Imagine devoting countless hours to take part in a five-year-long clinical trial for a life-saving drug that then gains approval by the Food and Drug Administration (FDA), only to be told by your health insurance provider that they will not cover it for you.
That’s the situation faced by Fayetteville-Manlius sophomores Jack and Nolan Willis.
Jack and Nolan, who are 15-year-old twins, have a life-threatening disease called Duchenne’s muscular dystrophy (DMD). This disease causes the deterioration and weakness of muscle mass throughout the body, including organs and skeletal muscle. DMD typically starts showing symptoms in children at around 4-years-old and, by 12, most diagnosed with the disease will lose their mobility. Those diagnosed with DMD have a life expectancy in the early-20s.
Though there is no known cure for the disease, for the past five years, Jack and Nolan were involved in a clinical trial for a drug called Exondys 51, which helps to manufacture a protein that will slow down the deterioration of muscles mass. Their mother, Alison Willis Hoke, said they spent 32 consecutive weeks out-of-state to receive the infusion, and eventually they were able to get their weekly infusions at a facility in Liverpool.
The drug was approved with an accelerated approval pathway in September 2016, which, according to a press release from the FDA, “provides for the approval of drugs that treat serious or life-threatening diseases and generally provide a meaningful advantage over existing treatments.”
Though the drug was approved and available for patients with DMD, Alison said their insurance company, Excellus Blue Cross Blue Shield, will not cover Jack and Nolan because they are wheelchair bound. Luckily, she said, the drug company who produces the drug is still allowing the Willis twins to receive the drug.
“Being involved with changing this disease feels empowering, instead of accepting the fact that I have a terminal illness. Moving to fight it really brings hope to the situation and others,” said Jack. “It’s very disappointing that after years of testing and commitment compiled with fighting the FDA for approval has all be halted by insurance coverage.”
Out-of-pocket, Alison estimates the drug would cost about $700,000 per child per year, something she does not have the means to support.
Alison said she believes, despite the fact Nolan and Jack are wheelchair bound, the drug could slow down the deterioration of their organ muscle mass, including the heart and lungs, which would cause less complications for her sons in the future.
“I feel they’re making an uneducated decision and that more ‘homework’ needs to be done on their end to show how beneficial this drug is,” said Alison.
Currently, Jack and Nolan are fighting back against Excellus’ decision to deny them coverage for the drug. A change.org petition was created to support their situation and has garnered more than 3,000 signatures.
“It’s understandable that the FDA and insurance companies have regulations but, when dealing with rare diseases, time is of the essence, specifically when it’s one of the only drugs out there,” said Jack. “In the long run, having access to a multitude of drugs only has benefit on the situation and is vitally important to prolonging people’s lives who have this disease.”
“I just hope they [Excellus BCBS] realize the magnitude of this situation and the profound effect it can have on children with this disease,” said Alison.
The Willis twins previously made news in Cazenovia when they were barred in 2014 from participating in the annual July 4 foot race by the Syracuse Chargers Track Club. The club said the boys’ racing buggies, pushed by a team of runners, would be a safety hazard to other runners. After 36 hours of national, negative coverage, the track club reversed its decision and allowed the brothers to race.
To view the petition, go to change.org/p/excellus-bcbs-ceo-christopher-c-booth-excellus-bcbs-is-denying-us-the-duchenne-muscular-dystrophy-drug-we-helped-get-approved.
