The FDA approved a new drug to treat hemophilia B on November 9, 2022, the latest gene therapy to be approved in the United States. Currently, the price tag that the pharmaceutical company put on the drug is $3.5 million per dose, which makes it the single most expensive drug on the market to date.
Hemophilia B is a disease that is classified by the lack of factor IX in the blood. This is an important enzyme that is part of the intrinsic coagulation cascade. Having a nonfunctional version of this protein leads to bleeding episodes and an inability for the blood to clot. The bleeding caused by it can move into internal organs, including the brain, making it a deadly disease. The current treatments include protein replacement therapy injections several times a week, which can become very expensive, but they are necessary for someone living with hemophilia B to lead a semi-normal life. The introduction of this new drug allows people with this disease to lead a more normal life, as it is a one-time injection. It works by using a non-infectious adenovirus that delivers DNA to liver cells that code for factor IX. This allows the body to produce a correct version of the protein without the need for weekly injections, meaning that patients are no longer constrained to living in the immediate vicinity of their healthcare provider.
While this is a very exciting treatment for a debilitating disease, the only problem with it is its price. As stated earlier, it is the most expensive drug ever to be put on the market. This should make us ask the question: who is responsible for bearing the cost? While there are government programs and insurance to help pay for therapies such as this, the patient still likely has to pay an exorbitant amount of money that many people do not have. Justice is a large component in this discussion, as it describes the current situation. A very small population needs to pay millions of dollars just to have a normal life while the rest of us get to live for free (comparatively). People may be crushed with medical debt just to receive life-saving treatment, so is this price tag really worth the quality of life of others?
Center for Biologics Evaluation and Research. (n.d.). Hemgenix. U.S. Food and Drug Administration. Retrieved November 28, 2022, from https://www.fda.gov/vaccines-blood-biologics/vaccines/hemgenix
Upham, B., Heid, M., Dunleavy, B. P., Marks, J. L., Haelle, T., & Upham, B. (2022, November 23). FDA approves Hemgenix, first gene therapy to treat adults with hemophilia B. EverydayHealth.com. Retrieved November 28, 2022, from https://www.everydayhealth.com/hemophilia/fda-approves-hemgenix-first-gene-therapy-to-treat-adults-with-hemophilia-b/#:~:text=How%20it%20works%3A%20Hemgenix%20uses%20AAV5%2C%20a%20noninfectious,become%20a%20part%20of%20a%20person%E2%80%99s%20own%20DNA.
This is definitely interesting, $3.5 million per dose? WOW! This new FDA Approved Drug for Hemophilia B is a big step forward, but the cost is way too much. What about the underserved communities with Hemophilia B? This definitely breaks the non-malfeasance and justice rule because the low-income communities will be facing unfair treatment as well as in a way be harmed because, without the treatment, their condition only worsens.
ReplyDelete1 in 40,000 people has Hemophilia B of which most of them are men, so that is a lot of money to be made from that. CNN reported that the approval of this treatment signifies a progressive innovative therapies development with individuals experiencing a higher burden of any disease associated with this form of hemophilia (McPhillips, CNN, 2022). However, this makes me question the same thing as you Bryan, who pays for it? Will insurance even cover it? Because for the low-income communities, I still do not see how the quality of life is going to improve if they cannot afford the treatment and insurance isn’t covering it.
CITATION:
McPhillips, D. (2022, November 23). FDA approves $3.5 million treatment for hemophilia, now the most expensive drug in the world. CNN. Retrieved November 28, 2022, from https://www.cnn.com/2022/11/23/health/hemophilia-drug-hemgenix