Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Duchenne Muscular Dystrophy
Issue under review: Emflaza
The insurer denied the Emflaza. 
The denial is overturned. 

The patient is a male with Duchenne muscular dystrophy (DMD) diagnosed and confirmed by genetic testing.  The patient's provider noted the patient is being evaluated for absence seizures and that he has documented behavioral and impulse control issues and this precludes him from steroid therapy.  

The patient saw his provider in regards to his episodes of staring and unresponsiveness, including an episode involving urinary incontinence. The patient's parents also noted he was becoming weaker, and his physical therapist noted he was not able to travel stairs as he had been previously. The patient was very impulsive and had gotten in fights with other children at school, and he did not follow his teacher's instructions. The patient's parents want to try him on a small dose of steroids to see how he tolerates it.  

On exam, he was noted to be hyperactive and he had mild scoliosis. He had tight adductors, hamstrings, and heel cords.  Muscle tone was noted to be low and he had low muscle bulk and hypertrophic calves. He had a tendency to walk on his tiptoes. Muscle strength was 4/5 throughout. He was not cooperative with confrontational testing.  Gait was waddling.  Continued stretching exercises were recommended including swimming and riding a bike.  He was also recommended to wear orthoses.  The patient's provider recommended the patient start on Emflaza. Famotidine, vitamin D, and iron were also recommended. EEG was to be obtained to rule out absence seizures. Continued physical and occupational therapy was also recommended. 

Yes, the proposed treatment, Emflaza is medically necessary. 

According to the treatment guidelines published in Lancet in 2018, reference 3, as well as the practice parameter by the American Academy of neurology, reference 4, deflazacort (Emflaza) is indicated and is medically necessary in cases of Duchenne muscular dystrophy. Subsequent publications, references 1 and 2, indicate that deflazacort causes lesser weight gain comparing to prednisone, and preserves motor strength for longer. Therefore, in the patient's case the requested drug, deflazacort, is being used for a medically necessary indication as supported by the current medical literature and by treatment guidelines, and is medically necessary since each is expected to prevent deterioration of the patient's strength and avoid side effects at the same time. 

The letter of denial indicates that "Emflaza is likely to give the same effect as other corticosteroids. An example is prednisone." However, the plan does not consider the risk/benefit analysis, where the medical literature clearly indicates that deflazacort/Emflaza is expected to maintain the patient's motor strands for longer comparing to prednisone, and is expected to cause lesser weight gain. In patients with Duchenne muscular dystrophy, the combination of declining motor skills and becoming overweight, commonly lead to loss of ambulation. Therefore, considering the combination of these two factors and plasma is much more favorable than prednisone in order to maintain the patient's ambulation for longer.