Appeal type: Experimental/Investigational
Decision: Overturned

Nature of Statutory Criteria/Case Summary: An enrollees parent has requested authorization and coverage for Botox injections (200 units every 12 weeks). Medical literatures describe botulinum toxin as a potent muscle relaxant that has been found to have analgesic effects in patients with a variety of pain syndromes. The authors further state, Both in vitro and in vivo studies showed the ability of the toxin to block the release of pain neurotransmitters, such as substance P, glutamate, and calcitonin gene-related peptide. The effect of the toxin, and specifically of one of its serotypes, botulinum neurotoxin type A, on headaches, has been extensively studied. This serotype is available in the United States in three forms, including as onabotulinumtoxinA. Data from clinical trials confirmed the efficacy, safety, and tolerability of onabotulinumtoxinA in the prophylactic treatment of chronic migraine, the most severe and debilitating type of migraine, in adults. The authors further note that the U.S. Food and Drug Administration (FDA) approved the use of this drug for this indication in 2010. In a retrospective review of available data of all patients who received onabotulinumtoxinA for chronic migraine in a large pediatric headache center from 2004 to 2010, researchers concluded that Botox is recommended to any pediatric patient with chronic headache if they fail two or more preventive medications. Their study showed a major change in the frequency of the headache with a statistical difference in the improvement of headache days per month, with a 30-point drop in the pediatric disability scoring between first injection and follow-up injection with a change from severe disability to moderate disability. Researchers found that pediatric patients who had been treated with onabotulinumtoxinA for chronic migraine had statistically significant improvements from baseline over a five-year longitudinal period. Likewise, researchers concluded that botulinum toxin type A injections were a safe and effective therapy for chronic migraine in children recalcitrant to medical therapy. In this case, the patient has tried numerous medications for prophylactic and acute treatment of chronic migraine without relief. Current medical literature supports its effective use, safety and tolerability in several pediatric studies. Therefore, Botox injections (200 units every 12 weeks) are likely to be more beneficial for treatment of the patients condition than any available standard therapy.Findings:  Two of the three physician reviewers found the requested Botox injections (200 units every 12 weeks) are likely to be more beneficial for treatment of the patients medical condition than any available standard therapy.Final Result: The reviewers determined that the requested services are likely to be more beneficial for treatment of the patients medical condition than any available standard therapy. Therefore, the Health Plans denial should be overturned. Credentials/Qualifications: Physician reviewers 1, 2, and 3 are board certified in neurology with special qualification in child neurology and are actively practicing. The reviewers are experts in the treatment of the enrollees medical condition and knowledgeable about the proposed treatment through recent or current actual clinical experience treating those with the same or a similar medical condition.