As someone who has lived with spinal muscular atrophy for all 30 years of my life, I was perplexed and disappointed that the recent approval of Novartis’ gene therapy Zolgensma was immediately overshadowed by outrage over the drug’s price: $2.125 million.

The Food and Drug Administration’s decision was a pivotal day for those of us in the SMA community. Zolgensma, approved for children under 2, is the only one-dose treatment option for any category of SMA patients and has been highly effective in clinical testing so far.

Sure, it’s the world’s priciest drug. But instead of debating the level of financial profit that is appropriate for Novartis, let’s focus on the needs of patients. How are we going to get treatments for rare diseases if there’s not a financial incentive for doing it? Therapies are being developed because people think they can sell them for a profit. We don’t like to talk about it, but pharmaceutical companies exist to make money. Don’t we realize, though, that all of society profits from each disease we cure and each baby that is saved from SMA and other deadly diseases?

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As a professor of economics and finance, I believe that the cost-related complaints being thrown around social media are short-sighted. Shortly after Zolgensma’s price was announced, I even told a friend, “This is a good problem to have.” Why? It’s a twofold answer:

Competition. Zolgensma is the second treatment approved for SMA, following Biogen’s Spinraza. Now that there’s competition in the marketplace, that will lead to lower prices over the long term, especially if Zolgensma is approved for older SMA patients down the road. Roche currently has a daily pill called risdiplam for SMA in clinical trials. Should it also be approved, there will be further competition and downward pressure on prices for all three therapies.

Long-term value. The first two years of treatment with Spinraza cost around 50% of one Zolgensma infusion, but Spinraza treatments must continue for life at a cost of $375,000 each year. The four initial loading doses of Spinraza in the first year of treatment total $750,000. Over a 10-year period, the cost-effectiveness of Zolgensma is clear.

In terms of quality of life, one IV infusion of Zolgensma is definitely more palatable than multiple spinal infusions of Spinraza each year. I’m currently thrilled with the improvements in stamina and strength I’ve received from five doses of Spinraza. But I certainly wouldn’t miss the pain of having a needle inserted in my cervical spine every four months. (Due to my scoliosis, the less invasive, and less risky, lumbar puncture procedure isn’t possible. Other SMA patients have similar challenges.)

Critics of the eye-popping cost of Zolgensma need to take into account its long-term benefits. Zolgensma’s approval, and that of Spinraza, is the beginning of a new paradigm for treating SMA and for the fight against muscular dystrophy in general. Without any treatments, SMA would remain the top genetic killer of children under 2.

I’m not saying that cost is entirely irrelevant. I currently receive financial assistance through Biogen’s Spinraza copay assistance program. Biogen pays for any part of the drug and infusion procedure costs that my insurance company doesn’t cover (until I meet my $7,900 annual out-of-pocket maximum). In the end, though, I’m going to meet the annual out-of-pocket maximum no matter what due to other medical costs.

You might be skeptical of me saying $2.1 million isn’t too high a price while I receive help paying for Spinraza. A $2.1 million treatment for SMA, though, is no more ludicrous than paying more than $1,000 a month to rent a portable ventilator, especially when that one treatment will likely eliminate the need for the ventilator and many other costs associated with SMA care. I do hope Novartis will work with payers (public and private) as well as patients to maximize the number of children who receive Zolgensma.

We should not put a price tag on life, though. “$2.1 million is too much to spend for that baby’s survival” isn’t the kind of “value assessment” we should ever accept as a society. Instead, think about the parents who will no longer have to receive the heartbreaking news that my parents were given 29 years ago: “Your child has spinal muscular atrophy, and there’s nothing we can do. Survival beyond early childhood is unlikely.” The price of Zolgensma seems insignificant now, don’t you think?

Nathan Yates, M.S., is an adjunct economics and finance professor at Southern New Hampshire University and the founder of ForwardView Consulting.

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  • I could understand the price if the research wasn’t financed through federal grants and charities. Congress needs to focus on this instead of wasting time.

  • Given the use of insurance coverage, discounts, and patient access programs is there evidence to support an earlier commenter’s claim that Zolgensma is “out of the reach of most people”?

  • You’re right! It’s not expensive because it saves and changes life’s, but tell me how will a regular family be able to treat their kid, if they usually figure it out around 10 to 12 months old and you have to give it till 24 months? They have a year or less to get that amount, even more they live overseas, there’s a little girl in Brazil right now that has till February to raise R$ 9.300.000,00 the minimum wage there is R$900,00 is that a fare price!? Come on !

  • No one denies the need for profit of pharmaceuticals, I don’t think pricing the medicines so high, the ones already on the market nor the forthcoming ones, will be on benefit of all SMA patients. For sure there will be still thousands of SMA parents and patients that will translate the words “there is a 2.1 million dollars cure” into “we can’t afford it and our insurances won’t pay for them, so we are still condemned”. Also there is the ethics behind pricing so high this medicines that were initially funded with public money and donations.

  • How are we going to get treatments for rare diseases if there’s not a financial incentive for doing it?

    IDK ask Fredric Banting

  • The difference between American and European capitalism is that human lives are valuable in Europe.

    Except in Europe they do cost risk analysis to see if health care is worth it and they ration care because there are not enough doctors and nurses. The good news is, if you are rich in Europe you can get good care and if not you get the public system which is very poor indeed by American standards.

    • Hello, Can I tell you about the real world ?!

      “The good news is, if you are rich in Europe you can get good care and if not you get the public system which is very poor indeed by American standards.“

  • With all due respect.

    What your comment does is defend the practice of drug companies charging as much as they possibly can charge because people have no choice to save a life. Yes it is a good drug. And yet it is greedy and immoral and irresponsible for the drug companies not to make it so families who DON’T have your help can save their children too. And families should not have to go bankrupt for it. No one is saying it is expensive therefore it should not exist. That is a false paradigm. You claim that the fact that it is a necessary drug means that it is ok, should not be controversial, that it be out of the reach of most people. I feel you are the one missing the point.

    There is no reason, by your philosophy, that other drugs should not also be a million dollars. No problem with insulin or the epipen suddenly increasing in price by tenfold. As long as it is a good drug, no one should mind that it is unaffordable for most; that people go into bankruptcy for it. If they can force some people to pay for it while others die, it is good.

    Like all drugs, I assume a large part of the research was done for free by the US government and the patent was given for free to the pharmaceutical company. And yet there are no cost controls. You and I paid for that drug to be developed. But some, like you, will be able to get it. Many others will not.

    The fact is, it is immoral. Yes, it is the way capitalism works in this country, which is why you as an economist like it. American capitalism is immoral, and this is one of the best examples there is. If the drug companies have something someone needs to live, it is NOT ok for them to hold lives ransom for health just because they CAN.

    The difference between American and European capitalism is that human lives are valuable in Europe.

    • “Like all drugs, I assume a large part of the research was done for free by the US government and the patent was given for free to the pharmaceutical company. And yet there are no cost controls.”

      No, not at all. Read my other comment on how it costs an American biotech/pharma over $2.6 billion in research and development.

      “The difference between American and European capitalism is that human lives are valuable in Europe.”

      A laughable statement considering over 75 million Europeans lost their lives in two world wars. And Britain uses aborted babies to fuel their factories.

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