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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.

8 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?

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    1. 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

    2. 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.

  2. 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.

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  3. 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...

     

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  4. 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.

  5. 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...

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  6. 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.