Two gene therapies approved by the FDA last week for the treatment of sickle cell disease will have multimillion dollar price tags for patients (and insurers), according to a news brief posted by Reuters.1
The news agency reports that Casgevy, the CRISPR/Cas9-based gene therapy from Vertex Pharmaceuticals, will be priced at $2.1 million; meanwhile, Lyfgenia, from Bluebird bio, will have a list price of $3.1 million.1
Health experts worry that the therapies will not get to the parts of the globe where they are needed most, however.
According to the New York Times, the therapies will initially be rolled out in countries like the US, UK, some European Union nations, and Saudi Arabia, which have an estimated 2% of the world’s sickle cell patients.2 The Times reports that the therapies are unlikely to reach sub-Saharan Africa, however, which has an estimated 75% of the world’s sickle cell patients.2
The reason for this is twofold, according to the Times:
First, price: The treatments are far too expensive for governments that struggle to pay for basic health services. In some cases, there may be substantial additional costs, such as for a patient’s extended hospital stay to receive gene therapy.
The second barrier is medical infrastructure: Administering the treatment is a monthslong process at medical centers that can perform stem cell transplants. Patients must have their cells harvested and flown to a lab for manufacturing, undergo grueling chemotherapy and endure a long hospitalization.
The New York Times. Read more at: https://www.nytimes.com/2023/12/08/health/casgevy-lyfgenia-sickle-cell-africa.html
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