Senators Ask GAO to Probe Orphan Drug Act as Questions Pile up Over Marathon’s Pricey Steroid

As US Duchenne Muscular Dystrophy (DMD) patients may be hit with a 50 to 70 times more expensive price for a steroid they have been importing from the EU and Canada for about $1,000 per year, eight Democratic senators sent a letter on Friday to Marathon Pharmaceuticals questioning the orphan drug Emflaza’s (deflazacort) $89,000 list price, while three Republican senators on Friday also asked the US Government Accountability Office (GAO) to look into orphan drugs in general.

The letter sent by Sens. Orrin Hatch (R-UT), Chuck Grassley (R-IA) and Tom Cotton (R-AR) to the comptroller general of the GAO seeks a more in-depth look into potential abuses of the Orphan Drug Act:

"To inform Congress' work on this important issue, please provide a list of each drug for the past year for which there was a request for a designation of orphan status and which were approved and which were denied. Of those drugs, please list any indications for the drug, other than the orphan designation. Additionally, please include a list of all orphan-approved drugs since enactment of ODA and whether resources at the FDA have kept up with the number of requests received at the Office of Orphan Products Development (OOPD). Furthermore, we feel that it is important to include the patient voice in your review, and to the extent possible, please include patient experiences in your review.

"Additionally, we request the Government Accountability Office to investigate whether the ODA is still incentivizing product development for diseases with fewer than 200,000 affected individuals, as intended, and provide any regulatory or legislative changes that may be needed in order to preserve the intent of this vital law," the letter says.

The Republicans’ letter comes as Grassley has said he is looking into abuses of the act that allow such incentives, meant to encourage drug development for rare diseases, to be bestowed on a drug that actually reaches a larger population than what it won the orphan designation for, or companies that conduct limited research to win approval for such a drug.

Marathon

The Marathon letter, meanwhile, following a similar letter sent in mid-February from Sen. Bernie Sanders (D-VT) and Rep. Elijah Cummings (D-MD), calls on Marathon to provide more information on how much it paid to acquire clinical trial data necessary to win approval of Emflaza, expected earnings during the orphan drug’s 7-year exclusivity period, and explanations on the discrepancy between Marathon’s estimate that 7-9% of those with DMD use deflazacort and CDC’s estimate of 22%.

In addition to the high price tag for Emflaza, the company was also awarded a priority review voucher as the treatment is for a rare pediatric disease, which, if Marathon chooses to sell it, will likely bring in more than $100 million (the last voucher sold for $125 million in February).

Following the outrage after the approval of Emflaza, industry group PhRMA, which brought Marathon in as a member, said it is reviewing and may revise its list of member companies.

The letter was signed by Sens. Patty Murray (D-WA), Tammy Baldwin (D-WI), Cory Booker (D-NJ), Al Franken (D-MN), Margaret Hassan (D-NH), Angus King (I-ME), Elizabeth Warren (D-MA) and Sheldon Whitehouse (D-RI).