Appeal type: Medical Necessity
Decision: Overturned

Summary Reviewer 

The patient is an 11-year-old male who has been diagnosed with medically refractory epilepsy. The patientas parent has requested authorization and coverage for Epidiolex 100 mg/ml solution. The physician reviewer found that Epidiolex 100 mg/ml solution is medically necessary for the treatment of the patientas medical condition. He has medically refractory epilepsy. His seizures are complex partial which secondarily generalize into tonic clonic and drop seizures. His EEG was markedly abnormal, capturing multiple clinical and subclinical seizures. His drop seizures in particular place him at significant risk of injury. In an extensive review of injuries in patients with epilepsy, Nguyen and Tellez Zenteno noted that risk factors for injuries associated with epilepsy include seizure frequency, type of seizure, and associated use of anti-epileptic drugs. Polypharmacy increases risk of injury, and may also predispose to osteopenia and osteoporosis, which can increase the risk of fractures. The authors described that the most common injuries were burns, fractures, soft-tissue injuries, and submersion accidents. Epidiolex is a cannabidiol medication that has a high potential to decrease seizure frequency in the patient. Silvestro and colleagues noted, aCannabidiol (CBD) is one of the cannabinoids with non-psychotropic action, extracted from Cannabis sativa. CBD is a terpenophenol and it has received a great scientific interest thanks to its medical applications. This compound showed efficacy as anti-seizure, antipsychotic, neuroprotective, antidepressant and anxiolytic. The neuroprotective activity appears linked to its excellent anti-inflammatory and antioxidant properties.a The authors go on to state, aThe results of scientific studies obtained so far the use of CBD in clinical applications could represent hope for patients who are resistant to all conventional anti-epileptic drugs.a The pharmaceutical company which manufactures Epidiolex obtained U.S. Food and Drug Administration (FDA) approval for the drugas use in Lennox-Gastaut syndrome and Dravet syndrome, both of which are conditions eligible for the orphan drug pathway because this enabled them to bring the medication to market with fewer phase 3 pivotal efficacy randomized controlled clinical trials. The drug does not have biological specificity for these two conditions, however; OaConnell and colleagues noted, aOpen-label studies with 100 mg/ml CBD (EpidiolexA, GW Pharmaceuticals) have provided additional evidence of its efficacy along with an adequate safety profile (including certain drug interactions) in children and young adults with a spectrum of treatment resistant epilepsies.a Sekar and colleagues reviewed the use of Epidiolex as an adjunct therapy in the treatment of intractable epilepsy. They reported several open-label, expanded-access trials studying the use of Epidiolex in patients with treatment-resistant epilepsy of multiple etiologies as showing similar benefits to the studies of Epidiolex with Dravet and Lennox-Gastaut syndromes. There is sufficient support for the requested medication in this clinical setting. Therefore, Epidiolex 100 mg/ml solution is medically necessary for the treatment of this patient.