top of page
  • Writer's picturebxgenetics

Keeping up with the Cost

Updated: Jun 14

By Sumi Lama

Gene therapies aim to cure genetic diseases by managing the root of the problem and altering genomes. In the past few years, many companies have been releasing gene therapies for rare and lifelong diseases. The one catch is the price tag, which averages around $1-2 million. The record for highest price is continuously being broken with each new treatment. As of now the most expensive is $3.5 million for Hemgenix. How do people manage to pay for these treatments?

Gene therapies treat rare diseases and may not be administered often. Companies have to perform hundreds of clinical trials before the treatment can be approved, which adds to the overall cost. Since production and manufacturing costs are so high, companies try to earn a profit by increasing the price of their treatment. Some treatments are even tailored to the individual, making it more expensive. Gene therapies for rare diseases may only be used by hundreds of people, In contrast, most medical drugs are used by thousands of people. After being approved by the FDA, drug treatments can cost companies $5 billion. Additionally, many gene therapies are one-time treatments. This means that one dose worth a million can replace the need for frequent, costly treatments. The cost is worth it for many people. The price takes into account patient benefit, manufacturing costs, demand, and more.

Zolgensma treats a rare disease, spinal muscular atrophy (SMA), and is priced at $2.1 million. Families in Brazil went to court over the treatment and the government was forced to pay for the most severe cases of SMA. However, it can cost the government $1 million per patient and puts a strain on Brazil’s underfunded healthcare system. The government pays installments over 4 years, but can stop after 2 years if the patient is still ill. The price of the treatment is decided after negotiations between companies and governments. The government makes an upfront payment that allows the cost of treatment for individuals to be lowered. If companies are unsatisfied with the price, they can simply choose not to supply the country. Depending on the treatment, some companies propose alternative methods of payment (installments) or reduce prices. Zolgensma can only be administered for children two years and younger in the U.S. This is about 700 children and not all of them may have access to Zolgensma. High costs and a small demand result in a high price tag that is difficult to lower.

These prices raise concern over access and innovation. Even after a cure is found, the government may not be able to provide it to the people who need it the most. With high-income countries struggling, how will middle and lower-income countries fare? The future of gene therapy seems uncertain as prices soar.


Castronuovo, C. (2023, January 23). World’s most expensive drug revives push for new payment models. Bloomberg Law.

Robbins, R., Nolen, S., & Galdieri, D. (2023, January 24). A dilemma for governments: How to pay for million-dollar therapies. The New York Times.

Sterling, J. (2023, June 5). Cell and gene therapy manufacturing costs limiting access. GEN.,and%20%242%20million%20per%20dose.

Paying for CRISPR cures: The economics of genetic therapies. Innovative Genomics Institute (IGI). (2022, May 18).

1 view0 comments

Recent Posts

See All
bottom of page