Viewpoint: Will the public be able to afford one-time gene therapy for hemophilia just approved by the FDA?

On April 26, Pfizer issued a press release announcing that:

U.S. FDA Approves Pfizer’s BEQVEZ™ …, a One-Time Gene Therapy for Adults with Hemophilia B

This sounds like good news. There is a lot more in the announcement, which runs over 2000 words, but one item is conspicuous by its absence: the price.

What the treatment costs is neither a secret nor a surprise… Hemgenix was approved by the FDA in November 2022, and caused a stir since it was priced at $3.5 million for a one-time treatment, the highest price ever at the time.

Pfizer argues that at $3.5 million,  Beqvez is a “compelling value proposition” compared with previous treatments, which can involve intravenous transfusions several times a month. Pfizer says those transfusions can cost from $600,000 to $1.1 million a year, potentially more than $20 million over a patient’s lifetime. They may be over-optimistic: So far, Hemgenix is not as successful in the marketplace as once hoped, much to the frustration of some advocates.

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The science may be advancing rapidly but as Crispr therapies start heading to the clinic the social, political and economic context, specifically the funding of research and the pricing of products, seems to be failing to keep up.

This is an excerpt. Read the full article here