Rare Disease Report

Another Duchenne Boy Fighting to Get Approved Drug (Exondys 51)

MAY 23, 2017
James Radke
Every week there seems to be another story of a patient with Duchenne muscular dystrophy (DMD) being denied access – permanently or temporarily – to Exondys 51 (eteplirsen). 
This week, the story involves Jonah, a 17-year-old young man with DMD living in the Minneapolis / St Paul area.
Exondys 51 was given accelerated approval by the FDA last fall for the treatment of a patients with DMD amendable to exon 51 skipping therapy.
Jonah was diagnosed with DMD when he was 5 years old and is currently requires a wheelchair to get around.

For those of you following the Exondys 51 story, you probably know where this one is going. Since Jonah is non-ambulatory and since the approval of Exondys 51 was based on data involving DMD patients who were ambulatory, insurance companies have been reluctant to provide it to non-ambulatory patients even though the indication for the drug does not specify it is only for ambulatory patients.
And while that is a common story line with Exondys 51, that may or may not be the underlying reason behind Johan being denied coverage. In Jonah’s case, there appears to be a lot of groups trying to pass the bill along to other parties – be it the pharmacy, the hospital, or the patient, and as will be explained below, it is still not clear who will be paying for the $300,000 treatment.

Medicaid and Exondys 51

Jonah is covered by Medicaid and although the family had received 2 denial letters for coverage of the drug, they learned last week that following a Department of Human Services (DHS) hearing, Medicaid will pay for the medication as long as the treatment is performed in a hospital.
In a statement, the Minnesota DHS said, "Medicaid generally covers this drug. However, state law requires that medications administered by a physician need to be billed by the treating physician and not a pharmacist. This is to ensure drugs are handled properly. DHS routinely works with patients and doctors to resolve billing issues and ensure clients get the care they need."
So for now, Jonah will be getting the treatment but it is still unclear who will be paying for the drug.  Dr. Steven Koop with Gillette Children's Hospital, where Jonah gets treatment, says the DHS has not guaranteed that the hospital would be reimbursed by Medicaid if they buy the $300,000 drug for Jonah. Normally, such drugs would be bought through a pharmacy but as per the DHS’s statement, Medicaid will not reimburse a pharmacist.
In an interview with NBC News, Dr Koop noted, "I think the conversation has to be not through us but between those who create and market the drugs and those who pay the bills."
Jonah’s family is relieved they will be getting the treatment soon but they wished it could have been resolved earlier. Their appeal process lasted 10 months and in that time, irreversible damage has been done to Jonah’s muscles.

Image obtained from the original story aired on NBC news. 

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