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United States Businesses Government Medicine The Almighty Buck Politics

The Cost of Drugs For Rare Diseases Is Threatening the US Health Care System (hbr.org) 311

An anonymous reader shares an article: There are 7,000 rare diseases affecting 25 million to 30 million Americans. The average drug approved under the Orphan Drug Act of 1983 (ODA), which governs rare disease approval, costs $118,820 per year. Assuming a similar cost, if a single drug were approved under the ODA for 10% of rare diseases, the total would exceed $350 billion annually -- more than 10 percent of the total amount that America spends on health care and much more than the health care costs attributable to either diabetes or Alzheimer's disease and other forms of dementia. If this seems far-fetched, consider the two drugs for treating Duchenne muscular dystrophy that the FDA approved in the last six months: eteplirsen, which is sold by Sarepta Therapeutics and costs $300,000 annually per patient, and deflazacort, which is sold by Marathon Pharmaceuticals and costs $89,000 annually per patient. However, approval of such costly drugs exposes an uncomfortable truth: scientific discovery has outpaced health care economics. [...] In the United Kingdom, the National Institute for Health and Care Excellence (NICE) determines the cost effectiveness, or value, of newly approved drugs based on their impact on quality-adjusted life years. These determinations inform the National Health System's (NHS) treatment-coverage decisions. In contrast, the FDA is prohibited from considering cost or value in its decision making, and there is no U.S. governmental equivalent of NICE.
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The Cost of Drugs For Rare Diseases Is Threatening the US Health Care System

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  • Wait! (Score:5, Insightful)

    by war4peace ( 1628283 ) on Friday April 07, 2017 @12:26PM (#54192507)

    I wonder what's the markup on those drugs.
    Are they that costly to produce?

    • They might since for some of these there'd be no economics of scale... it is like the difference between buying parts off the shelf versus having them machined.
    • by Luthair ( 847766 )
      While I think these are likely a rip-off, you do also need to consider R&D costs and for rarer disorders the cost spread over a smaller number of dosages.
      • You could perhaps spread research costs around to all projects. Such as in the olden days when major corporations had research arms out of which a few major advances and profit engines would arise as well as lots of smaller advances that would contribute much less to the bottom line. But then this really messes up costs for the commonly used drugs as well. This would make drugs for rarer drugs cheaper, but the drawback is that it makes the common drugs more expensive. The profits from Viagra are not paying

    • Do you mean produce as in to manufacture. Or do you mean to research and development and manufacture? If it's a rare disease then it by definition doesn't achieve economies of scale.

      Some rare diseases, if untreated, will lead to a person's death. That's bad of course.
      Other rare diseases primarily impact quality of life and treatment can turn an sick unproductive person into a functional productive one. That's good for society, although you will probably have trouble justifying it by the numbers alone if you

      • I picked the train of logic AFTER the question of whether it makes sense to spend X billions of dollars on research for disease Y and save Z amount of people, where Z is under 1000 and the positive economic impact is near zero.

        R&D is tricky to measure, a study costing a million dollars can yield 10 billion dollars, while another study costing 500 million dollars might yield 5 million. therefore one R&D could cover another.

        Hell, a simpler solution would be to ever-so-slightly raise the price of commo

    • Re:Wait! (Score:5, Insightful)

      by Solandri ( 704621 ) on Friday April 07, 2017 @12:43PM (#54192695)
      When it costs $2.5 billion to get FDA approval for a drug [scientificamerican.com], and your potential market is a few thousand or tens of thousands of people, you have to price it that high just to recoup your costs.

      The real problem is that we have buyers (insurance and government) who will pay that price regardless of whether the drug actually provides that much value to the patients. It's a noble sentiment to believe that every life is worth saving. But practically, when you try to do that you just end up burdening society with costs which give you a negative return on investment (you're throwing away money - people's productivity that they've sent in good faith to government and to insurance companies).
      • by Luthair ( 847766 )
        Your quote is very misleading, that isn't the cost of FDA approval not even the real cost. Your article states 1.4 billion to bring to market (from nothing) then decides investors could have somehow made 1.2-billion in that period by investing - a 90% return is ludicrous over 10-years is unlikely and since its not all spent at the start of 10-years the supposed rate of return is more like 180%.
        • ...a 90% return is ludicrous over 10-years is unlikely and since its not all spent at the start of 10-years the supposed rate of return is more like 180%.

