Insurance provider approved cost of Kennedy Township teen’s muscular dystrophy treatment drug – Timesonline.com

KENNEDY TWP. -- A township teen and his family have been elated to learn that his health insurance provider will cover the cost the expensive drug that has likely slowed the progression of his Duchenne muscular dystrophy.

Billy Ellsworth, 16, of Kennedy Township, had been on a clinical trial for eteplirsen -- now known by its commercial name exondys51 -- at the Children's Hospital of Pittsburgh of UPMC for several years.

The drug was tentatively approved by the Food and Drug Administration in September, but that meant the cost of the drug -- around $300,000 a year -- would eventually be passed onto the family.

Highmark approved the cost of the life-extending drug on Jan. 30 after the Ellwsorths were initially denied coverage twice last month.

"These trial boys, and there were 12 of them, were kind of in a state of flux or a gray area because the trial is officially over, but they still needed research until our insurance went through," said Terri Ellsworth, Billy's mother. "Billy never went without drugs, and of course, no, we never had to pay a dime."

According to the FDA, people with Duchenne progressively lose the ability to perform activities independently and often require use of a wheelchair by their early teens. Life-threatening heart and respiratory conditions can occur as the disease progresses.

However, Billy is able to walk to his classes and to his doctor's appointments. He credits his continued independence from the drug he's been taking, which is manufactured by Sarepta pharmaceutical company.

Terri Ellsworth understands why the drug is so expensive. She doesn't see it as price gauging, but it's simply how the system works because the research behind finding effective drugs for rare diseases is time consuming and costly.

"All orphan, rare disease drugs, such as Billy's, for example, all come with a cost. They average between $300,000 and $500,000 (per year). That's just the nature of a rare disease drug," she said.

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Insurance provider approved cost of Kennedy Township teen's muscular dystrophy treatment drug - Timesonline.com