The eyes have it

What is “it,” you ask? Spark Therapeutics’ new gene therapy Luxturna which can cure blindness in a single treatment. The condition causing the blindness only affects a few thousand people, so the FDA has designated it as an orphan drug. The single dose and orphan drug aspects of this gene therapy combine for a rather expensive R&D bill, and translate into a bit of sticker shock at the selling price: $425,000 per eye. Spark is trying out some interesting commercialization practices to get the drug to patients, like giving rebates to patients whose eyes don’t see better over time. They’re also considering selling the therapy directly to insurance companies so that health care providers don’t have to pay and store the treatment without a guarantee it’ll ever be used.

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