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.

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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.

  • Having the cure at such high price is just like not having the cure at all. If the miraculous treatment works, you can happily work all your life to pay for it. What quality does that life have anyway? Having a 2 million $ debt for the rest of their lives – they shall just work, not eat, sleep or enjoy it, just work for it. What if the treatment isn’t successful? They remain in debt and have no way to pay it. The price is the same, whether you live in the states or some small European country. It is pure cruelty of the industry. Let the rich live and the poor die

  • You ask this question “How are we going to get treatments for rare diseases if there’s not a financial incentive for doing it?”
    I’m sorry that you feel that there are no people or organisations left on this world which are willing to do something for other people just because they want to help, but you are wrong and you should keep such opinions to yourself because maybe some will believe you, or think that it’s normal as you suggest.

    • Hi, Mircea ~

      You said:
      “I’m sorry that you feel that there are no people or organisations left on this world which are willing to do something for other people just because they want to help … ”
      My response:
      Individuals can and do work for free as volunteers to help others, but organizations cannot do that.
      They must get money somehow to keep the lights on and pay their employees — and that is just for the non-profits and charities.
      Corporations in addition have an absolute fiduciary duty to their shareholders to earn a profit for their shareholders’ investments.
      If they cannot repay the stockholders, the corporation must cease to exist.
      For one thing, it would not be able to raise operating money through the sale of stocks because no one would buy stocks that do not pay a return on the investment.

      Furthermore, very large-scale problems cannot be addressed at no cost by some well-meaning volunteers.
      Probably any problem-solving group larger than a no-kill animal shelter needs an army of paid professionals.

      You said:
      ” … but you are wrong … ”
      My response:
      I’d like to see the details of this opinion.

      You said:
      ” … you should keep such opinions to yourself … ”
      My response:
      If your opinions are valid, they will be able to withstand a challenge.
      Your fear of contradictory ideas is a demonstration that your opinions are weak and wrong — or you would not have to wipe out contradiction. You’d be able to answer it.

      You said:
      ” … maybe some will believe you, or think that it’s normal as you suggest.”
      My response:
      Yes, that is exactly what you are afraid of.
      Your notion that corporations are supposed to operate with no income, but just on “wanting to help others” is exposed as silly and senseless.
      No wonder you fear letting other people hear your ideas get contradicted.
      —————–

      If you want, you can sign up for a course in basic economics at a community college near you.
      If you approach the professors asking permission, they will probably agree to let you audit the course for free. (You don’t get college credits.)
      The classes will explain why corporations need to make money, and even why they need to make a profit over their expenses.

  • @Martin Siry

    Yet it turns out that allowing corporations to compete against one another in order to earn money is the greatest inducement to productivity, invention, and innovation that the world has ever known.

    But since you hate capitalism, I assume that you will now not use any product or service created by capitalism.
    Modern medicine, electricity, modern manufacturing techniques and their products, the internet, the computer … you will forgo these products and services on the grounds that free enterprise in capitalistic societies created them … right?

  • Hooray for you and your eloquent tactful but to the point comment i had 2 brothers with sma and watched the disease take it course and the answer was always their nothing we can do but supportive care u know the drill physical therapy etc my brothers had a dry sense of humor and said sis when i die they will fi d a drug for this my first brother died and my secod brother died while doing paperwork for a drug trial both of my brothers worked contributed to the community and paid their taxes we spend millons on drug rehab and most addicts rehab 9 times have multiple hospializations for infections et etc but that politicallly correct the general population doesnt understand orphan drugs or other ecpensive drugs or how the research findings and their drugs help othet diseases we spend millions in kidnettransplants and dialysis for diabetes who were totally noncompliant with their diet and meds but once again that expendiature is fine u and my brothers did nothing except get a bad roll of the genetic dice …. competition drives prices down and your improvement promotes the drug i hope u continue to remain stable and or improve and keep making your comment

  • I am sorry you feel it that way.
    The research was done in France, thanks to the generous donations from people who wanted to help and thanks to subsidies from the government, in other words, from the taxes French people paid. It costed beteween 12 and 15 millions euros.
    But because of politic, this laboratory (Généthon) does only research, not production. Otherwise, the price would have been far less than this, because as long as it was in the hands of this laboratory, it was not about profit, but about life. They sold their research to a start-up for about 13 millions euros. Because what was the most important for them was that the drug become available for all. Then, there was some tests to finance, some speculation, and Novartis owns it now. And sells a single shot for about 2 millions euros.
    Yes, it is indecent. And no, you’re wrong, we don’t need them to do research and find new medications. They only seek profits. They don’t even do the research themselves, they’re nothing but greed.
    This is just the story of an already big fish who bought a small fish who took the risk to buy and test the production of a smaller one who did all the work and couldn’t go further because of government.
    What’s hard today, is that the people who made that kind of profit are “friends” whith politics. That’s why they don’t want to autorize Généthon to go all the way and make the drug.
    Money rules, while we die.
    Sorry for my english, I’m not use to write/talk in english.

