Newborn twins fight rare disease, family says it’ll cost $4.2 million to save their lives
www.wbtv.com
New parents in Kansas City are fighting to save the lives of their newborn twins. They said the boys have a rare genetic disease that will cost the family millions of dollars to treat.

america is so fucking based man

in any proper country that company at least gets forced to pay by the government then ordered to shut down forever due to wanton cruelty. all the employees get generous severance except whoever made that call. depending upon your view of carceral punishment there are a few ways to go with that guy.

    • catloaf@lemm.eeEnglish
      184·
      7 months ago

      I assume it doesn’t, and that includes R&D cost that can’t be spread across a large number of other doses.

    • cm0002@lemmy.world
      121·
      7 months ago

      While a healthy portion is, ugh, profit margin

      Often with these rare diseases, if there’s a cure or treatment for it often they require bespoke drugs. Sometimes with specialty equipment designed and built just for that drug (or drug family). Ofc with rare diseases, they probably only make like 5/year or some shit so they never scale well. In some cases (like if there’s a short shelf life), they even make the drug on an as needed basis, further increasing cost

      One of these specialty 2 mil dollar drug might still cost an actual 1 mil to make taking out profit margin/R&D recovery costs.

      • Viking_Hippie@lemmy.world
        72·
        7 months ago

        R&D recovery costs.

        Except the r&d was funded by the government. I highly doubt that they’re paying back those funds (though they definitely should), so, as usual, that excuse won’t fly.

    • JoBo
      3·
      7 months ago

      It doesn’t cost that much because the company are making a hefty profit, of course. And much more profit off it in the US as per usual, the NHS pays considerably less

      The deal struck [in 2021] with Novartis Gene Therapies, secures the drug for NHS patients in England at a substantial confidential discount and paves the way for the National Institute for Health and Care Excellence (NICE) to publish draft guidance recommending treatment with Zolgensma.

      The terms of the deal mean that some young children that currently fall outside the NICE recommendation criteria will also be eligible to be considered for treatment by a national multidisciplinary clinical team (MDT) made up of the country’s leading experts in the treatment of SMA.

      This means as many as 80 babies and young children could potentially benefit from the life-changing gene therapy a year.

      But profiteering aside, the number in the final paragraph is your answer. Up to 80 kids in the UK per year, so up to ~400 in the US, ~500 for the EU. It’s not a big market but the cost of drug development doesn’t get cheaper just because the number of cases is small, it gets more difficult and more costly. And there’s more than one drug company chasing the market.

      None of that is a defence of Pharma. But it is inevitable under capitalism. Eat the rich etc etc.

    • boatsnhos931@lemmy.world
      11·
      7 months ago

      It doesn’t cost that much to manufacture. The costs usually come from the time and people to research and develop this drug over years of trials playa…