Twin boys with muscular dystrophy have been denied insurance coverage for a drug that they helped get approved by participating in a clinical trial.
Sufferers of Duchenne muscular dystrophy, 15-year-old twins Jack and Nolan Willis of Fayetteville, New York, took part in a clinical trial that led to the recent approval of the first drug to treat their rare disease.
Now their mother’s insurer, Excellus BlueCross BlueShield, refuses to pay for the drug, Exondys 51, that costs in excess of $1.5 million annually to cover both teens. The drug is covered by the insurer for Duchene patients capable of walking, but the Willis twins can no longer walk and must use wheelchairs.
Jack and Nolan dedicated five years to the clinical trial, along with 12 other boys, and appeared before a Food and Drug Administration panel in 2016 to lobby for approval of the drug.
The boys’ mother, Alison Willis Hoke said, “After sacrificing their bodies, time, friends and lives for more than five years, it’s horrifying they are being denied this drug.”
Jack and Nolan are currently receiving Exondys 51 free of charge from Sarepta, the company that makes it. But Willis Hoke and her sons are unsure how long that will continue. According to Willis Hoke, Exondys 51 has helped preserve her twins’ heart and lung function. Most sufferers succumb to the disease in their 20s.
“If this drug was taken away, they would circle the drain so quickly,” Willis Hoke said.
Ian Estepan, a spokesman for Sarepta, said via email that the company will provide “continuity of care” for the boys as long as possible. “However providing free drug is an unsustainable long-term business model for a company highly focused on R&D (research and development) and the continued development of drugs for unmet medical needs.”
Excellus continues to deny Willis Hoke’s claims. She is currently appealing the decision for the third time. Jack and Nolan, sophomores at Fayetteville-Manlius High School, are doing their part by posting an online petition to fight the Excellus decision.
“As patients with a disease that worsens every day, we have hoped and prayed that someday everyone would have a chance,” the boys wrote in the petition. “With Exondys 51, that day is here.”
The Willis twins have hope due to the case of Andrew Longwell, 19, a Duchenne muscular dystrophy patient from Greece, New York, who also uses a wheelchair. Longwell was not a participant in the clinical trial, but he launched an online petition in December after Excellus refused to cover Exondys 51 for him.
After an independent review deemed the drug medically necessary for Longwell, Excellus reversed its decision in January.
Willis Hoke worries what will happen if her sons have to be taken off of Exondys 51.
She said, “I don’t have a million bucks in my pocket to pay for an infusion drug for my sons.”
The twins were featured in an Muscular Dystrophy Association telethon in 2013.
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