Best Healthcare System in the World

The FDA has approved the first genetic therapy for Sickle Cell disease, and it only costs $2,200,000.00 per person treated.

Given that there are 100,000 Americans with Sickle Cell, that ain't chump change.

I would also argue that at that level of remuneration, we can be pretty sure that good folks at Vertex Pharmaceuticals are NOT going to share any issues regarding effectiveness or side effects with regulators.

 Same goes for the treatment from Bluebird Bio, which costs an even more ridiculous $3.1 million.

That price is obscene:

The FDA approved the first gene therapy treatments for sickle cell disease on Friday, but it will cost patients millions of dollars.

One of the approved therapies, developed by Vertex Pharmaceuticals and called Casgevy, is the first of its kind to use the CRISPR gene-editing tool, according to the FDA.

………

Vertex and CRISPR Therapeutics said in a statement that about 16,000 sickle cell patients who are over the age of 12 may be eligible for the therapy, which offers a "potential of a functional cure for their disease."

That therapy, however, could cost a single patient more than $2.2 million, not including the cost of associated care, such as a hospital stay or chemotherapy, according to an SEC filing.

………

The second treatment the FDA approved on Friday is called Lyfgenia. Lyfgenia modifies a patient's blood stem cells and transplants them, but it instead adds normal hemoglobin that is uninfected with the disease to the cells so that they have a lower risk of sickling, according to the FDA.

Lyfgenia will come with an even higher price tag of $3.1 million, Bluebird Bio, the biotech company that developed the treatment, said in a news release.

These are all dependent on the CRISPR gene editing tool, which was developed with federal funding, and the isolation of the mechanism and the genes behind Sickle Cell disease were discovered with federal funding, so maybe it's time to get serious about executing march-in rights under the Bayh-Dole Act, which would allow the US government to license these technologies to other (far less larcenous) entities.

It should be noted that march-in rights have never been used in the 43 years that this law has been on the books.

It's about time to start.