On his 14th birthday, RJ Sullivan got a gift a true Lego lover, like himself, can appreciate, “It’s the White House.” But it was another gift out of Washington D.C. that brought great relief to his family.
The Food and Drug Administration approved the first steroid, this month, used to treat Duchenne Muscular Dystrophy.
"The letter stated, it was very exciting news, that this drug was going to be produced by Marathon here in the U.S.,” said RJ’s mom Suzanne Sullivan.
RJ was diagnosed with the fatal muscle-wasting disease eight years ago. "The best option, and it is a daily option, is to take a steroid," said Suzanne Sullivan.
Pending FDA approval, the Sullivan’s have been buying Deflazacort from the U.K. "We pay anywhere from $700 to $1400 a year" said Suzanne.
So imagine their surprise when Marathon announced their version Emflaza would come with an $89,000 price tag. "This is the standard of care, the only treatment out there for boys with Duchenne... It doesn't seem right that they can do that."
But they can, because of the 1983 Orphan Drug Act signed into law by President Ronald Reagan. The act incentivizes companies to develop drugs for rare diseases by allowed them to have seven year monopolies, meaning no other company can sell that drug during that time.
"To save your son you have to live in a shoe box," said Jennifer Dumm
Right now Dumm’s son, Owen is on a trial for the only other FDA approved specialty drug for Duchenne. She says in 96 weeks, the change is noticeable. "He was not only able to scratch his knee or his ankle but sit back up again, where before he couldn't, " said Dumm.
That progress is the reason she says she'll give up everything to keep him on this lifesaving drug.
"The baseline of this drug is $325,000 a year… Right now there is a lot of controversy with the insurance companies not wanting to cover the cost of this drug."
So parents are left fighting to obtain two drugs that are the only hope for the estimated 15,000 living with this rare, incurable disease.