Slashdot Mirror


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

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.

27 of 311 comments (clear)

  1. Wait! by war4peace · · Score: 5, Insightful

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

    --
    ...gis sdrawkcab (usually not responding to ACs; don't bother posting as AC)
    1. Re:Wait! by Solandri · · Score: 4, Insightful

      When it costs $2.5 billion to get FDA approval for a drug, 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).

    2. Re: Wait! by AutodidactLabrat · · Score: 5, Interesting

      The various drug companies spend 3 1/2 x as much on advertising and marketing as research, with MOST basic research paid for by governments and sold for pennies or given away for free
      Welfare starts at the top

    3. Re: Wait! by ShanghaiBill · · Score: 5, Informative

      The various drug companies spend 3 1/2 x as much on advertising and marketing as research

      The actual ratio is about 1.8 to one, but still an unreasonable amount considering all the taxpayer subsides that these companies receive. Much of that is spent marketing directly to doctors in ways that are nearly indistinguishable from just bribing them to write prescriptions.

    4. Re:Wait! by Interfacer · · Score: 3, Interesting

      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.

  2. value of human life by Anonymous Coward · · Score: 3, Insightful

    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.

    1. Re:value of human life by Altrag · · Score: 5, Insightful

      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.

  3. Nonsense by c · · Score: 5, Insightful

    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.

    --
    Log in or piss off.
    1. Re:Nonsense by blindseer · · Score: 3, Interesting

      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.

      --
      I am armed because I am free. I am free because I am armed.
    2. Re:Nonsense by Areyoukiddingme · · Score: 3, Insightful

      The problem is not that we put profit ahead of lives but rather that we haven't fixed health care so it properly comes under traditional market forces.

      When are you braindead idiots going to get it? When the choice is pay or die, there is no market! Goddamnit, how stupid can you possibly be? Markets require choice. Healthcare is not optional. End of discussion. Healthcare can not be managed via a market.

      If you've fallen off your roof and broken your leg and nicked your femoral artery, do you:

      a) Methodically call up each of the six independent healthcare providers in your area and ask them for their up-front pricing for repairing your artery and setting your broken leg, then carefully research their quality of care online, paying appropriate attention to their average outcomes specifically for treating a broken leg before calling back your provider of choice and making a reservation for them to come and get you; or

      b) Dial 911, frantically convey your situation, and pray the EMTs get to you before you bleed the fuck out?

      I'm gonna guess option b). And if in some fever dream, you really think you'd pursue option a), I hope you fall off your fucking roof.

      God DAMN I'm sick of you anonymous, cowardly fucks. You know your psychopathic ideas are wrong, or you'd post with an account and accept the downmods you deserve.

  4. Re:Stupid summary is STUPID by avandesande · · Score: 3, Interesting

    It's not a free market. By law, US consumers cannot import drugs from other countries. The $50,000+ dollars for hepatitis drug is under a thousand in India.

    --
    love is just extroverted narcissism
  5. Re:It's likely the PRICE, not the cost by MightyMartian · · Score: 3, Interesting

    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 world's burning. Moped Jesus spotted on I50. Details at 11.
  6. Not just "rare" diseases by PopeRatzo · · Score: 5, Informative

    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...

     

    --
    You are welcome on my lawn.
    1. Re:Not just "rare" diseases by rhazz · · Score: 4, Informative
      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

      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.

  7. The US subsidizes healthcare for the rest of the w by DatbeDank · · Score: 4, Informative

    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-...

    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.

  8. 75% Margins by Anonymous Coward · · Score: 5, Interesting

    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.

  9. Re:Attn Americans by amicusNYCL · · Score: 4, Interesting

    The problem is not that we aren't spending enough money. The problem is the combination of the pharmaceutical and insurance industries getting all of the money they can out of anything they have with no limits. Even generic drugs that aren't under patent production are many, many times more expensive in the US than in other countries (one example my wife gave me last night is $0.18 per pill abroad, versus $30 per pill here - for a generic medication). If the pharmaceutical companies are not actively engaged in collusion and price-fixing for generic medications then I would be shocked. Additionally, they spend so much money buying legislators that it is effectively impossible to get any legislation passed which would force a resolution to this issue by capping the price of medications or making it easier for additional companies to manufacture generic medications and compete with the established players. The free market is obviously not working correctly when every company making a certain generic medication sells it for the same amount, or when generic drugs which are readily available in other countries are not available here because they would compete with products from established companies. There is an opportunity there for a competitor to sell it for less and undercut the competition and make money, but for some reason that doesn't happen. If the free market is not allowed to work, and instead there is price gouging going on, then it sounds like legislation is required to correct that issue and bring drug prices down as a matter of law. If anyone thinks that such a thing would limit development or force companies out of business then I would invite those people to look at the P/L statements for the major pharmaceutical companies. A good start may be to outlaw advertising to consumers for pharmaceuticals, followed by a way to cap prices on medication based on metrics similar to those used by NICE.

    --
    "Our two-party system is like a bowl of shit looking at itself in a mirror." - Lewis Black
  10. I'm shocked the drug companies are so greedy by frovingslosh · · Score: 4, Insightful

    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.

    --
    I'm an American. I love this country and the freedoms that we used to have.
  11. The cost of drugs for rare diseases? by Lucas123 · · Score: 4, Insightful

    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.

  12. No it won't by rsilvergun · · Score: 5, Insightful

    The industry doesn't do much research. Not the expensive kind. They do a few clinical trials after the government has done the really expensive stuff (what's called "Basic Research", IIRC).

    Also, what you're seeing here with these rare disease drugs is the style of capitalism popularized by Bane Capital: Find something undervalued and buy it up then extract the value for yourself. Usually this takes the form of liquidating the company. But in these cases they're selling life saving medicine. It's literally a matter of life or death (or a life worse than death). These are small markets with a high barrier to entry where the customers depend on the product to live. This is exactly the sort of thing no decent society would leave in the hands of unregulated capitalism. American on the other hand...

    --
    Hi! I make Firefox Plug-ins. Check 'em out @ https://addons.mozilla.org/en-US/firefox/addon/youtube-mp3-podcaster/
    1. Re:No it won't by avandesande · · Score: 4, Informative

      The majority of drug makers spend more on marketing than R&D

      --
      love is just extroverted narcissism
  13. Government to the rescue; post-scarcity by mi · · Score: 3, Interesting

    Perhaps the government ought to produce those orphaned drugs themselves

    Could you cite a few examples of where the government proved to be more efficient at producing a product or delivering a service, than a privately-run firm?

    drug companies that are charging prohibitive amounts to citizens

    The hate towards the drug companies is misplaced — and whether they are sinfully greedy or not is irrelevant. The simple fact is, had they not existed, the drugs would not have existed — unavailable at any price.

    If only K of something — anything, from LeBron's sneakers to life-saving medicines — is available despite there being N people desiring it, then whichever way you pick to distribute it:

    • Lottery
    • Charge the highest price at which there are still willing buyers
    • Minorities first
    • Government employees first
    • Celebrities first
    • ...

      N-K people will still not receive it — and no amount of "outrage" will help.

      The only hope for the rest is that the second method — charging whatever the market will bear — will be chosen, because then the profits (however "obscene") may be used to produce more of the stuff... Incidentally, Capitalism is all about the second method and that is why we tend to enjoy an abundance of most things — to the point, where some people are already talking about "post-scarcity".

    --
    In Soviet Washington the swamp drains you.
    1. Re:Government to the rescue; post-scarcity by Actually,+I+do+RTFA · · Score: 3, Interesting

      Could you cite a few examples of where the government proved to be more efficient at producing a product or delivering a service, than a privately-run firm?

