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.
There’s some debate as to whether cancer patients should have drugs that are proven to work, or whether instead they should have access to as many drugs as possible that might work. At least that’s what we’ve gathered from a recent study identifying that more than half of cancer drugs approved by the EMA between 2009 and 2013 showed no benefits for either survival or quality of life. While that’s not extremely terrible considering, 1) the EMA focuses on timely patient access to new drugs and, 2) trials focusing on survival outcomes are expensive and (more importantly) time-consuming, one has to wonder if we can’t do better than half. Otherwise we’re just provoking cancer patients to the point of rampage.