The Food and Drug Administration (FDA) has recently approved
of a gene therapy drug for a muscular wasting disease prevalent in children. It
currently costs $2.125 million for a single dose. It is the most expensive drug
in the nation.
Approved in May, Zolgensma treats spinal muscular atrophy (SMA)
that affects children under the age of two. It is an autosomal recessive neuromuscular
disorder characterized by the weakening and wasting of skeletal muscles through
a defective gene resulting in the loss of motor neurons (Wurster & Ludolph,
2018). Infants who are affected have weak respiratory muscles, heart defects,
are unable to sit without support, and difficulty swallowing. Because of these
complications, most do not live past the age of two. SMA affects 1:10,000 babies
(Wurster & Ludolph, 2018).
Previous treatment involved Nusinersen (Spinraza™), which
was the first drug approved to treat SMA. It is administered via spinal
injections at four loading doses (days 0, 14, 28, and 63) with maintenance
dosing every four months afterward (Institute for Clinical and Economic Review,
2019). The cost of this drug? $125,000/ dose (Thomas, 2016). The cost of the drug
in the first year will be approximately $625-750,000 and $375,000 annually. It is
assumed that the patient will take Spinraza for the rest of their lives (Thomas,
2016).
Although the hefty price point of $2.125 million for
Zolgensma is insane, it is important to note that the patient will only need one
single dose of the medication. Of course, this is the justification for such
a large price tag. In our society, we are used to the idea of taking chronic
medication as opposed to having a one-time cure. For Novartis, the company that
manufactures the medication, it is seen as a high cost-effective drug. The lifelong
treatment of SMA with Nusinersen would cost approximately $4 million, so
Zolgensma may be worth the price from this perspective.
Of course, this raises the question, what happens when a
family cannot afford to pay for the medication? Gene therapy medications are
generally not covered under most insurance, so the money has to be out-of-pocket.
In a recent case, a baby, Axel, was diagnosed with SMA Type I and Louisiana
Medicaid has refused to pay for Zolgensma, TWICE, because he did not meet
the eligibility requirement because of his trach tube (Weiss, 2019). The
family is currently in the process of appealing this decision again. Axel is now
almost two years old and has lost his ability to smile.
His family has been managing the disorder, but
the injection of Zolgensma would prevent it from progressing. The Louisiana Department of Health does cover
all treatments of SMA that have been approved by the FDA, but they are denying
Axel a chance to live without being hindered by the lifelong treatment he has
to have (Weiss, 2019).
Regardless of eligibility or not, this case is an example of
how our nation places the ability to make a profit over the well-being of people.
Peter Bach, a health policy reviewer at Memorial Sloan Kettering Cancer Center,
equates our leniency to price increases to frogs who are slowly being boiled to
death (Stein, 2019). We never notice it until it is too late to stop it.
References:
Institute for Clinical and Economic Review. (2019). Spinraza
and Zolgensma for Spinal Muscular Atrophy: Effectiveness and Value [Final
Evidence Report]. Retrieved from
https://icer-review.org/wp-content/uploads/2018/07/ICER_SMA_Final_Evidence_Report_040319.pdf
Stein, R. (2019, May 24). At $2.1 Million, New Gene Therapy
Is The Most Expensive Drug Ever. Retrieved December 7, 2019, from NPR.org
website:
https://www.npr.org/sections/health-shots/2019/05/24/725404168/at-2-125-million-new-gene-therapy-is-the-most-expensive-drug-ever
Thomas, K. (2016, December 30). Costly Drug for Fatal
Muscular Disease Wins F.D.A. Approval. The New York Times. Retrieved from
https://www.nytimes.com/2016/12/30/business/spinraza-price.html
Weiss, B. (2019). Baby denied life-saving drug by Louisiana
Medicaid. Retrieved December 7, 2019, from WBRZ website:
https://www.wbrz.com/news/baby-denied-life-saving-drug-by-louisiana-medicaid/
Wurster, C. D., & Ludolph, A. C. (2018). Nusinersen for
spinal muscular atrophy. Therapeutic Advances in Neurological Disorders, 11.
https://doi.org/10.1177/1756285618754459
Your post raises a great question about the ethics of healthcare and making a profit. While I believe that healthcare should be universally accessible and not pose a great financial burden upon the consumer. The health care companies should still be able to turn a profit. When it comes to gene therapy medications - such as the one you described - it makes sense that the medication cost so much due to low demand and cost of production. However, refusing treatment due to eligibility requirements is when turning a profit becomes unethical. This unethical practice of raising medication prices and posing a financial burden upon consumers has become very prevalent. For example, the price of Lantus (insulin glargine) has rose by 252% since 2007 (Cefalu, et al., 2018), and two doses of EpiPen now cost between $650-$700 - two doses used to cost $100 in 2007 (Generics)(Kiersz, 2016). As consumers we should question the power that pharmaceutical companies have before it is too late.
ReplyDeleteReferences:
Cefalu, W. T., Dawes, D. E., Gavlak, G., Goldman, D., Herman, W. H., Nuys, K. V., … Yatvin, A. L. (2018). Insulin Access and Affordability Working Group: Conclusions and Recommendations. Diabetes Care, 41(6), 1299–1311.
Generics for EpiPen: What Are Your Options? (n.d.). Retrieved from https://www.drugs.com/article/epipen-cost-alternatives.html.
Kiersz, A. (2016, August 25). People are furious about the price of the EpiPen - here's how much it's increased in the last decade. Retrieved from https://www.businessinsider.com/how-much-price-of-mylans-epipen-has-increased-2016-8.
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ReplyDeleteIt seems as though the pharmaceutical companies are beginning to play games with the consumer- "how much is a life worth?" I am sure this drug is expensive to produce, but I wonder how much it is inflated for profit. Shouldn't the intention of this "life saving" medicine be to true, honest, and pure to that idea? What is the threshold between consumers and pharmaceutical companies when the inflation becomes too overwhelming. How can we help children like Axel and is family who desperately need this drug, but simply cannot afford it. It feels a bit manipulative, like pharmaceuticals and insurance are teasing patents at this point. It is interesting that it is a one time drug, which could alleviate the burden of chronic prescription bills and medical expenses. I don't think that the one time use justifies the cost of 2 million dollars. If a person of lower socioeconomic status needed this drug they would simply be pushed aside and have to face the reality of the mutation. This drug and cost is meant to create a further divide between the different classes and statuses. Essentially valuing one more than the other. Have studies shown the long term side effects? How much longer do the children live? Does it completely reverse the mutation and have no further complications?