          One word: compounding

          The reality is that using the doubling formula [ time-to-double = log(2) / log(1 + r) ] with a 7% annual interest rate gets you to 10.25 years. As far as estimated rates of return go, 7% is pretty average in terms of investment planning. Nothing ridiculous there.

          The Wikipedia article on internal rate of return [wikipedia.org] has some examples of how the rates of return on capital projects are calculated in terms of cash flow.

    • 75% Margins (Score:5, Interesting)

      by Anonymous Coward on Friday April 07, 2017 @12:43PM (#54192697)

      Bristol-Meyers Squib has a 75% margin on their drugs. And almost 30% return on equity.

      They like to blame R&D but one Summer I worked at one of their research labs. It was a very very nice place. Parts could have been from a country club. The head of the place helicoptered in from NY every morning - which is all considered R&D "costs". The cafeteria food was 5-star but cost as much as a McDonald's meal.

      The only sucky part was the animal section.

      I miss that place.

      • The question is: what fraction of the cost did that overhead reflect? I've seen gross-mismanagement, and I've seen stuff that's so damned expensive that a 0.5% extra expense is... a lot.

        Executive salaries are typically interesting in that respect: the actual cash compensation for most executives amounts to pennies per hour. The CEO of Ford gets, from revenue, about 1.05 cents per employee per hour, or $21 per year; stock and options compensation come at the expense of diluting stock--basically the bus

      • Then introduce competition. Bristol-Meyers can get away with paying for a helicopter ride very day because the FDA has created rules that makes getting into the drug business nearly impossible. Someone could bring this all to a screeching halt by bringing in a competitor that offers products for less because they aren't paying for aviation fuel and a helicopter pilot.

    • There is a far bigger systemic problem behind this than market gouging. Governments poor billions into medical research: the salary for medical researchers is 2-3 times that of scientific researchers and the government budgets for medical research far outpace those for science in many countries because it is politically easy to justify developing a new drug or a new treatment.

      This is the long term problem behind spiraling medical costs. Money spent on developing new medical treatments end up costing soci
    • by Jhon ( 241832 )

      "I wonder what's the markup on those drugs. Are they that costly to produce?"

      Most certainly, when you factor in the cost in R&D the cost per pill over a given period of time, the cost of FDA approval -- basically everything it takes to bring medication to market. Most people don't consider any of that -- just that when they put the powder in one end and the pill comes out the other end how much is the pill?

      By definition, rare diseases are RARE and they wont be making huge numbers of pills to spread ou

      • By definition, rare diseases are RARE and they wont be making huge numbers of pills to spread out the total cost.

        Rare and yet ten times more Americans suffer from them than the margin by which Hillary 'won' the popular vote.

        And yet Democrats still whine about that so much you'd think it was a really significant portion of the US population.

        If 3 million people is enough to consider that Hillary 'really won' the election then is 30 million people enough to consider these diseases worth fighting?

        • by gnick ( 1211984 )

          If 3 million people is enough to consider that Hillary 'really won' the election then is 30 million people enough to consider these diseases worth fighting?

          That's 25-30 million people with 7,000 different diseases. That's about 4k people per disease. They're not researching a single cure for all those 30 million people.

          WTF does this have to do with the election or number of votes cast? There's only one person still obsessed with the election results and TFA isn't about him.

    • Gross margin? Huge.

      Net margin? It costs several billion to get drugs to market. All that FDA safety and efficacy stuff is about $310 million, from lab rat studies over 3 years to a 3-year follow-up with patients to document side-effects and efficacy once the drug is out in the market. Only about 10% of new drugs make it to market, and a lot goes into the pipeline and doesn't even make it to being a "new drug". The sunk research costs are $3-$5 billion per drug.

      For these drugs, they have to recover

    • by epyT-R ( 613989 )

      The costs involved in their development.

    • There is nothing much we can do about the new, branded medications in this list other than wait for the patent to expire, but compounds like Daraprim, the one Martin Shkreli wants $750 per pill for, are off patent and sell for pennies on the world market. If Trump wants to be taken seriously on this whole populism thing, it's time to strip the FDA of its power to prevent us from freely importing these long-since-approved ompounds.

    • Re:Wait! (Score:4, Interesting)

      by Interfacer ( 560564 ) on Friday April 07, 2017 @04:52PM (#54194897)

      Quite late in the discussion, but yes. I work as a sysadmin in a big biotech pharma company where we make special treatments for a specific rare disease that will kill the people who have it if they don't get it. It cannot be cured, because it is due to a genetic defect. The only option is to supply the patient with enzymes that their own body doesn't make.

