‘Tis the season for gene therapy approvals. Reported by Bloomberg, the U.K. just approved its most expensive medication to date. The treatment is for severe combine immune deficiency, or more popularly known as the “bubble-boy disease”. The price is currently listed at 594,000 euros, which equates to about $716,000.
The treatment is said to be a one-time pop and would leave newborns potentially cured from the disease. Severe combine immune deficiency, also known as SCID, is a disease that weakens the immune system so immensely, making every day germs a threat. To learn more about SCID, click here.
Britain’s biggest company for drug development is Glaxo and they developed this treatment, along with Strimvelis, which was a gene therapy approved back in May of 2016. Since the approval date, there have been four successfully treated patients. Is that enough? Do the benefits outweigh the costs? Fortunately for Glaxo, the drug was recommended by the respected National Institute for Health and Care Excellence, which may help the treatment’s lifespan. While gene therapies are on the rise, and Glaxo has dealt with success, the financial factor still haunts them.
It seems that people are believing in the treatment, as Glaxo stock continues to rise. Two more patients are on the dock to be treated with Strimvelis. Glaxo, in London, will most likely continue to be the main source of Strimvelis treatment as it is not available in the U.S. Prior to gene therapy, stem-cell transplants were the go-to treatment known to rejuvenate the immune system (and still remains a current treatment as gene therapy progresses), yet it wasn’t always successful. U.K. drug-price monitors shared that the patients didn’t always have a positive response to the transplants and finding matching donors was always difficult. Sometimes a match wasn’t found at all.
Finances and benefits continue to circulate the U.K. medical industry, as well as in the United States. High prices seem to be unjustifiable, yet at the same time, no one can put a price on someone’s life. We can only hope gene therapy continues to be of success and pricing is regulated to benefit all.