    • Very well said. Instead of donating our money to various health causes, a protest would be easier, but what difference would it make?

    • @Sally

      Really no price is too high?

      Is saving lives to be potentially infinite in costs?

      Will you pay my share for me?
      I don’t want to pay, say, $20,000 a month to save the lives of some strangers, so I’ll just be sending that tax bill over to you.
      You can add it to your own tax bill.

      After all, you shouldn’t put a price tag on life, so here’s your chance to put your money where your mouth is.

    • @Sally

      You said I misinterpreted your comment.
      Sorry about that.

      May you please state your meaning again in such a way that it cannot be misinterpreted?

    • @Anna Graystone

      I repeated the quote from the story, and said that’s the question. Which it is, and a debate. Should be a debate for the country, imo, rather than pretending we need never have it.

      I would come down on the side that as long as resources are public, and are not unlimited, the US like virtually every other country should establish and adopt cost-effectiveness limits.

    • @Sally

      Of course I understood what you posted — that it was a quote and that you thought it should be debated.

      That’s why I posted a point within the debate.

      Possibly you intended to have a debate as long as it did not involve you.
      In that case, you may consider it rhetorical only that I addressed my response to you.
      ————

      You said:
      “I would come down on the side that as long as resources are public … ”
      My Response:
      I don’t see why I should be taxed to pay for the health care of others.
      Example:
      In a city, dogs cause certain municipal expenses (dog control officers, police and traffic problems, etc.)
      These costs are covered by DOG OWNERS (not the general public) by charging owners a dog license fee.
      Example:
      Most people don’t go hunting or fishing, so the government covers the costs of the Fish and Game Service by charging a “user’s fee” for hunters and fishers to pay for a license.
      Not everyone is going to need a $2million treatment, so the cost is covered by the people who do need it (through their insurance), not shifted on to everyone.

      Here’s a quote for you:
      I previously said that every time I am taxed to pay for the medical care of someone else, that is one more dose of my own meds that I can now not afford.
      Here’s another quote, as long as you’re so fond of being taxed to pay for other people’s health care:
      “Will you pay my share for me?
      I don’t want to pay, say, $20,000 a month to save the lives of some strangers, so I’ll just be sending that tax bill over to you.”
      ———————-

      You said:
      … and [resources] are not unlimited … ”
      My response:
      I am interpreting “resources” t mean “costs.”
      Of course costs are unlimited.
      That’s the essential problem.

      You said:
      ” … the US like virtually every other country should establish and adopt cost-effectiveness limits.”
      My response:
      What does this even mean?
      What is a “cost-effective limit”?
      How do you “establish” one?

      Does it mean “putting a cap on how much the taxpayer will pay for any one treatment”?

      Is this what “virtually every other country” does?

      Then what?
      When the treatment costs, let’s say, $2 million per dose, do we refuse to pay it?
      Rich people will pay for their own treatment, and poor people will die.

    • @Anna Graystone:

      This is tiresome.

      Resources are assets, not costs.

      “Cost effectiveness” gauges bale of purchase against cost of purchase.

      Why would you ever assume I am “so fond of being taxed to pay for other people’s health care?”

      You seem to have a hard libertarian line here. You also seem to have a comprehension problem.

      Over & out.

    • @Sally

      You said:
      ” … as long as resources are public, and are not unlimited, the US like virtually every other country should establish and adopt cost-effectiveness limits.”

      My response:
      1) What “resources” do you have in mind which are “public”?
      2) Which resources do you mean which are “not unlimited”?
      3) What does this even mean?
      ” … cost-effectiveness limits.”

      You defined “cost effectiveness this way:
      “Cost effectiveness” gauges bale of purchase against cost of purchase.”
      > What is a “bale of purchase”?

      Would you be able to provide a working example of how the US should “establish and adopt cost-effectiveness limits”?

      When it comes to the problem described in this article:
      “Should the general public be taxed to pay for the medical care of everyone?”
      Can you describe how establishing and adopting a bale of purchase against the cost of purchase would work?

      Would health care be paid for by taxes?
      Who would be in charge of establishing and adopting a bale of purchase against the cost of purchase?
      ————-
      Sally, my friend ~
      You almost sound like you are starting to become a little incoherent.
      But your ad hom attacks come through all right. LOL!

    • @Anna Graystone

      Autocorrect type. Value (not bale).

      Most of American medical care is paid for by the federal government. That is just the way it is.

      As to cost effective analyses, look to Europe or other countries where they are done regularly, and by which drugs are approved or not.

