Sarepta Prices Its Gene Therapy at $3.2M ... And Brings Receipts

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Yesterday, Sarepta won FDA approval for its Elevydis gene therapy for Duchenne muscular dystrophy. The price tag: $3.2 million. (Dive gets into the details of the price announcement here, and I’ll write more later about my admiration for how Sarepta handled the pricing comms.)

The hard-to-answer question around gene therapies is always: is that a lot?

I mean, whenever something’s price has a “million” in it, that’s a big number. But the pitch for gene therapy has been that these are 1) one-time therapies, 2) with dramatic health impacts that also 3) take costs out of the system, so sticker shock isn’t necessarily warranted.

In the case of Sarepta, the company brought receipts. Unbeknownst to me, and, apparently, everyone else, the company published a peer-reviewed cost-effectiveness study that suggested the medicine would be a deal even at $5 million and could -- depending on your assumptions -- justify a $13 million price tag. (I couldn’t find any coverage in the media or on social platforms, following the publication last month.)

Here is the key table from the paper, which shows there are some pretty big swings in “maximum treatment cost” depending on what you think of willingness-to-pay thresholds (“WTP” in the chart) and discount rates. For reference, the Institute for Clinical and Economic Review uses a WTP threshold of $150,000, even for rare diseases, and a 3% discount rate.

I’ll let you all come to your own conclusions.

• An SSR Health exec posted first-quarter data on drug prices on LinkedIn yesterday, noting that NET PRICES ARE FALLING. I hate to go all-caps on y’all, but sometimes it seems like that message isn’t sinking in.

• As I noted yesterday in unveiling my handy chart on the state of IRA lawsuits, the legal actions running on the everything-everywhere-all-at-once theory. STAT has a bit more about the strategery of where the suits are being filed.

• I haven’t written much on the issue of drug shortages, in part because I have little to add, and the solutions seem both easy to describe and hard to implement. The Association for Affordable Medicines has a new white paper out that underscores that easy/hard dynamic.

• Endpoints goes long on the FTC and why they’re content to keep taking Ls in their ever-more-aggressive approach to biopharma antitrust theory.

• This Health Affairs Forefront piece suggests a number of approaches that the government can take to set prices under the IRA. The mathematical diligence is admirable, but I’m a little taken back that the team here -- from the University of Washington -- would even endorse (with caveats) a cost-plus approach to price-setting.

• CMS is out with more on how the new registry for Alzheimer’s meds will work. But advocates say there still aren’t enough details. What kind of details are needed? This just-dropped Health Affairs piece lays out all of the elements to be considered.

• There isn’t a lot of marketing out there around patient assistance programs, so I’ll be watching closely this Lilly effort -- complete with celebrity spokesperson -- to promote its $35 insulin out-of-pocket price cap. Also: Go Sox.

• Bernie Sanders needs an editor.