Monday, May 6, 2024

Heady Times: First Patient Receives Vertex's ~$3 Million Sickle-Cell "Cure" -- Six Months After FDA Approval.


No doubt this is a piece of very good bio-science news: the cure for sickle-cell anemia -- a painful and often debilitating disease is now being administered to select patients, in the US. That's a huge win. [Here is one of several backgrounders we posted, over the years -- on it.]

But the patients are "select" precisely because the therapy is priced somewhere between $1.8 million and $3.1 million for a full course of treatment, depending on whom one believes. Do read all the excellent NYT piece has to offer on the topic, this morning:

. . .For the estimated 20,000 people with sickle cell in the United States who qualify for the treatment, the start of Kendric’s monthslong medical journey may offer hope. But it also signals the difficulties patients face as they seek a pair of new sickle cell treatments.

For a lucky few, like Kendric, the treatment could make possible lives they have longed for. A solemn and shy adolescent, he had learned that ordinary activities — riding a bike, going outside on a cold day, playing soccer — could bring on episodes of searing pain.

“Sickle cell always steals my dreams and interrupts all the things I want to do,” he said. Now he feels as if he has a chance for a normal life. . . .


As I say, do go read it all. It is especially gratifying to finally see life-science efforts aimed (at a very robust scientific level) at a disease that disproportionately afflicts a population other than whyte european descended humans.

That said, I am unconvinced that the treatment should be priced by reference to what a patient without it will incur / cost, over a life-time, in hospital stays and other out of pockets. Perhaps a better metric for the already wildly profitable Vertex (one of the makers of the cure) would be. . . what it would cost to recover the R&D burden Vertex shouldered over say a 15 year life span for the remaining patents -- if X number of patients were granted access to the drug, per year. [If even only about half of the 20,000 potential patients receive it, at $3 million per course of treatment, the companies will have generated added revenue of. . . $30 BILLION! There is a nearly-zero chance that the R&D cost more than $2 billion. So a 15X return on investment seems. . . a little too high.] Just a thought. Onward.

नमस्ते

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