Zolgensma: Saves Children with SMA from Certain Death, but Costs $2.2M Per Dose

A parent would do anything to save their child– Elissa and Jacob Schultheis were no different. They were willing to sacrifice anything to save their son, Jackson Schultheis, who was born with a rare genetic disorder called spinal muscle atrophy. Spinal muscle atrophy is characterized by weakness in muscles due to a loss of motor neurons that control muscle movement (“Spinal Muscle Atrophy,” 2022). There are multiple types of spinal muscle atrophy, but the most common is type 1, where the life expectancy is less than 2 years in children. Jackson was diagnosed with type 1 spinal atrophy when he was around 6 months of age, when his parents noticed that his legs were not functioning properly and reported it to a physician (Court, 2019).

In the past, situations like this were hopeless and all the parents could do was to make the child’s life comfortable until the duration of the disease. the child’s life as happy as possible and wait for them to die. Because of continued advancements in medicine and technology, treatments have become available for spinal muscle atrophy, resulting in a drastic increase in lifespan. For instance, gene therapy is a possible treatment option due to its compatibility with a genetically inherited disease like spinal muscle atrophy. A treatment that in particular, has outstanding results in increasing life expectancy and overall quality of life is Zolgensma. Zolgensma was developed by AveXis in 2010 and is a gene therapy drug that replaces the corrupted SMN1 gene with a working copy (Swanson, 2019). This greatly alleviates the symptoms of spinal muscle atrophy and improves the overall quality of life. The news of this treatment gave hope to the Schultheis family and they were determined to get Zolgensma for their son. Unfortunately, this proved to be exceedingly difficult due to the price tag of the drug: 2.1 million dollars, making it the most expensive drug on the planet. The Schultheis family desperately tried to get their child Zolgensma, but health insurance refused to cover the cost and the family could not afford such a price. On top of that, Zolgensma needs to be given sometime before the age of 2 and it is a one-time treatment. Jackson’s condition continued to deteriorate, decreasing the chance of recovery with each passing day as the drug needed to be given as soon as possible as the damage from spinal muscular atrophy accumulates over time and can be irreversible. Time was not on their side and the family struggled to get their child the treatment. When Jackson was 18 months old, he was denied treatment by his health insurer twice.

After their failed efforts with the insurance company, Jackson’s parents sought the media to spread awareness of their unfortunate circumstance. It took multiple media appearances and protests from families for the insurance company to agree to the cost of Jackson’s treatment. Jackson received the treatment when he was 19 months old, only 5 months short of the drug deadline (Court, 2019).

Jackson was one of the lucky cases of spinal muscle atrophy treatments. Spinal muscle atrophy affects 1 in 11,000 infants and many of them don't make it to their second birthday (Children’s National, n.d.) . After considering the severity of spinal muscle atrophy, I do not understand how a company can place such an unreasonable price on a life-saving treatment. Zolgensma has become more affordable in the sense that insurance companies are more likely to cover the cost, but it is still not guaranteed. There are many cases in which insurance companies decline coverage and families, unable to afford the treatment, have to see their child die. Parents should not have to feel so hopeless especially when a treatment is available. Whatever the cause for insurance company to reverse the decision for Jackson does not matter; what matters is that families should not have to suffer and weigh their child’s life over a ridiculous price tag.

Zolgensma is an amazing genetic therapy that has unimaginable results. However, that should not tempt pharmaceutical companies to place such a price tag on a life-saving drug. Novartis, one of the directors of AveXis, believes Zolgensma’s price tag is appropriate due to the drastic improvement in the quality of life of families that experience spinal muscular atrophy. Fookes (2020) review explained the following: “Many companies, such as AveXis, 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” (p.1).

Treatments such as this unethically place a value on human life and ensure companies make a reasonable profit. Companies should seek profit to a reasonable degree that does not play with the desperation of parents who are willing to risk anything to provide for their child. The argument that these companies make is that drug development costs billions of dollars and reducing the price will delay the development of other treatments. This is a valid point, but what use is a drug if patients are not able to access it? Many people cannot afford a 2.1 million dollar treatment and they must rely on insurance companies. Even with Zolgensma’s amazing results, insurance companies are not always convinced that they should cover the treatment because there is still some significant risk that is present. Zolgensma does not cure the original defect that caused spinal muscle atrophy, but instead treats the symptoms to produce a higher quality of life. Due to this effect, insurance companies don't always count drugs like Zolgensma as a medical necessity which means they can deny treatment. Without the help of insurance companies, many middle or lower class families are not able to afford Zolgensma and must accept their child’s demise.

Zolgensma does not need to be priced at 2.1 million dollars. For instance, Institute for Clinical and Economic Review, a research group that evaluates drug pricing, claims that Novartis could reasonably set the price of Zolgensma in the 310,000 to 900,000 dollar range (Pipes, 2020). This range is still very expensive but is accessible to a lot more families. Completely covering all medical treatment is idealistic, but medicine should not be a source of unreasonable profit from another’s misfortune. Treatments cannot be viewed the same as materialistic items in a competitive market. When giving such a price tag, companies should also provide reasonable options that do not lead families to bankruptcy.

References

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Insurers won't pay for a $2.1 million drug for kids, and parents say they're running out of Time. Business Insider. Retrieved April 17, 2022, from https://www.businessinsider.com/health-insurance-companies-deny-kids-with-sma-gene-therapy-zolgensma-2019-7

Fookes, C. (2020, September 14). Why is Zolgensma so expensive? Drugs.com. Retrieved

April 17, 2022, from https://www.drugs.com/medical-answers/zolgensma-expensive-3552644/

Pipes, S. C. (2020, February 7). How this gene therapy drug earned its $2.1 million price tag.

Fortune. Retrieved April 17, 2022, from https://fortune.com/2020/02/07/zolgensma-high-drug-prices/

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Swanson, J. (2019, May 24). FDA approves Avexis' zolgensma for treatment of spinal

muscular atrophy in pediatric patients. Muscular Dystrophy Association. Retrieved April 17, 2022, from https://strongly.mda.org/fda-approves-avexis-zolgensma-for-treatment-of-spinal-muscular-atrophy-in-pediatric-patients/?_ga=2.199439854.1597479129.1650249806-1011086227.1650249806