While the EpiPen saw its price go from around $100 for two pens to over $600 over the course of nine years, the margin of difference for deflazacort, which is prescribed to patients with the fatal disease Duchenne muscular dystrophy, is far greater. Previously, families were able to offer afflicted members relief with the drug for around $1,200 a year. Now, the price will be 74 times that amount. Actually, if you count rebates and discounts, you’ll be able to get a deal for $54,000.
Before Marathon Pharmaceuticals got the drug FDA approved late last week, patients in the U.S. simply imported the drug — which has been around for years — from overseas. But now, Marathon Pharmaceuticals has gotten approval for the drug under “orphan” status, which means that the company has a seven-year exclusive on selling the medicine, alongside a voucher to accelerate future approvals.
“It seems like it’s yet another example of gaming the system,” Aaron Kesselheim, an associate professor of medicine at Harvard Medical School, told the Washington Post. “How many examples of this do we have to see before we can start to rethink the priority review voucher as a means of incentivizing innovation? This also seems to be another example of gaming the Orphan Drug Act, which was intended to try and encourage research into new therapeutic entities for people who have rare diseases — and it doesn’t seem like this is that.”
When it comes down to it, the actual out-of-pocket expenses for patients who need the drug (which will now be known as Emflaza), will be “zero to low.” And indeed, getting the drug FDA approved could make access to it easier (and will allow for insurance to cover for it). In a statement, patient advocacy group Parent Project Muscular Dystrophy said it was excited about the approval, but also added, “There are many questions still to be answered, including pricing, and we look forward to talking to Marathon with the community to begin to answer those questions.”
