In December 2017, the US Food and Drug Administration 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 United States each year. The therapy was priced $850 000 per treatment, just below the million dollar threshold some had anticipated.
Kaltenboeck A, Bach PB. Value-Based Pricing for Drugs: Theme and Variations. JAMA. 2018;319(21):2165–2166. doi:10.1001/jama.2018.4871
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