      The medicine itself is 'reasonably' easy to make. But not when you have to comply with the regulatory requirements of various countries.

      All research, marketing and other costs aside, we have a half a billion dollar plant, with over 500 employees, to make a drug for only 2500 patients worldwide, give or take. You can do the math on that. The reason we need that many employees is because the rules surrounding biotech drugs is incredibly strict. We are audited several times per year by various agencies, and things like change control, exception and deviation management, etc, are gruesome. Top heavy and very labor intensive.

  • by haruchai ( 17472 ) on Friday April 07, 2017 @12:31PM (#54192559)

    Just how many Martin Shkrelis are there in the (supposedly) (legal) drug business?

    • by MightyMartian ( 840721 ) on Friday April 07, 2017 @12:38PM (#54192647) Journal

      Plenty. It's because we allow sociopaths to run companies. Sociopaths should be banned from all management positions, should be outlawed under pain of horrible death from having any power over any finances. They should be permitted, under heavy surveillance, to work flipping burgers at McDonalds or cleaning streets, but they should never ever be put in a position where they can affect a market or a pension fund or any significant transaction, and if they're caught trying to fuck around with their coworkers, they should be removed permanently from society.

  • The average drug approved under the Orphan Drug Act of 1983 (ODA), which governs rare disease approval, costs $118,820 per year. Assuming a similar cost, if a single drug were approved under the ODA for 10% of rare diseases, the total would exceed $350 billion annually

    In this chain of healthcare, who is making the money?

    To be more precise, who is making a killing?

    It's a fact that some entity (cabal) is making big cash, but find it hard to come to terms that this cabal or cabals are profiteering over the misery of others in these United States.

    • In this chain of healthcare, who is making the money?

      To be more precise, who is making a killing?

      It's a fact that some entity (cabal) is making big cash, but find it hard to come to terms that this cabal or cabals are profiteering over the misery of others in these United States.

      Without the profiteering, many of the drugs would not exist. It is difficult to conceive of a different model that maintains the current level of R&D.

    • The researchers in lab coats are making big money. A lot of it is their wages. The wages of people at chem synth companies, mining facilities, and other supply chain services is also a big part of the majority cost.

  • by Anonymous Coward on Friday April 07, 2017 @12:35PM (#54192605)

    We have a culture built around the concept that human life is worth any amount of money. At the end of our lives we spend and spend to pry out another 2 weeks of lifespan.

    Instead maybe we should realize that with 7.5 billions of people, the value of any given human life is very close to zero. It sounds harsh, but it is also the truth. If people want to pay to save themselves, great have a go, but we should not be foisting that cost onto society when there is no rational reason. Spending those sums is absurd.

    We don't even treat our pets in that way: we realize that life is finite, and we try to keep them comfortable at the end so they do not suffer, but we are willing to acknowledge that there is a time when it is best to let them go. Ease their passing so they can go in peace and as much comfort as possible. We need to adopt this attitude for people as well.

    We're not a scattered band of 10K hunter-gatherers on the brink of extinction any longer. A random human life is simply not valuable.

    • by sinij ( 911942 )
      Old people vote, so good luck advocating common sense that 2 weeks of lifespan is not worth couple mil of other people's money.
    • by Altrag ( 195300 ) on Friday April 07, 2017 @02:42PM (#54193893)

      That's just not true. We value human lives very little. What we value is the lives of our friends and families.

      If your brother gets shot in Iraq, its a tragedy to you personally. If an Iraqi's brother gets shot, its a statistic to you. And the Iraqi thinks the exact same thing except in reverse.

      This is a good part of why Americans are so resistant to a decent healthcare system.. Joe is happy to spout off things like "well I'm not sick and nobody I know is sick so why should I be paying for Jane from 6 miles down the road?" But of course if Joe gets sick himself a year later, suddenly he's bitching about the cost of doctors and medicine. Because now it actually matters to him personally. His life is worth paying for but Jane's isn't. And Jane thinks the same way about Joe.

      Basically it all comes down to that "Monkeysphere" concept we hear about, combined with "greed solves everything" capitalism and some American-dream style eternal (if unfulfilled) optimism about a person's own potential.

