Rare Disease Report

Wheelchair Discrimination: Non-ambulatory Duchenne Twins Denied Drug Coverage

APRIL 04, 2017
RDR Staff
After participating in the clinical trial that fast-tracked Exondys 51 (eteplirsen) for approval by the FDA, the Willis twins are now being denied coverage for the same drug.

Jack and Nolan Willis are 15-year-old twin brothers both suffering from Duchenne Muscular Dystrophy (DMD), a rare genetic disease. They are being denied the drug because they are no longer ambulatory and Excellus BlueCross BlueShield, their provider, is only providing the drug to those who can walk.

When the brothers were around 10-years-old they began participating in a clinical trial for the drug, Exondys 51. In September 2016 the drug was approved under the accelerated approval pathway that the FDA uses to fast track treatment approvals for life-threatening diseases that provide meaningful advantages over other treatments. The accelerated approval means that the company developing the drug, Sarepta, needs to complete an additional Phase 3 study to determine once and for all that is it safe and effective. The approval in September was based on a Phase 2 study that was very controversial.
Dr. Robert Dracker, who has been administering Exondys 51 to both Jack and Nolan, notes that the treatment is more effective the earlier a DMD patient receives it. But without the drug the twins’ “deterioration would worsen.” 

Exondys 51 Indication Does Not Restrict the Drug to Non-ambulatory Patients

The indication for Exondys 51 is for DMD patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. The indication is not restricted to patients who are ambulatory.

In an exclusive interview with Rare Disease Report, Christine McSherry, mother of a wheelchair bound DMD patient, noted that the drug still made a significant impact on her son’s abilities.  That interview is shown below.

Change.Org Campaign

The Willis twins hope to change the position of Excellus and currently have a campaign running on Change.org.

In a letter to supporters, they wrote, “We’ve been in the drug’s clinical trial for more than five years and the drug was approved based on data from us. Other boys in New York and across the country are now able to get access to it. However, our insurance company, Excellus BlueCross BlueShield, is denying us coverage – it’s absurd! In short, we feel that we’re being discriminated against by our insurance company because we are confined to wheelchairs.”

Sarepta to the Rescue (for now)

For now Sarepta will continue to provide the drug to the twins free of cost as “continuity of care.” But that cannot last forever. Ian Estepan, a spokesman for Sarepta says that although they want to help the Willis twins, “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.”

Without the help of Sarepta, the drugs for both boys will run the family approximately $800,000 to $900,000 annually for each child.

About Duchenne Muscular Dystrophy (DMD

DMD is caused by lack of a functional dystrophin protein, a protein that helps keep muscle cells intact. Patients with progressive muscle disorder experience symptoms in early childhood, losing the ability to walk as early as age 10. These patients, mostly boys, experience life-threatening heart and lung complications in their late teens and twenties.

There are many subsets of the Duchenne population based on the type of mutation found in the dystrophin gene.

Exondys 51 (eteplirsen)  will provide treatment for approximately 13% of those in the DMD population with a specific mutation of the dystrophin gene susceptible to exon 51 skipping. The drug allows the RNA to skip over the mutation allowing for a truncated, yet functional dystrophin protein to be produced. The addition of functional dystrophin protein in turn slows down the degenerative effects of DMD.

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