Duchenne Muscular Dystrophy denials in California external review

What the reviewers wrote

Where the denial was overturned

Findings: The physician reviewer found that The patient’s 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.

Nature of Statutory Criteria/Case Summary: The parent of the patient has requested authorization and coverage for gene therapy with delandistrogene moxeparvovec-rokl (Elevidys). Based on the records, gene therapy with delandistrogene moxeparvovec-rokl (Elevidys) is likely to provide greater clinical benefit for this patient with Duchenne muscular dystrophy (DMD) compared to any available standard therapy. Elevidys is U.S Food and Drug Administration (FDA)-approved for use in DMD patients aged four years and older with confirmed mutations in the DMD gene, including deletions such as exons 45 to 58, as well as in patients who are ineligible for exon skipping therapies. This patient falls within this broader molecular eligibility and has no access to gene-targeted exon skipping interventions.

Where the denial was upheld

Nature of Statutory Criteria/Case Summary: The patient has DMD with progressive weakness and loss of mobility. The record establishes that the patient is currently able to ambulate household distances but cannot ambulate community distances. Given the progressive nature of his neuromuscular disease, the patient requires a power wheelchair for mobility and appropriate positioning and pressure relief with power tilt and recline and power elevating leg rests. However, the power standing feature is not required for the patient. There is insufficient clinical documentation indicating how the power standing feature will objectively improve his independence in performing daily activities. Further, there is also insufficient medical literature supporting its need in patients with DMD. There is some medical literature suggesting that these devices may be helpful in patients with DMD.

Nature of Statutory Criteria/Case Summary: The patient has requested authorization and coverage for power wheelchair accessories including standing feature and seat elevator. Findings: The physician reviewer found that muscular dystrophy is a condition caused by mutations in the dystrophin gene, resulting in degeneration of cardiac and skeletal muscle. Patients with this condition typically need to use a wheelchair for mobility to address issues such as fatigue and falls. Replacement wheelchairs are expected to be needed over time for with muscular dystrophy due to equipment wear and tear, which can progress to the point of breakdown. The typical life expectancy of a wheelchair has generally been reported to be up to five years. This patient’s records document a history of Duchenne muscular dystrophy with severe weakness affecting all extremities.

These outcomes describe eligible cases completed through California DMHC’s Independent Medical Review program. They do not estimate the chance that an internal appeal, an external review in another jurisdiction, or your individual case will succeed. Use the record to identify evidence patterns involving duchenne muscular dystrophy, then check the rights and deadlines that apply to your plan.