ICER developed a report assessing the comparative clinical effectiveness and value of voretigene neparvovec (Luxturna®, Spark Therapeutics), a potentially one-time gene therapy for vision loss associated with biallelic RPE65-mediated retinal disease. Voretigene neparvovec is the first gene therapy to be approved by the US Food and Drug Administration (FDA).

The appraisal committee vote revealed differences in judgments on value given pricing far above traditional cost-effectiveness levels. Policy recommendations call attention to the growing stream of treatments for rare disorders that cannot all be priced at similar levels without threatening the financial sustainability of the health system.

Date of review: January 2018

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Final Documents

Below you will find the final documents from the assessment review process:

“Voretigene neparvovec has a very high cost, and the long-term durability of its effect is uncertain. However, this is an effective treatment that gives people with a serious, ultra-rare condition a treatment option where they previously had none. Payers and other policymakers must strike a difficult balance in judging the value of this therapy, recognizing the treatment’s broader benefits to affected individuals and society while simultaneously working to maintain health insurance affordability. As an increasing number of therapies for ultra-rare conditions enter the market, stakeholders must collaborate to develop new approaches to pricing and payment that can reward innovation while preserving access to high-value care for all patients.”