FDA approves sickle cell treatment by gene-editing patient's own blood stem cells

Treatment is a several month process, including the extraction of the patient’s bone marrow, gene-editing them, using chemotherapy to destroy the patient’s remaining bone marrow, injecting the modified stem cells, and waiting for them to grow back and make red blood cells that without sickle cell.

It costs $2.2 million per patient.

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In spite of the length of the treatment and the cost (which, of course, is totally arbitrary), this is a huge, huge advance. Being able to tinker with your own genes and then put them back so your body works correctly is absolutely amazing. I can’t wait to see what else they come up with.

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I would bet that the costs involved with the current treatment (over time) required for a person with sickle cell is extremely expensive … but because we don’t think that way with our healthcare system, that won’t be a consideration. It will be very interesting to see what happens with this treatment (which, for the record, is incredible).

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The Cost of Living with Sickle Cell Disease - Hematology.org says that lifetime costs for sickle cell treatment are around $1.7 million (including spending by insurance, employer, and/or government).

This does not include indirect costs like limitations on work and other activity.

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There are other things that patients considering this newly-approved treatment need to consider:

Not to mention pain. Sickle cell disease is excruciatingly painful and makes it difficult for patients to attend school, work, etc.

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This is going to alleviate so much suffering. It’s beautiful to contemplate.

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I’m not sure if people noticed in the OP article, but a second drug for sickle cell was also approved yesterday. It is not CRISPR, but is a gene therapy. Lyfgenia’s list price is $3.1 million.

The costs of these products are not arbitrary at all. Extensive market research is done prior to launch to understand the marketplace dynamics and what the market will bear, similar to product pricing in other industries. Cost of development, manufacturing, delivery, etc are also taken into account when setting prices.

Having two products launch at the same time will likely result in more contracting discounts between the payers and manufacturers as well. Although I’m not sure how serious the market perceives Lyfgenia’s boxed warning. That could well impact relative pricing and contracting going forward.

What does the boxed warning say?

Via google…

Boxed WARNING: HEMATOLOGIC MALIGNANCY

Hematologic malignancy has occurred in patients treated with LYFGENIA. Monitor patients closely for evidence of malignancy through complete blood counts at least every 6 months and through integration site analysis at Months 6, 12, and as warranted.

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An explanation of what the formerly know as Black Box Warning means:

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