Imagine how it feels knowing that just one dose of a medication could change your child's life, but it costs more than $2 million.
It sounds like something from a cruel futuristic film, but this is the very real problem facing patients and their parents today.
The price of medication has long caused upset, with some Americans racking up thousands in debt for treatment.
The drug in question is called Zolgensma, and it is a one-time-only gene therapy treatment for children under two years old.
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It is used for the treatment of a condition called spinal muscular atrophy, SMA for short.
It works by treating the genetic root cause of SMA, as it replaces a 'missing or nonworking SMN1 gene' with a working copy of a human SMN gene.
It is done so through the use of a carrier called a 'vector'.
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Put simply, the carrier for this treatment is a virus that has had its DNA removed.
The DNA is then replaced with the correct working gene.
Patients don't get sick from the virus, and instead it travels through the body to deliver the new gene to where it's needed. It then tells the body to make the new working protein instead.
The vectors are then broken down and shed from the body.
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The downside is, it costs an eyewatering $2.1 million for a single treatment. Families of patients are infuriated by how much it costs, and are calling for the price to be lowered.
In the UK in 2021, a deal was struck meaning they could access the drug at a cheaper price, so 80 babies a year could benefit from it.
The staggering price of the drug, which is sold by Novartis, was supported by independent body Institute for Clinical and Economic Review (ICER), which suggests fair prices for medication.
Dr. Steven D. Pearson, then-president of ICER, said of Zolgensma's $2.1 million price tag when it was announced back in 2019: "Zolgensma is dramatically transforming the lives of families affected by this devastating disease, and given the new efficacy data for the pre-symptomatic population, the price announced today falls within the upper bound of ICER’s value-based price benchmark range."
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Something else that has ruffled feathers, is the early stage development of Zolgensma was funded and financed by the National Institutes of Health and several charities.
Many charities based in the US such as Sophia’s Cure, Cure SMA, Getty Owl Foundation, Fighting SMA, Jadon’s Hope Foundation, the Gwendolyn Strong Foundation, and Miracle for Madison used donations to invest in clinical trials and to help research.
A previous medication used for treating SMA, Spinraza, is taken four times a year for life.
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Spinraza is priced at $750,000 for the first year, with each following year costing $350,000 per year.
A decade of treatment would set you back something close to $4 million.
Horrifyingly, when it comes to pricing a drug, many companies calculate how many years of life the patient gains.
Novartis purchased Zolgensma when they acquired the firm AveXis for a whopping $8.7 billion.
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What's worse, most health insurance schemes won't insure one-time genetic treatments such as this due to the price tag.
Novartis offers payments in instalments, at $425,000 a year.
They have also said they will give partial refunds if the treatment doesn't work.
UNILAD has contacted Novartis for a comment.
Jen Thomas