Appeal type: Medical Necessity
Decision: Overturned Decision of Health Plan

Findings:  The physician reviewer found that The patients parent has requested authorization and coverage for Emflaza 30 mg tablets. The American Academy of Neurology guideline on corticosteroid treatment of Duchenne muscular dystrophy (DMD) reports, Prednisone 0.75 mg/kg/d has significant benefit in DMD management and should be considered the optimal prednisone dose. Prednisone 10 mg/kg/weekend is equally effective over a 12-month period, although long-term outcomes of this alternate regimen remain to be seen. While the U.S. Food and Drug Administration (FDA) does not specifically approve the use of prednisone for the treatment of DMD, it approves the use of deflazacort (Emflaza) for the treatment of DMD in patients age five and older. In a study characterizing disease progression over 48 weeks among boys with DMD receiving deflazacort versus those treated with prednisone/prednisolone, the authors concluded that deflazacort-treated patients experienced significantly lower functional decline over 48 weeks. Marden and colleagues studied the outcomes of long-term prednisone and deflazacort in patients with DMD and reported that, Deflazacort was also associated with a lower risk of scoliosis and better ambulatory function, greater percent lean body mass, shorter stature and lower weight, after adjusting for age and steroid duration. The authors concluded that deflazacort was associated with improved clinical outcomes. In a comparison of the efficacies of deflazacort and prednisone/prednisolone in providing clinically meaningful delays in loss of physical milestones in patients with nonsense mutation DMD, Shieh and colleagues noted that there were significant improvements in change in 6-minute walking distances, and significant and clinically meaningful improvements in 4-stair climbs and 4-stair descends for deflazacort versus prednisone/prednisolone. The authors concluded, Deflazacort provides clinically meaningful delays in loss of physical milestones over 48 weeks compared with prednisone/prednisolone for patients with nonsense mutation DMD. Abutaleb and colleagues described the risk posed to patients with DMD who are suddenly changed from deflazacort to prednisone, stressing the potential danger of changing between corticosteroid classes in DMD cardiomyopathy. In this case, the records documented intolerable behavioral side effects including screaming, inattention, and hyperactivity when the patient was treated with prednisone. The patient has tolerated deflazacort without side effects. Given that suddenly replacing this medication with an equivalent dose of prednisone would place the patient at life-threatening risk for the development of acute myocarditis, and as current medical literature demonstrates the efficacy of deflazacort, which is associated with significant decrease in functional decline when compared with prednisone, along with a lower risk of scoliosis, greater percentage lean body mass, and lower weight, the requested medication is medically indicated. Therefore, Emflaza 30 mg tablets are medically necessary for the treatment of this patient.Final Result: The reviewer determined that the requested medication is medically necessary for treatment of the patients medical condition. Therefore, the Health Plans denial should be overturned.