  • Nonsense (Score:5, Insightful)

    by c ( 8461 ) <beauregardcp@gmail.com> on Friday April 07, 2017 @12:36PM (#54192615)

    What's threatening the US health care system is putting profit ahead of lives.

    Good luck. You poor saps are going to need a lot of it.

    • Re:Nonsense (Score:4, Interesting)

      by blindseer ( 891256 ) <blindseer@noSPAm.earthlink.net> on Friday April 07, 2017 @02:07PM (#54193549)

      If there is no profit in saving lives then it cannot go on for long. If drug companies cannot profit making drugs then their won't be any drug companies. If a brain surgeon gets paid as much as a truck driver then you aren't going to have many brain surgeons, at least not many that's any better trained than a typical truck driver.

      This is not a choice between lives and profit, we can have both.

      What a lot of nations chose to do is, out of "compassion", put government in charge of providing medical care. This has been and will always become a disaster. The people that provide care may be the best people at the start but without competition, which requires a profit motive, the quality of care will fade. People need motivation to improve, provide quality care, and profit provides that.

      It sucks that labor and materials cost money but that is the world we live in and that cannot be changed. Removing the profit motive to providing quality medical care and people WILL die needlessly.

      Even the most compassionate person needs resources to provide medical care to those in need. The best, and perhaps only, means to make sure that the compassionate have those resources is with a free market. A free market means people will profit.

  • by Jim Sadler ( 3430529 ) on Friday April 07, 2017 @12:38PM (#54192641)
    Our society is so corrupt that we do not regulate the costs or profits made by drug producers. There are drugs that are dirt cheap to produce that cost a king's ransom. I suppose that the right wing will consider that as a freedom issue which is nonsense. Do they mean free to die in agony?
    • by wiggles ( 30088 )

      See, here's the issue.

      NHS and other non-US markets negotiate prices with drug companies, often forcing them to sell those medications at a loss or lose access entirely to that market. In order to make that loss back, they have to shift the cost of the drug to markets who do not force pricing - especially the USA. That's one big reason why the US has jacked up prices - we are effectively subsidizing medical treatments for the rest of the world.

      When you implement price controls and force a producer to sell

    • That's precisely their argument. They'll tell you that if you don't like the price of drugs, you should refuse to buy them and let the free market decide. There are always [technically] alternatives, because the was a time before the new drugs existed. But the old treatment may have been to lay in bed doped up on morphine while you wait to die.

  • by PopeRatzo ( 965947 ) on Friday April 07, 2017 @12:39PM (#54192655) Journal

    The cost of Sovaldi and Daklinza (used together) to treat Hepatitis C (which infects 3.5-5 million Americans), is $336,000 for the 24-week course of treatment. $1000 for each pill. The cure rate of Sovaldi and Daklinza is approximately 90%. The same drugs in India cost about $4 per pill.

    Hepatitis C currently kills more Americans than any other infectious disease.

    https://www.cdc.gov/media/rele... [cdc.gov]

     

    • Do you know if India is producing the drug legitimately or is it violating intellectual property laws? If the former then... wow... it is like DVD region control taken to the extreme...
      • by rhazz ( 2853871 ) on Friday April 07, 2017 @03:30PM (#54194283)
        The drug is licensed to drug manufacturers in India who produce it and sell it. The difference is that the Indian government negotiates an acceptable price range for the drug. Most western governments do the same price negotiation. The US does not, at all, by law. Source [cure-hepc.com]

        However, in the United States, price negotiation for medicine doesn’t exist. Medicare is required to accept the price given by the pharmaceutical company. Federal law doesn’t even allow Medicare to bargain or negotiate bulk discounts. It is only logical that under these circumstances the prices skyrocket, and it becomes the norm to pay high prices for medication.

    • Well, I've never wanted to travel to India, but a nice long vacation there seems like a no-brainer if you get Hep-C and aren't extremely wealthy.

        I could just do a quick round trip to get the drugs and not even worry about missing work or having to pay for a hotel room.

      Customs would probably steal the drugs from me at the border, I suppose, and maybe lock me up as a mule.

      • Well, I've never wanted to travel to India, but a nice long vacation there seems like a no-brainer if you get Hep-C and aren't extremely wealthy.

        Unfortunately, that doesn't work any more. India has placed limits on pharmaceutical tourism. They're probably worried about Big Pharma launching a Tomahawk missile strike on them or something.

