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.

  • The Liver@lemm.ee
    38·
    6 months ago

    Why does the medicine cost 2.1 million in the first place? Is it just price inflation or it’s made of antimatter or something?

    • state_electrician@discuss.tchncs.deEnglish
      38·
      6 months ago

      From https://www.drugs.com/medical-answers/zolgensma-expensive-3552644/

      Zolgensma is a one-time-only gene therapy treatment for children aged less than two years with spinal muscular atrophy (SMA) that costs $2.1 million for the single treatment. The reason Zolgensma is so expensive is because that is the price Novartis has decided it is worth because it “dramatically transforms the lives of families affected by this devastating disease” and the claimed cost of bringing new drugs to market. But this price is not without controversy. For a start, the early development of Zolgensma was financed by the National Institutes of Health and several charities devoted to finding treatments for SMA, including many U.S. charities such as Sophia’s Cure, Cure SMA, Getty Owl Foundation, Fighting SMA, Jadon’s Hope Foundation, the Gwendolyn Strong Foundation, and Miracle for Madison. Many of these charities use donations by patient families and friends to subsidize research and clinical trials into new medicines for SMA. The CEO of Novartis, Vas Narasimhan, argues that gene therapies represent a medical breakthrough in the way that they offer hope of a cure for deadly genetic diseases with a single dose. In some cases, the alternative is a multi-dose treatment with incremental improvements. For example, an alternative to Zolgensma is Spinraza that is taken four times a year for life. The list price is $750,000 for the first year and then $350,000 per year after that, so about $4 million a decade. But how do they calculate the actual cost? Many companies use a value-based pricing model which calculates the cost based on how many years of life gained as well as taking into account the effectiveness of the drug. But this means putting a price on how much a life is worth, as well as ensuring the company makes a reasonable margin on their investment. Little is disclosed about the true cost of bringing a new drug to market, and Novartis didn’t develop Zolgensma but acquired it through the $8.7 billion purchase of US firm AveXis.

      So there is no tangible reason why it’s this expensive.

      • citrusface@lemmy.worldEnglish
        17·
        6 months ago

        “Because we said so and if you don’t want your kid to be a fucked up cripple loser you’ll fucking pay us, bitch.”

        • Novartis
    • Liz@midwest.socialEnglish
      16·
      6 months ago

      I have no fucking clue about this particular medicine, and Americans are getting an absolutely raw deal on healthcare

      BUT

      Non-greedy reasons that can raise the price of a drug:

      1. manufacturing costs. Maybe the ingredients are expensive. Maybe the synthesis has a very low yield. Maybe storage is very expensive. Maybe storage is such a bitch the drug needs to be made on-demand. Maybe storage is straight-up impossible so the drug needs to be made on-site.

      2. Low demand. If very few people need the drug, you can’t spread out the cost of R&D or manufacture. Furthermore, it’ll force you to use low-volume manufacturing methods, which will be more expensive. It might be so low volume that you literally just pay a chemist to synthesize the drug on a bench top, which could take weeks of labor, depending on the synthesis.

      3. delivery mechanism. Suppose the drug itself is relatively cheap, but it needs to be delivered by a long-term release capsule implanted in your spleen. Suppose it needs to be delivered by IV drip continuously for a week. Suppose it needs to be taken under direct supervision for some reason.

      Probably other shit, it’s been a while since I studied where healthcare costs come from.

      Edit: lol, sounds like the justification the pharma company is going with is “fuck you, is not a child’s life worth everything you’ve got?”

      • AeonFelis@lemmy.worldEnglish
        64·
        6 months ago
        1. FDA regulations. Specifically - the insane amounts of tests and trials required to approve a drug.
          • AeonFelis@lemmy.worldEnglish
            14·
            6 months ago

            I mean the part where we make sure the medicine that can potentially save your otherwise doomed life doesn’t give you a mild rash.

            • Mirshe@lemmy.world
              4·
              6 months ago

              Historically, large-scale withdrawals of drugs from markets ONLY occur, and large-scale marketing ONLY is barred when the side effects are deemed dangerous enough to not risk at any significant percentage. If you look through the list of withdrawn drugs throughout the world, almost all of them are withdrawn for either abuse reasons, or significant side effects like organ toxicity, serious risk of overdose even inside prescriber control, carcinogenicity, or neurological reactions (like some fungicides/bactericides causing blindness/deafness even when used properly).

              SOME of these have been returned to market (like thalidomide) under very strict guidelines, used for very strict reasons (thalidomide is used for leprosy and multiple myeloma treatment now in certain situations, in combination with certain drugs to help reduce teratogenicity). Others, which were formerly seen as helpful, have been removed from markets because of newly-found dangers involving them (like Zantac, which was found to spontaneously break down into a carcinogenic compound).

              • Liz@midwest.socialEnglish
                3·
                6 months ago

                Zantac? No shit? Well I’m sure glad all those zantac commercials I used to see didn’t work on me!

              • AeonFelis@lemmy.worldEnglish
                11·
                6 months ago

                This is not about the cost of withdrawing or barring drugs as much as it is about the cost of running all these tests and trials. And yes, drugs can potentially have terrible side effects, but not being able to afford the drug can also have terrible results.

                If the FDA requirements were much less strict, the drug company would have had to spend much less on R&D. That, of course, would not be enough to lower the price - but the other effect of cheaper R&D is that it’s easier for other companies to compete, and competition does drive prices down. The the point either the mother could either afford it herself, or the insurance wouldn’t be so stingy about paying it.

                Now, of course, less strict requirements also mean we know less about the drug’s safety and efficiency. Let’s say that, because of the lack of knowledge, we assign a 50% probability for the drug to kill the patient and even if it doesn’t we only assign 50% probability for it to work (that does not mean it killed half the test subjects and failed for half of the remaining ones - just that we didn’t test enough to get significant results that say otherwise, and these are the worst case estimates under our lack of data). That means, that there is only 25% chance for each one of these twin babies to survive if they take the drug.

                Which is better than the 0% they get now, being unable to afford it.

    • You999@sh.itjust.works
      11·
      6 months ago

      Definitely not because Novartis is trying to recoup the 8.7 billion they spent on AveXis to acquire Zolgensma…

      • lolcatnip@reddthat.comEnglish
        33·
        6 months ago

        And denying it to people who desperately need it but don’t have that kind of money is helping them recoup the costs how?

        • You999@sh.itjust.works
          51·
          6 months ago

          Novartis isn’t the company who are denying it, that would be the insurance company Mosaic’s Health Care Trustees who aren’t covering it because it’s too expensive as stated in the article.

          Insurance companies will do anything possible to get out of paying for specialty tier medications from using ‘step therapy’ where before they will approve the medication your doctor approved you must first try and fail all the available medications starting from least expensive. Insurance companies typically also charge a presentage on speciality drugs for the copay as a deterrent from being prescribed those medications. Currently the advocacy side of some large disease-focused charities such as the arthritis foundation are trying to get congress to federally ban step therapy and set a cap for specialty tier drug copays like they have for the other drug tiers.

    • Alex@lemmy.world
      6·
      6 months ago

      Shit legislation with lack of price-negotiation through collective bargaining, is the main issue, costs land at the individual enduser in the end. They could’ve likely gotten half the money and save the twins but instead they’ll get none with that pricetag calculated from putting a number to the value of a human life - that makes most insurers pull out the fineprint and drop coverage overnight…