Diagnosis: Tuberous sclerosis
Treatment: Epidiolex
The insurer denied Epidiolex.
The determination is upheld.

This is a patient with tuberous sclerosis. As a consequence of his tuberous sclerosis, he has epileptic seizures. The seizures are focal in onset and consist of behavioral arrest, eye twitching and hand twitching and have a duration of 10-20 seconds. Despite treatment with levetiracetam and Onfi, he has continued to have multiple seizures per day. In an attempt to better control the seizures, his neurologist has prescribed Epidiolex (cannabidiol). At issue is the medical necessity of the Epidiolex.
The use of Epidiolex for the treatment of seizures due to tuberous sclerosis is not medically necessary. This is a patient who has epileptic seizures secondary to tuberous sclerosis. Tuberous sclerosis is an autosomal dominantly-inherited disorder that affects many organ systems through abnormalities in growth and differentiation of tissues. In the central nervous system, tuberous sclerosis often leads to cortical tubers and epilepsy. The epileptic seizures of tuberous sclerosis are often refractory to medical treatment. Epidiolex (cannabidiol) is a compound derived from the marijuana plant that has anti-epileptic properties in some forms of seizure disorder. In particular, Epidiolex has been shown to be effective as an anticonvulsant in Lennox-Gastaut Syndrome and in Dravet Syndrome, which are both epileptic encephalopathies. While Epidiolex is effective against some seizure types, it is not known to be effective against the seizures of tuberous sclerosis. There have been no double-blind placebo-controlled studies published demonstrating the efficacy of Epidiolex against the seizures of tuberous sclerosis. It is not considered standard of care and is not medically necessary for the treatment of this patient's seizures.