By Rachel Sachs
Yesterday, the FDA approved a steroid, deflazacort, for the treatment of Duchenne Muscular Dystrophy (DMD). DMD is a rare, heartbreaking, and ultimately fatal genetic disease with few if any real treatments, and the steroid may be helpful to patients. Deflazacort’s sponsor, Marathon, has offered the drug at a list price of $89,000 per year. High, but actually much lower than the typical prices charged for new orphan drugs, which can easily run to $300,000 or more per year.
Here’s the big problem: deflazacort isn’t really a new drug. As the Wall Street Journal and Endpoints have pointed out, the drug is approved in many other countries, and its list price is about $1,000-$1,600 in Canada and the UK. Patients have been importing the drug and accessing it since the 1990s. Now, patients will pay many times those prices for the same product they had already been purchasing.
But the drug had not previously been approved in the United States, and surely Marathon conducted new clinical trials to demonstrate the drug’s benefit? Not clearly. Marathon mostly relied on clinical trial data from the 1990s that had not been fully analyzed. In return, Marathon gets 1) a seven-year market exclusivity period for the drug (as required by the Orphan Drug Act) and 2) a valuable priority review voucher (as required by law for rare pediatric diseases).
This is not acceptable. Full stop. It is the worst sort of gaming that other companies have engaged in over the years. And at a time when the drug industry is under fire for its high prices, PhRMA cannot afford to have its members (of which Marathon is one) acting this way. If PhRMA and patient groups funded by pharmaceutical companies are serious about drug pricing, here are three things they should do/encourage right now: