Diagnosis: Becker Duchenne Muscular Dystrophy.
Treatment: Emflaza.

The insurer denied the Emflaza.
The denial is overturned.

The patient is a male with diagnosed Becker/Duchenne muscular dystrophy. His diagnosis was confirmed with genetic testing. He began treatment with deflazacort (Emflaza) at the time of diagnosis, consistent with the standard of care. While still ambulatory, he requires assistance to stand and has an elevator in his home. He falls on average once a month but has so far suffered no head or other serious injuries. He has undergone surgery for cataracts, has dyspnea walking a block, and has chronic dry cough. He recently has been diagnosed with gastroesophageal reflux disease and started on PPI (proton pump inhibitor) therapy. He continues on Emflaza therapy.

Yes, the requested treatment with Emflaza is medically necessary.

Corticosteroid use is standard of care for patients with Duchenne muscular dystrophy. Studies have shown that corticosteroids yield short-term improvement in both muscle strength and function (up to twelve months) and strength up to two years. Deflazacort, an oxazolone derivative of prednisolone, has been associated with less side effects that prednisone. This young man has history of abdominal pain and GERD (gastroesophageal reflux disease), and he is not a good candidate for prednisone therapy. Deflazacort is not without side effects, but in the interest of providing this young man with standard of care treatment and a chance at prolonged muscle strength and function, deflazacort has a better side effect profile than prednisone and is therefore medically necessary and appropriate for him.