    • The cost of Sovaldi and Daklinza (used together) to treat Hepatitis C (which infects 3.5-5 million Americans), is $336,000 for the 24-week course of treatment. $1000 for each pill. The cure rate of Sovaldi and Daklinza is approximately 90%. The same drugs in India cost about $4 per pill.

      Honest question: If the US had the same laws as India, would the drugs exist? If everything were government funded or if wild success did not necessarily mean wild profits, do you think we'd get the same quality results?

      Both drugs were developed in New Jersey.

      • Honest question: If the US had the same laws as India, would the drugs exist? I

        A better question might be, "If the drugs were developed in New Jersey, then why does a smaller percentage of Hepatitis C patients in the US have access to them than Hepatitis C patients in India?"

      • by pnutjam ( 523990 )
        They would, we're just used to hearing Big Pharma's side of the argument.
        citation: http://www.huffingtonpost.com/... [huffingtonpost.com]
    • And what would India have to pay if the US consumers weren't subsidizing that price?

      Fundamentally, the Western (and increasingly the rest of the world) has a problem with medicine: we are capable of amazing feats of medicine and healing. At what point do we have the courage to start deciding what human lives are worth?

      1) Yes, we can save that hemophilic fatally allergic-to-everything crack child. It'll only cost $300,000 a year in constant hospitalization, therapy, and treatments. And if we're lucky, the

  • by DatbeDank ( 4580343 ) on Friday April 07, 2017 @12:41PM (#54192683)

    A little known secret: Most countries' governments arbitrarily set the price of drugs and medical devices during negotiations and force pharma and medical device manufactures to sell it at a loss (or simply not have access to that market). To make up for the R&D and marketing, they have to jack the price up in the US to make up the loss. http://www.ibtimes.com/how-us-... [ibtimes.com]

    With the upcoming collapse of Obamacare, the rest of the world should be afraid of the US doing the same to the drug and med device companies. The cost of healthcare for the rest of the world will go up while it goes down for the US. I shudder to think about the hoards of angry folks when NHS starts becoming moderately expensive.

    • by jopsen ( 885607 )

      The cost of healthcare for the rest of the world will go up while it goes down for the US.

      Nope, but we might see reduction in research and development of new drugs...

      But in fairness, there is value to balancing how much money we spend on researching new drugs and how much we spend on treating people with existing drugs..Clearly, it's not cost efficient to develop drugs for rare diseases..

      Or, maybe the end result is higher government subsidies for research and development, which probably could be more efficient economically than financing drug research and development through the stock marked

      • We build and maintain roads using taxes because it's expensive, has a large benefit to society, and is hard to pay for without destroying those benefits. We should do the same with drugs- all research, production, and distribution done by government. Doing so will eliminate the high risk to corporations (failed drugs=lost money), and ensure our healthcare dollars are only spent on costs not private helicopters.
    • by ccguy ( 1116865 )

      A little known secret: Most countries' governments arbitrarily set the price of drugs and medical devices during negotiations and force pharma and medical device manufactures to sell it at a loss (or simply not have access to that market). To make up for the R&D and marketing, they have to jack the price up in the US to make up the loss.

      Yes, this sounds like something the US would be OK with :-)

      Many of the big pharmas are European. https://www.tharawat-magazine.... [tharawat-magazine.com]

      The US treats drugs as commodities, subject to supply, demand, and buyer desperation. That's all. Europe does not, at least not as much as the US would like. If the US imposes a limit on prices what will happen is that companies will be forced to be more efficient, make less money, or let new players take their place in the market. All of those options are fine by me.

    • Re: (Score:2, Informative)

      by Anonymous Coward

      With the upcoming collapse of Obamacare

      You've been watching too much TrumpNews. The "collapse," if it happens, only refers to there being no providers on the ACA-created health insurance exchange marketplaces. We can use scary words like "explosion" but the fact of the matter is that unless and until there are ZERO policies sold on the exchanges in the ENTIRE country, we're a step ahead of where we were in 2010, and that leaves aside every other reform in the entire act.

  • by frovingslosh ( 582462 ) on Friday April 07, 2017 @12:44PM (#54192707)

    We all know these drugs have an insane markup. The drug companies are getting rick because they set astronomical prices for drugs the might help people, even a little. And they get it because insurance is forced to pay for it, not individuals who could never come up with the money on an individual basis. We have created the problem by mandating insurance and then letting the drug companies pilfer it blind.

