Health Problem: Epilepsy is one of the most common neurological disorders in the United States and has a prevalence of approximately 3 million adults and 470,000 children. Management generally involves treatment with antiepileptic drugs (AEDs), but for approximately 30% of patients, seizures will remain uncontrolled by medical therapy. In these cases, other, more invasive, procedures might be warranted, including intracranial surgery or neurostimulation. Starting in 2013, the United States began to allow access to medicinal cannabis and individuals began to evaluate the effectiveness of seizure reduction in forms of refractory epilepsy. Lennox-Gastaut syndrome (LGS) and Dravet syndrome (DS) are 2 rare and severe forms of childhood-onset epilepsy that are known to be resistant to treatment with AEDs alone.

Technology Description: Epidiolex is an oral liquid formulation of highly purified extract of cannabidiol (CBD) in a sesame oil–based solution at a concentration of 100 milligrams per milliliter (mg/mL). This solution is given orally twice daily at a recommended starting dose of 5 mg per kilogram (kg)/day, which may then be increased based on patient response and tolerability to a maximum recommended dosage of 20 mg/kg daily. It is the first natural marijuana product to be approved by the FDA and was scheduled as a category V drug for limited addictive potential by the Drug Enforcement Agency. Epidiolex as an adjunct to AEDs is an alternative to more invasive treatment options for patients with LGS and DS, but other less well-studied forms of epilepsy remain off label for this treatment.

Controversy: Approximately 20% to 40% of patients with epilepsy have seizures not controlled by medical therapy, and surgical intervention or neurostimulation may be considered to reduce or halt seizures. Adjunctive treatment with Epidiolex in addition to AEDs represents a less invasive treatment option. While Epidiolex is FDA approved for the treatment of seizures associated with LGS or DS in patients 2 years of age and older, patients and providers may seek to use it off-label for other forms of treatment-resistant epilepsy.

Key Questions:

  • Is Epidiolex (CBD) effective in treating epilepsy in adults and children for epilepsy types other than LGS or DS?

  • Is Epidiolex safe for the treatment of non-LGS or DS epilepsy and are there long-term safety considerations?

  • Have definitive patient selection criteria been established for Epidiolex for the treatment of non-LGS or DS epilepsy?

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