The prices of medications are continuously rising in the United States and around the world. According to the CMS website, the healthcare spending in the United States for the year 2019 reached $3.8 trillion, which is approximately $11,500 per person per year.
With recent advances in technology, a new type of medication was made, Zolgensma, which functions through gene therapy used for the treatment of spinal muscular atrophy. The cost of a 1 time treatment is $2.125 million.
Spinal Muscular Atrophy is a rare genetic motor neuron disease that leads to muscle weakness, causing difficulty in holding up the head, breathing, swallowing etc. Most patients do not survive past the age of 2 years due to respiratory failure.
Zolgensma works by using a vector to replace the missing/defective gene in the body. It does not reverse the damage already done but can prevent progression of the disease which is why this needs to be administered early in the course of the disease. It significantly improves the quality of life of the patient.
The reason the medication is so expensive is due to the fact that the market for the drug is very limited since it treats a very rare genetic disorder and has a potential to save lives. Its a highly specialized drug that required a lot of research around it.