    • @Sally
      You said:
      ” Most of American medical care is paid for by the federal government. That is just the way it is.”
      My response:
      When you say “the federal government,” that is a code word for “the taxpayers,” right?
      ————-

      You said:
      “As to cost effective analyses, look to Europe or other countries where they are done regularly, and by which drugs are approved or not.”
      My response:
      So is it worth it to pay $2 million per dose to save this person’s life?

      > Upon what principles or morals does this decision rest?
      > Who is the agent of the government tasked with making this decision?
      An unelected functionary or bureaucrat?
      Religious leaders? University philosophers? Medical ethicists?

      What if it costs $2.5 million? Or $3.5 million? Or $100 million?
      What is the cut-off point after which we say “Sorry. Your life just isn’t worth this price.”

    • Yes, taxpayers.

      Please refer to my first post. Is America ever going to be ready to have this adult discussion? Because, behind the scenes, by those pulling the strings, it keeps getting avoided.

  • @Danica ~
    This is an excellent comment.

    The problem is compounded by the unfair practices of foreign countries who do not pay their fair share of the costs of developing new drugs.
    Instead, they buy one dose and reverse-engineer it so that they can sell it merely for the cost of its chemicals.
    Then they brag about how little drug costs are in their nation.
    Canada, India, China, and other countries with the capability are the main players in this form of theft.
    Many other countries just make poor-quality counterfeits.

    This is a form of patent violation that is hard to address because they take the precaution of changing the formula just a little.

    The United States needs to impose money-raising economic sanctions (such as stiff tariffs) on these thieves and turn the money over to the drug companies, who can use it to reduce the per-person costs of American customers.

    The financial penalties need to be much higher than the total of their gains to reduce their motive the next time.

    • Who did it You said ?
      Read the comment
      Khail made. Maybe you’ll open Your eyes.

      I am sorry you feel it that way.
      The research was done in France, thanks to the generous donations from people who wanted to help and thanks to subsidies from the government, in other words, from the taxes French people paid. It costed beteween 12 and 15 millions euros.
      But because of politic, this laboratory (Généthon) does only research, not production. Otherwise, the price would have been far less than this, because as long as it was in the hands of this laboratory, it was not about profit, but about life. They sold their research to a start-up for about 13 millions euros. Because what was the most important for them was that the drug become available for all. Then, there was some tests to finance, some speculation, and Novartis owns it now. And sells a single shot for about 2 millions euros.
      Yes, it is indecent. And no, you’re wrong, we don’t need them to do research and find new medications. They only seek profits. They don’t even do the research themselves, they’re nothing but greed.
      This is just the story of an already big fish who bought a small fish who took the risk to buy and test the production of a smaller one who did all the work and couldn’t go further because of government.
      What’s hard today, is that the people who made that kind of profit are “friends” whith politics. That’s why they don’t want to autorize Généthon to go all the way and make the drug.
      Money rules, while we die.
      Sorry for my english, I’m not use to write/talk in english.

  • Really well said piece. Look people- development of this type of drug costs money- and it is NOT funded by public funding or the government! A drug company takes a MASSIVE change and financial gamble to create a drug/vaccine. Most products do not make it through the clinical trials. It is a rare gem that is able to pass through all of the tests and make it to market. Plus money has to be set aside for any side effects which are not accounted for during testing (I recall male breast enhancement with the Risperdal?). This is not about being cruel or inhumane or not caring for lives. In fact, most research and development for drugs is carried out by the USA. If you want to reduce the financial incentives (or impose ridiculous, almost punitive taxes) then, shoot, they won’t develop them. And everyone will lose.

    • Yeaaaa that’s right…New Bentleys , trillion dollar houses for owners of big medical companies. Who cares that millions of American citizens bankrupt every year because of health problem. Let them pay for sick children millions of dollars. Haven’t you read comment that Khail made
      DECEMBER 30, 2019 AT 10:22 AM
      I am sorry you feel it that way.
      The research was done in France, thanks to the generous donations from people who wanted to help and thanks to subsidies from the government, in other words, from the taxes French people paid. It costed beteween 12 and 15 millions euros.
      But because of politic, this laboratory (Généthon) does only research, not production. Otherwise, the price would have been far less than this, because as long as it was in the hands of this laboratory, it was not about profit, but about life. They sold their research to a start-up for about 13 millions euros. Because what was the most important for them was that the drug become available for all. Then, there was some tests to finance, some speculation, and Novartis owns it now. And sells a single shot for about 2 millions euros.
      Yes, it is indecent. And no, you’re wrong, we don’t need them to do research and find new medications. They only seek profits. They don’t even do the research themselves, they’re nothing but greed.
      This is just the story of an already big fish who bought a small fish who took the risk to buy and test the production of a smaller one who did all the work and couldn’t go further because of government.
      What’s hard today, is that the people who made that kind of profit are “friends” whith politics. That’s why they don’t want to autorize Généthon to go all the way and make the drug.
      Money rules, while we die.
      Sorry for my english, I’m not use to write/talk in english.

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