Tuesday, May 21All That Matters

I see your $50K and raise you one $2.1 million dose of gene therapy for a baby in our NICU

I see your $50K and raise you one $2.1 million dose of gene therapy for a baby in our NICU

View Reddit by AmrwildcatView Source


  • ichoosetosavemyself

    Ok…how is someone able to just chill with a $2.1M box of medicine…taking pictures and posting to reddit?

    Jewelry stores literally have armed guards for less.

  • Brewe

    It’s almost like you guys are all bragging about how broken your healthcare system is.

    edit: listen up before you reply to this comment. This medication is not a vacuum case. unless you’re willing to defend the entirety of how the American healthcare system works, don’t bother. I know that this specific medication is rarely needed and therefore more expensive to produce, that’s not the point.

  • crew88

    From European company. Apparently it is $1.9m in Europe and some nations are protesting the price by threatening to not cover it. Others are stating they will only pay if it works.

    So how much are you actually laying OP? You are not paying out of pocket.

    Also, we forget, this is pretty amazing science. Its almost science fiction.

  • Raptorman_Mayho

    So the new thing I’m learning about American healthcare is the hospitals are being fucked as well as the patients. In places nurses are leaving because Wal-Mart pays more…WTF!

  • tienthinhbk

    A vietnamese friend of mine was offered to pay 2ml usd upfront to go to Japan and use this med for his son. But fot Japanese or us (japanese green visa) it is free.

  • hvgotcodes

    The gouging isn’t in a new drug that costs this much and alters the life of a newborn. And to OP I hope this cures your baby!

    The gouging is in medicines that are decades old that cost thousands and thousands of dollars, or in spending this much to prolong life a few weeks or months.

    Insulin costing thousands is criminal. Decades old chemo treatments that have price increases to match new cancer treatments is criminal. This price on this gene therapy is necessary.

  • WeirdMom

    Yay! Congrats! I commented about this one on the other post. My son received in clinical stages so we got it for free and didn’t get a cool box or vial.

    It is crazy expensive and there should be more transparency, but that isn’t the whole story. This is a gene therapy for a rare disease. Development and manufacturing are insanely expensive. Also it is a one time dose that lasts a life time.

    If we don’t put a high value on saving lives of people with rare diseases than we won’t. Without treatment most people with SMA die before age 2. This drug is also paving the way for curing or radically changing other types of genetic disease.

    Last but not least, the price tag is the same no matter what country you live in. More innovation needs to happen to make it affordable globally.


    Edited to add: American healthcare costs are a joke, but we actually take care of children and disabled children quite well. Most families will not pay much if anything for this drug. The difficultly is getting approval because insurance will try to deny due to the price tag. We have government insurance called BCMH with a high income cap that covers diagnosis and treatment. the income cutoff for Medicaid is higher for children than adults (our kids had it when we made about 60K for family of four).

    When we didn’t have Medicaid BCMH covered the out of pocket expenses until the cap was hit on our regular insurance. Now my son has a Medicaid waiver, so as long as we don’t become millionaires we don’t pay anything.

  • symolan

    former therapies for spinal muscular atrophy cost about 750k in the first year and 350k annually afterwards for the rest of the patients life.

    The 2.1m here is – if successful – a one time and healed thing.

    I don’t want to comment on the profit margin on it, just that the argument was that over the course of the patient’s life, the 2.1m was actually cheap compared to former therapies.

  • iamtwinswithmytwin

    Spinraza is a pretty remarkable wonder drug. My genetics professor in med school worked on the team that figured out the first pre-natal screening assay for it and got it on the NYS pre-natal screening panel.

    Imagine a disease where your infant is born, the damage already done, most likely quadriplegic and needed respiratory support. Almost always fatal.

    If you can detect it early enough (this the pre-natal screening) you can give it in-utero and that child will be born and grow up with mostly the same mobility as normal.

    Our professor was talking about how this is a good exam of how wonder therapies aren’t very good if you can’t detect diseases early. Sure a baby born with SMA who receives this will do better but most probably will utilize mobility aids and need OT and PT for the rest of their life. But if you get to them early you can save their life.

    And then she said that 3yrs prior they had successfully detected and administered Spinraza in utero and she flicks on a video of a toddler toddling across the room to hug her at a follow-up.

    Not a dry eye.

  • neospacebandit

    Sounds crazy until you compare it to the lifetime cost of Spinraza injections. My wife is a child neurologist and helped get SMA on the Ohio newborn screening and having that early gene therapy has been incredible for many of her young patients.

  • jawnlerdoe

    I would like to add a nuanced take here as someone who does limited work with gene therapies; it costs billions to develop these drugs, the population that can use them is very small, and each dose requires, likely, millions to Produce. Most gene therapies are only offered because of federal orphan drug programs and their cost is justified by how much hard work is required to make Them.

    Not saying that cost should be passed to consumer though.

  • Meme_Pope

    People in the comments are honestly brain dead talking about “it only costs $20 to manufacture. The system is broken”. First of all, this is likely billed to the insurance company and OP’s cost is minimal. People who are poor and on Medicaid pay nothing. More importantly, it’s mostly the research and development that the cost is covering.

    Developing a gene therapy costs billions of dollars and the rarer the condition, the more the drug costs to break even. Otherwise, nobody would ever bother finding cures to rare diseases. After a company develops a medication, they have a patent on it for 20 years, after which it can be produced generically and the cost drops >95%. This works out to more like 10 years, as half the lifespan of the patent is spent in development.

    If you want to get mad at drug prices, there are better things to be mad about. Be mad about the price of insulin, where the patent has long since expired, but all the companies that make it have colluded to astronomically raise their prices together.

  • azcat92

    You do know this is a one-time drug right? This drug is an actual cure where they never have to take medication again. Most drugs people take over a lifetime would cost more than this if you add up all of the doses, but this is an actual fucking cure. What is that worth?

  • writerVII

    These numbers sound really expensive, but then I can’t help but think – how many years work of PhD-level scientists, what expensive instrumentation, expensive reagents were required to come up with the idea, test the biology, test the drug etc? How many other drugs failed (that were also a large investment, and I think the failure rate in all stages of testing and clinic are quite high)?

    So too afraid to ask: I have a feeling that the extremely high prices are unavoidable if the drug is for a very rare disease – how else can the cost be recouped for a company to at least break even?

  • GirlsLikeStatus

    My friend worked on this drug. I bearly ever saw her. The amount of research that went into this was astounding. The patient stories made me want to cry. It was so expensive to discover and is so expensive to manufacture.

  • BlursedOut

    I know I’m going to piss a lot of people off but how much is your health worth to you? How much is your quality of life worth?

    $2.1 million is a ton of money for a gene therapy drug. The price doesn’t reflect just the company’s profit it reflects the time, effort, equipment, personnel, and multiple government approved studies needed to make the drug usable.

    What costs more, the 1x shot of gene therapy or the years of physical therapy that are never curative but instead are a panacea? What about the mental and emotional health of the patient and their family, can you put a price on that?

    What does bother me is when a pharmaceutical company takes a drug coming off patent and tries to squeeze every last ounce of profit from it by reconstituting the formula by adding some sort of benign filler or coating then charging more for it because it’s “new and improved”.

Leave a Reply

This site uses Akismet to reduce spam. Learn how your comment data is processed.