    This is just another facet of the problem that drug companies use U.S. public funding for the research to help develop most of these drugs, then turn around and charge the American taxpayer more for the drugs than they sell them in other countries, both third world countries like the African nations and first world countries like Canada. And, of course, they spend a lot on expensive lobbying to buy politicians to make sure we in America don't get access to those drugs they sell in Canada.

  • by Lucas123 ( 935744 ) on Friday April 07, 2017 @12:46PM (#54192735) Homepage

    Common statin's (for lowering cholesterol) can cost more than $700 for a 30-day prescription if you don't have insurance. I think the cost issue goes well beyond prescription drugs for rare diseases, and in fact, is more detrimental in a broader sense.

    But, when we as citizens don't insist our politicians address campaign finance reform, policies favoring corporations will continue to guarantee price gouging will continue. Campaign finance reform should be made the top issue... Every. Single. Election.

  • for someone's medicine? I said "Force" there. Because if you're getting into single pay with taxes funding health care then you are effectively forcing me and everyone else to pay for their medicine. Maybe I want to, but shouldn't that be my choice? Shouldn't I decide if I'm going to give that money to a charity or not? What if I oppose the medicine being give (like the "Morning After Pill")?

    I'm asking loaded questions here. I'm in favor of single payer because it's part of civilization. And I'm in favo
    • You understand that the American Revolutionaries never intended to set up a system where every single person had to opt-in for a tax. The issue was to have taxation with representation. You live in a representative democracy, which means that the obligation is that a majority of the elected representatives agree to the tax.

      Trying to turn this into a moral issue is bizarre, since essentially your position boils down to "I want the right to abrogate any moral obligation I have to my fellow man, and that's tot

  • Lower the production and compliance costs or increase the number of people with the rare diseases. The second one isn't possible so the first one is mandatory. That is within the FDA's control.

    Bureaucrats will tend to want to do neither and to make everybody else pay for their phoney baloney jobs but that does nothing to improve market incentives and only winds up increasing costs further.

  • The real reason why healthcare is so expensive in the US is because Americans want the "freedom" to have everything they want, but expect that someone else should have the responsibility of paying for it. This leads to attitudes such as:

    "I want to eat the cheapest and unhealthiest food and drink as much soda as I want, and expect my health insurance to pay for my diabetes, heart disease, morbid obesity, and metabolic syndrome, because it's my right to decide what to eat."

    "It's my right to feed my children

  • In the United Kingdom, the National Institute for Health and Care Excellence (NICE) determines the cost effectiveness, or value, of newly approved drugs based on their impact on quality-adjusted life years. These determinations inform the National Health System's (NHS) treatment-coverage decisions.

    In other words, if it's too expensive to treat you and therefore not worth it, they decide you don't get the medicine. This is what folks in the US were referring to as "death panels". They're actually correct, but the reality is that in the US anybody who can't afford the drug is going to die, also.

  • I've seen multiple stories about how a small number of Americans are responsible for most of the healthcare costs in the last week or two. These weren't part of the general healthcare debate leading up to the failed Obamacare re-write (I refuse to call it a repeal, they didn't have the guts to do that). They started springing up about a week or two ago.

    Yeah, these stories might be true and all, but what I find disturbing is how they keep showing up all of the sudden. Coincidentally right around the time
  • No, it's greed over people pure and simple. cancer treatments that cost $100k per treatment in the US vs $3k in Canada for the same drug by the same maker.
    Arsewipes like martin shkreli and Elizabeth Holmes are not poster children, but the norm.
    The US FDA went on a war against imported Canadian drugs a few years ago to increase profit to the US pharma cartel and their paid for elected pawns to ensure profit continues to roll in.
    UW, the University of Washington is developing a cure, a real cure, for
  • by morethanapapercert ( 749527 ) on Friday April 07, 2017 @06:19PM (#54195471) Homepage
    $89,000/yr for deflazacort? Big Pharm clearly has the US health industry blindfolded, bent over and reamed but good doesn't it? My son has Duchenne's Muscular Dystrophy and is taking deflazacort for it. It hasn't been approved for general prescription here in Canada, but getting approval for it to treat DMD is a straightforward rubber stamp through the exceptional access program. Because it isn't formally approved, we have to pay for it and then get reimbursed for it, Also because it's an EAP drug, we're paying only a little over wholesale. Currently we pay 85$ for a three month supply, or 340/yr. That includes shipping from the pharmacy associated with the research and teaching hospital my son is being treated by.

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