Gene Therapy, Pricing

You want to charge $1M for that gene therapy? Here’s where you’re wrong

Spark Therapeutics $ONCE doesn’t have a green light yet from the FDA to sell its gene therapy for one small group of patients with a genetic flaw that causes inherited blindness. And company execs haven’t told anyone exactly what its wholesale price will be for voretigene neparvovec.

But the odds look great for this treatment to quickly nab the first approval for a vector-delivered gene therapy in the US — and it’s game on for a pricing debate that starts with an auction forecast of $1 million per treatment.

ICER (the Institute for Clinical and Economic Review) has been crunching the numbers on quality adjusted life years and the impact of RPE65-mediated retinal dystrophy, and concluded in a draft report (subject to change) that $1 million — which would mark a new high water mark for the industry — is way too high.

Notes ICER:

(A)t a placeholder price of $1,000,000, the high cost makes this unlikely to be a cost-effective intervention at commonly used cost-effectiveness thresholds.

There are some caveats, including the rising cost effectiveness you’ll see the younger you go with the patient age, with the potential for improved sight over a long lifetime. But…

(I)n all base case scenarios, VN would require large discounts to reach commonly used thresholds of cost-effectiveness.

Steven Pearson, ICER

You can see the full review here for a clear idea about the formulations from ICER, which has been gaining steady influence in a country where there is no single payer system to leverage lower prices. Interesting to note, Spark gets docked for earlier gene therapy experiments that faded over time, underscoring the debate coming about therapies that almost — though not quite — promise to work a lifetime. So proving that you can do it better, longer, will be key to future successes.

Spark CEO Jeff Marrazzo has done his own math, looking at the value of being able to have a job, releasing a caregiver to some other task, and the court judgments centered on loss of sight. For him, there are plenty of reasons to justify more than $1 million.

There’s more at stake here than the fate of one drug or one biotech and its investors. Spark is in a position to set the performance bar for everyone that’s been jumping into the once and done club. And it will say a lot about which gene therapies are developed — going where the money is — and how much they all cost.

This debate is just beginning.

Image: Shutterstock

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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