Muscular Dystrophy: when insurers say no, reviewers often say yes

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 parent of an enrollee has requested authorization and coverage for the medication Spinraza.Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder characterized by degeneration of spinal motor neurons. The condition causes significant morbidity and mortality in affected children, who have profound weakness affecting the limbs and also the respiratory muscles. SMA is caused by low levels of survival motor neuron (SMN) protein owing to deletions or mutations of the SMN1 gene. An almost identical gene SMN2, present on the same chromosome, produces a smaller, truncated protein (SMN2) because of skipping of exon 7 from translation due to translation silent C6U substitution in exon 7 of SMN2 pre-mRNA transcript. Only 10% of the SMN2 mRNAs produce full length SMN2 protein by including exon 7 in healthy individuals.

Nature of Statutory Criteria/Case Summary: The patient has requested authorization and coverage for oxycodone-acetaminophen 10-325 mg (#120 tablets per 30 days, one tablet every six hours as needed with six refills). The submitted documentation does not support the medical necessity of the requested medication. Manchikanti and colleagues state, “To establish medical necessity for opioid therapy, it is essential to have a physical diagnosis and information on inadequacy of multiple modalities of treatments including conservative, various other alternatives, and consultations if necessary.

These outcomes come from California’s external review program — an independent physician panel whose decision binds the insurer. Every state has an equivalent, and internal appeals succeed even more often. If your care for muscular dystrophy was denied, the published record says the denial is worth fighting.