At the beginning of February, the FDA announced that a new drug (not really new just not technically available to patients in the US) had been approved. For patients with Duchenne muscular dystrophy- a rare and fatal disease that only around 12,000 people in the US have- a corticosteroid called Deflazacort had been shown to be useful and life-prolonging.
While bringing the drug here and finally making it legal is great, the price hike is not. The drug, which will be marketed under the name Emflaza, will cost $89,000 for a year’s worth of pills. If that seems like a lot, it is. But don’t worry, the CEO of Marathon Pharmaceuticals, the company marketing the drug, said that after discounts and insurance rebates the cost per patient would be more like $54,000 per year. The bigger issue is that currently, for families who can import it from countries where it’s available, the price ranges from $800-1600 a month- that’s a savings of over $34,000. For patients with insurance, the cost will be almost nothing but insurers are going to have to shell out much more than necessary. Will they later pass that along to customers in the form of premium hikes? The system is broken.
From the article:
“Marathon Pharmaceuticals didn’t invent or even perform its own clinical trials on deflazacort, but bought the rights to data from clinical trials performed on Duchenne muscular dystrophy patients in the 1990s, using that to submit an application to the FDA. The clinical trial data showed that the drug helped patients keep more muscle strength, and patients taking it retained their ability to walk for longer.”
Now, the reason they were able to do this is because of a law called the Orphan Drug Act which “gives pharmaceutical companies exclusive patent rights for a few years after developing a new drug or gaining approval for a different use for an existing one.” When the program is used as it’s intended it’s great, but that’s not really the case here.
So, Sen. Bernie Sanders and Rep. Elijah Cummings sent Marathon a letter asking Marathon for “detailed information” about the drug’s development and for info on how much it really cost to bring the drug to market. And that seems to have helped because days later Marathon announced they were delaying the launch to discuss the price with caregivers and other stakeholders.