Cross-country trip calls attention to huge price spike for muscular dystrophy drug

This isn’t the type of summer vacation most families would want to embark on. It’s not a week on the West Coast or multiple days at Walt Disney World.

For Scott Griffin, an Alabama father, along with twins Gabe and Addie and family friend Michael Staley, it is, instead, a mission.

The four, who stopped in Kansas City earlier this week, are making their way across the country in a giant RV with 12-year-old Gabe’s face on the side. They want to spread awareness about children like Gabe, who was diagnosed with Duchenne muscular dystrophy, a progressive muscular disorder, when he was just 3.

They’re especially concerned about a recent price spike in a steroid that treats Gabe’s disorder.

For seven years, Gabe has taken a daily dose of deflazacort — a common glucocorticoid prescribed around the world for conditions like asthma, juvenile chronic arthritis and ulcerative colitis.

The Griffins had been buying the drug from foreign pharmacies for $418 a year. But the common pill has now been approved by the U.S. Food and Drug Administration to be marketed in the U.S. as Emflaza by the drug firm PTC Therapeutics.

The price in the U.S.: $35,000 a year for a 55-pound child. Gabe weighs 75 pounds, so the drug firm charges his insurance company $50,000, Scott Griffin said.

Some insurance companies don’t cover Emflaza, like Gabe’s does, and force families to pay out-of-pocket for a drug that used to be easily accessible.

“There are 169 companies making this drug around the world,” Griffin said in a release. “Tax payers get the bill when Medicaid is getting charged 11,000-12,000 percent above the global market price.”

The FDA justified the higher price under the Orphan Drug Act, a law that gives companies incentives, like setting a private market price, to sell drugs for rare diseases. Although this drug was issued around the world, it had never been approved in the U.S. and the FDA considered it a new drug.

PTC Therapeutics was unable to comment for this article but told NBC that the drug “represents sustainable pricing, which balances providing access to eligible patients in the United States ... while maintaining sufficient infrastructure and programs including continued investment in Duchenne.”

Duchenne syndrome is a rare form of muscular dystrophy that typically affects young boys. Symptoms include frequent falls, trouble getting up or running, big calves and learning disabilities.

While these children already struggle to fight their disease, they are now faced with fighting for access to a common drug that is now labeled a “breakthrough” drug.

The Griffin family isn’t just driving around in an RV with Gabe’s picture on it to raise awareness of this issue and Duchenne muscular dystrophy in general.

This is the third summer they’ve embarked on a mission known as Ride4Gabe. This year, the motor home is following a bicyclist, Brian Toone. He’s not only riding to finish “The Toughest Bike Race in the World,” the 3,070-mile Race Across America, but to create awareness and raise money for “The Toughest Fight in the World” — Duchenne muscular dystrophy.

The Hope for Gabe Foundation has a GoFundMe page where people can sponsor a mile.

“Turning 12 years old is an important milestone in a child with Duchenne, so that’s why we decided to try to raise $12 per mile this year,” Griffin said.

The race began June 13 in Oceanside, Calif., and top finishers will end this week in Annapolis, Md., with Toone representing the Griffins’ nonprofit Hope for Gabe Foundation.

“I am really excited for the race and to carry our message across the country. Something needs to be done to make sure that boys like Gabe have access to life-saving and life-changing medicine,” Toone said in a release.

With 2,474 out of the 3,070 miles already completed, the Griffins have raised $6,089 through Toone’s 2017 Racing for Accelerated Access to Medicine campaign and hope to reach their goal of $20,000 not just for their son, but for every other kid facing this disorder .

Gabe acts like any other 12-year-old boy, besides taking the daily medication and shots, with red hair and freckles and an admiration for all sports. He has a sense of optimism about him, saying one day he just hopes to be able to ride a bike and climb the stairs like other kids.

Griffin calls his son his hero. He smiles as his son relays all the sports he plays, when really because of his disease, he can only watch from the sideline as an “honorary captain.”

“Typically, based on statistics, they lose their life around 17 to 24 years old,” Griffin said. “No statistic is good enough for us until I see him married and walking down the aisle and having grandchildren of his own. ...

“That’s why we’re fighting, and that’s why we’re going to continue to fight. I believe that this generation of patients with Duchenne could be the first generation to actually survive it.”

This story was originally published June 21, 2017 at 5:18 PM with the headline "Cross-country trip calls attention to huge price spike for muscular dystrophy drug."