead of the more common A, no pharmaceutical company would go through expensive FDA trials to treat a rare disease when they can focus on the more common one first using the same technology. Then I realised that this is the rare strain the European royal family has.
Feels good when you can redirect the resources of the entire globe to solve your own person problems before everyone else's.
It looks like that particular strain died out in the 1940's [wikipedia.org] so I'm not sure it's a big concern in the present day.
It also accounts for 15% of Hemophilia patients, so unusual, but not "very rare".
I suspect the reason for Hemophilia B is something less conspiratorial like the particular mutation somehow being easier to treat or of the different groups trying to do this with different diseases at different companies this particular Hemophilia B group was the fastest.
Ransom-esque. Just Say "No" FDA (Score:2)
This is largely why our health-care costs keep going up: pharma makes an expensive treatment, and insurance feels obligated because the alternative is great suffering or death of the patient. They pull on our heart-strings. Just up-front say *no* approving anything above a threshold and they'll stop trying.
It's like paying ransom: the more you pay up, the more kidnappers & hackers get into the biz.
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They pull on our heart-strings.
I know what you mean. It's like when Republicans say they're passing anti-abortion laws to protect the "child" even if it kills the mother.
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They pass abortion-restricting laws to increase the population and then refuse to introduce gun reform laws, which helps to counter the population increase. It's a happy balance.
Re:Ransom-esque. Just Say "No" FDA (Score:5, Interesting)
Looked it up, yep 3.5M is cheaper. Expected lifetime cost of the IV method is 21M. https://www.tandfonline.com/do... [tandfonline.com]
Is is cheaper long-term? (Score:3)
If the normal treatment costs $5k/month for 60 years then this drug is marginally cheaper.
it's hereditary, edit the germline (Score:2)
There's a simple solution (Score:2)
Generics for everyone!
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There'd be quite a few years for this drug. Sufferers from the genetic defect are fairly rare, so the patent would probably expire first, even at this large of a charge. Which is part of the reason for the large charge - the company did have to spend the money getting the treatment approved.
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Even simpler than that. Just have the US government declare a "war on hemophilia B" and invoke its rights to bypass the patent holder's claims [wikipedia.org].
Who pays? We all do. (Score:2)
And just where do insurers get the money to pay for these exorbitantly priced drugs? Us... the people paying the monthly premiums. Can a $1B/dose drug will be next? Wouldn't surprise me.
Compulsory treatment (Score:2)
Wait, what?
Re:Thanks Biden (Score:4, Insightful)
Good job to Biden and Fauci. They sure like their pharmaceutical allies. Payback!
You mean the orange faced goon and his pharmaceutical allies [forbes.com]. Over $18 billion for covid vaccines [thehill.com], approximately $10 billion of which [statnews.com] was secretly taken from a fund for hospitals and health care workers.
Re:Thanks Biden (Score:5, Informative)
You misspelled Mitch McConnell.
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lol /., you indeed know your poison. :'D
Re:Thanks Biden (Score:4, Informative)
That's a silly argument. The FDA cannot fail to approve a drug on the basis of cost. From
https://www.fda.gov/about-fda/... [fda.gov]
under point 16:
" However, the FDA has no legal authority to investigate or control the prices set by manufacturers, distributors and retailers."
If you want to change the price of drugs, you'll need to get a bill through Congress. Even then, I doubt the FDA would be tasked with those sort of financial decisions. Come to it, what agency would be tasked to making those decisions? What are the criteria? That would not be an easy bill to write much less get passed.
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inmates will get it free and if not sue and force (Score:2)
inmates will get it free and if not sue and force them to give it to you as if they do not then they are killing you.