Appeal type: Medical Necessity
Decision: Overturned

Nature of Statutory Criteria/ Case Summary:  The enrollees representative is requesting authorization and coverage for the medication, Xyrem. The enrollee has classical narcolepsy type 1 with cataplexy, anxiety, depression, migraine, and post-traumatic stress disorder (PTSD).  Her mother reports excessive daytime sleepiness (EDS) since age five. Symptoms progressed to lower extremity weakness and falling asleep during daytime activities. At age eleven, she underwent a sleep study with a resultant diagnosis of sleep apnea. She was referred to a neurologist, who performed a Multiple Sleep Latency Test (MSLT) that showed a single episode of sleep-onset rapid eye movement and normal sleep onset latency. The neurologist suspected narcolepsy, but because of the negative MSLT, opted to treat the enrollee symptomatically. A stimulant, Adderall, and an antidepressant, Celexa, were prescribed, which resulted in some symptom improvement with regard to hypersomnia, but her EDS continued. Her medications were then changed to Focalin XR and Celexa, resulting in a worsening of her symptoms. The neurologist repeated the MSLT and  formally diagnosed the enrollee with narcolepsy type 1.  Her dose of Focalin XR was adjusted and Celexa continued. Subsequently, her medication regimen was changed to Adderall XR and Celexa. This improved daytime alertness and reduced frequency and severity of cataplexy, but she continued to have marked EDS.  Her physicians added Xyrem to her regimen of Adderall XR and Celexa, to improve sleep maintenance and quality, and improve her ongoing symptoms of cataplexy and EDS. Per the medical literature,  Xyrem is a central nervous system depressant indicated for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients seven years of age and older with narcolepsy. They concluded in a study that sodium oxybate (Xyrem) was superior to placebo for reducing weekly cataplexy attacks, increasing maintenance wakefulness and reducing sleep attacks. The study showed that narcolepsy enrollees on sodium oxybate (Xyrem) had significant symptom reduction and the medication is well tolerated, with most adverse events being mild to moderate in severity.  Medical literature indicates treatment options for cataplexy in children with narcolepsy type 1 include sodium oxybate (Xyrem) and certain antidepressant medications. When cataplexy is the most bothersome symptom, or when cataplexy and sleepiness are equally severe, initial therapy with sodium oxybate is suggested. The rationale for sodium oxybate as the initial agent is that it targets both cataplexy and daytime sleepiness and is the most potent anticataplectic therapy available. The enrollee has been taking Adderall XR and Celexa and Xyrem was added.    However, the literature does not support the enrollee must trial these medications prior to treatment with Xyrem. Final Result: The reviewer determined that the requested medication is medically necessary for the treatment of the enrollees medical condition. Credentials/Qualifications: The physician reviewer is board certified in Pediatric Neurology and is actively practicing. The reviewer is an expert 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 similar medical condition.