Gene Therapy in a Capitalist Economy

Biopolitical Times
Scientist examining DNA

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. Health Canada approved Beqvez (no one but Pfizer seems to use the all-caps version) in January, having previously approved Hemgenix, a different hemophilia B treatment produced by pharmaceutical company CSL Behring. 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. So is Beqvez, as Fierce Pharma noticed:

Pfizer had the chance to undercut its rival on price but decided to charge the same $3.5 million for Beqvez. 

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.

Beqvez and Hemgenix are not now the most expensive treatments on the market. That dishonor currently goes to Orchard Therapeutics’ Lenmeldy™ for a terrible condition that affects less than 40 babies a year in the US. The company says:

Lenmeldy wholesale acquisition cost of $4.25 million for one-time treatment reflects its clinical, economic and societal value

That claim is questionable. The Institute for Clinical and Economic Review (ICER), a health-economics think tank in Boston, analyzed the treatment last year. They concluded that it might be cost-effective at a price “between $2.3m and $3.9m.” That range is of course high and wide, but the company chose a price point $350,000 to almost $2m above it.

In 2023 there were several initiatives around gene therapy. In March, the Third International Summit on Human Genome Editing focused largely on gene therapy. In April, Nature published an editorial titled “The gene-therapy revolution risks stalling if we don’t talk about drug pricing.” In July, Jennifer Doudna’s Innovative Genomic Institute published a 75-page report on “Making Genetic Therapies Affordable and Accessible” and in January Doudna wrote in Wired:

Gene Editing Needs to Be for Everyone
Crispr recently marked a major milestone in medicine. But it's not time for a victory lap—the race is just beginning. 

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.