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June 5, 2018

Value-Based Pricing for Drugs: Theme and Variations

Author Affiliations
  • 1Center for Health Policy and Outcomes, Memorial Sloan Kettering Cancer Center, New York, New York
  • 2Center for Health Policy and Outcomes, Memorial Sloan Kettering Cancer Center, New York, New York
JAMA. 2018;319(21):2165-2166. doi:10.1001/jama.2018.4871

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.

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