30 April 2018 - In December 2017, the US FDA approved voretigene neparvovec-rzyl (Luxturna), a one-time genetic treatment manufactured by Spark Therapeutics that must be injected into the eye and can improve the vision of individuals with biallelic RPE65 mutation–associated retinal dystrophy, a rare genetic condition that affects approximately 1000 to 2000 people in the US each year. 

The therapy was priced $850 000 per treatment, just below the million dollar threshold some had anticipated.

Perhaps because the price of the drug amounts to the annual commercial insurance premium for 32 families of 4, Spark promised to reduce the cost through pricing manoeuvres that the pharmaceutical industry has referred to as value based. These approaches include extended financing options, allowing payers to purchase voretigene neparvovec-rzyl directly, and offering rebates when patients do not have vision improvement following therapy.1 These efforts will at best produce minimal savings. 

Even rebates when a drug does not work will be both rare and small (ie, <20%), so they do not trigger mandatory discounts in Medicaid. Meanwhile, an independent analysis by the Institute for Clinical and Economic Review (ICER) reported that voretigene neparvovec-rzyl should cost at most $426 644 based on the benefit it provides, an estimate ICER termed a value-based price.

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