      The USPS. Providing libraries. Electricity (either government run or price set). Some would probably say healthcare in general (ex. Medicaid vs. private insurance).

      The simple fact is, had they not existed, the drugs would not have existed â" unavailable at any price.

      Most drug companies purchase completed drugs (often funded by the government), not develop them inhouse.

      The only hope for the rest is that the second method [Charge the highest price at which there are still willing buyers] â" charging whatever the market will bear â" will be chosen, because then the profits (however "obscene") may be used to produce more of the stuff..

      That's quite simply wrong across multiple dimensions. First, the highest price where there are still willing buyers is no where near proper profit maximization. Secondly, marginal costs on drugs are almost nothing, they're almost all fixed costs. There's no reason that there are a limited amount of pills any more than there are a limited number of CDs. Third, that "may" is troubling. Sure, it may be used for that, or simply pocketed. Since money is fungible, there's no real cause to suggest that it will directly fund the next round

      --
      Your ad here. Ask me how!
  14. Re:It's likely the PRICE, not the cost by geek · · Score: 4, Insightful

    You sound like a sociopath

  15. Re:It's likely the PRICE, not the cost by epyT-R · · Score: 3, Insightful

    ..and who decides who's a sociopath? The Local People's Social Justice Friendship House, which of course has absolutely no ideological bias against private business? I'll pass. It's just replacing one form of tyranny with another.

    There might be other solutions that could help. For example, if it is true that those companies are squandering large percentages of money on things like advertising, perhaps only allowing it to be funded from a set percentage of profits from sold product would be enough. This would hopefully limit the waste of research money on irrelevancies. While still not perfect, this is better than the witchhunt you suggested. Those never end well.

  16. Re:It's likely the PRICE, not the cost by WrongMonkey · · Score: 3, Funny

    ..and who decides who's a sociopath?

    Maybe a pharma company could develop some sort of test.

  17. Re:Attn Americans by amicusNYCL · · Score: 4, Interesting

    Here's a problem with your assessment, we don't have a free market with drugs.

    I know, that's my point. I said that if the free market was working, then we would see more competition and lower prices. The fact that we don't means that it is not working. The fact that no one is coming in to undercut prices on generic drugs that are not encumbered by patents is indicative of the fact that the free market is not working here. It might be evidence of collusion and price-fixing, or a situation making it impossible for competitors to enter the market.

    Prescription rules, if you want even a common drug for a chronic condition you will need a permission slip from a physician. Why? Can't people figure this out on their own?

    I understand that problem well. My wife is from Brazil, when we are there she can walk into any pharmacy and get whatever she wants, she can even consult with the people working there. There are certain limitations on what they're allowed to do, but they can sell her any of the drugs they have there. Many of those drugs are not even available in the US even though they do not have patents and are generic drugs. She can't get steroids that she needs for inflammation and other issues, and she can't even get the drug that works to get rid of her headaches without going to Brazil and getting it straight from the pharmacy without ever needing to see a doctor. She feels lied to after coming here and realizing that she cannot get the quality of care that she is used to from living in other countries, and the reason seems to be money, like so many of our other problems. So many people have their hands in the pie and what gets lost is actually providing good quality care to people who need it, even if it only means making drugs readily available like they are in other countries. She knows exactly what she needs, what works and what doesn't, and she simply can't get what she needs here. She feels lied to after hearing how great the US was supposed to be, and then getting here and realizing that it's all about money, and if someone can make money restricting access to health care then that's what they're going to do.

    More laws will not fix this problem. The problem is too many laws.

    Which laws do you think need to be removed in order to fix these issues?

    If you think that there is price fixing then I ask you to prove it.

    Really? You want evidence of a price-fixing scheme in a trillion-dollar industry? Well let me just hit Google, I'm sure there are signed documents online that will clear that right up.

    --
    "Our two-party system is like a bowl of shit looking at itself in a mirror." - Lewis Black