Christine McSherry is used to a fight. Her son, Jett, was diagnosed with Duchenne muscular dystrophy, a rare, debilitating condition, when he was 5. He lost the ability to walk just before he turned 14.
McSherry has become an outspoken advocate for Jett and other kids with Duchenne, particularly in fighting for access to drugs she's convinced help against the disease's devastating effects.
What she didn't expect to be fighting for was access to a medicine Jett was already using.
"It's all about choice, and choice of a reasonable price," McSherry, executive director of the non-profit Jett Foundation, said in an interview.
Jett, now 21, has been taking a steroid called deflazacort since he was 8 years old. He switched to it after trying another steroid, prednisone, to which McSherry says "he didn't have a great reaction."
Deflazacort wasn't approved in the U.S., so McSherry and a number of other families of kids with Duchenne imported the drug from overseas. She says it cost about $1,200 a year out of pocket.
Monday, deflazacort got a new price tag: $35,000 a year for a patient in the U.S. weighing 25 kilograms, or about 55 pounds.
To McSherry, the $35,000 is "pretty aggressive." What's worse, in her eyes, is that most kids who may take the drug weigh more than 25 kilograms, meaning the price will be even higher.
"You have young boys, as little as 30 kilograms, to young men who are 100-plus kilograms," McSherry said. "That price is up there over $100,000 annually."
PTC Therapeutics, which set the price, says it "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."
The route to deflazacort's new price is a complicated one. Earlier this year, a private drug company called Marathon Pharmaceuticals acquired rights to deflazacort in the U.S. and took it through the approval process at the Food and Drug Administration. Upon approval, Marathon set an annual price in the U.S. of $89,000, generating an immediate outcry from the patient community. Once the medicine got approval in the U.S., patients expected they wouldn't be able to legally import it from overseas.
The patient response to Marathon's price was so strong the company paused its plans to introduce the drug in the U.S. That's when PTC Therapeutics stepped in, paying $140 million to Marathon to acquire deflazacort — now with the brand name Emflaza.
"PTC came in, acquired the asset, and has sort of — for lack of a better word — re-thunk the commercial approach to the drug," said Ritu Baral, an analyst with Cowen.
But its new price tag is still drawing criticism.
"It's the drug industry back in trouble again over pricing," said Erik Gordon, a professor at University of Michigan's Ross School of Business. "They just don't seem to learn their lesson."
With its weight-based pricing, some say PTC may be taking a page out of the book of another maker of drugs for Duchenne: Sarepta, which received approval last year of Exondys 51. The company also set a weight-based price, of $300,000 a year for a 25-kilogram patient.
"We know from what Sarepta has said on public calls that the average age of patients they're seeing on eteplirsen (the generic name for Exondys 51) is in the 13- to 15-year-old range," said Matthew Eckler, an analyst with RBC Capital Markets. "These are much heavier than seven- to eight-year-old boys."
The heavier the patient, the more the drug costs.
"If you're trying to sell something, would you rather tell someone it costs $300,000 a year, or $500,000 a year?" Eckler said.
Sarepta supporters argue that drug should command a higher price because Sarepta took it all the way through research and development, and it represents a new way to try to treat the disease, whereas deflazacort is a decades-old steroid.
PTC CEO Stuart Peltz said on a conference call Monday that the company would have financial assistance programs in place to try to ensure patients pay as close to nothing as possible for the medicine. But those in the community, like McSherry, are concerned about the paperwork hoops they and their doctors will have to jump through to get coverage for Emflaza.
"What barriers do we have with prior authorization from payers?" McSherry asked, citing requirements pharmacy benefits managers and insurers often put on more expensive medicines to make patients try cheaper options first.
"Where does this fall — on drug companies or patients? The burden is really with the patients and families," she said. "We're the ones who are going to have to write all these appeals to get this drug covered, or find alternative ways to get ahold of the drug."
Pharmacy benefits manager Express Scripts confirmed today it will require prior authorization for Emflaza.
"For management of DMD, our criteria requires patients to try the lower-cost, equally effective prednisone before Emflaza," spokesman Brian Henry wrote in an email. "Except for rare cases, the lower-cost prednisone should work for patients."
Henry said prednisone's price varies from 8 cents to $1.50 per tablet, at maximum "$135 a month."
Many patients — and PTC — argue the drugs are not the same. McSherry cited concerns about weight gain with prednisone, which "does make a difference in Duchenne."
"Kids who have more to carry around will have more difficulty," McSherry pointed out.
Analysts said they expect the political scrutiny on Emflaza's price to continue. Sen. Bernie Sanders and Rep. Elijah Cummings wrote a letter to PTC in March requesting information on its pricing plans. Neither congressman responded to requests for comment Monday. PTC's stock declined 12 percent.
For McSherry, who's increasingly concerned about the effects health reform will have on her ability to pay for Jett's care, it's a headache she, and the rest of the community, doesn't need.
"You're talking about a population of rare-disease patients and families and physicians who are already burdened with a terrible disease," McSherry said. "To be burdened further on a daily basis with trying to get prior authorization for some of these drugs is very difficult."