To characterize real-world dosing patterns for Epidiolex®. 

A plant-derived highly purified pharmaceutical formulation of CBD (Epidiolex®, 100 mg/mL oral solution) is approved for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC) in patients aged ≥1 years. For patients without moderate or severe hepatic dysfunction, the recommended therapeutic dosage ranges from 10-20 mg/kg/day for LGS and DS and 25 mg/kg/day for TSC. 

We analyzed patient-level, de-identified US specialty pharmacy fill records for new CBD users from November 15, 2018 to July 12, 2023. Dosage was derived from patient weight, quantity dispensed, and days’ supply. Discontinuation was defined as a 60-day supply gap; patients with continuous supply beyond July 12, 2023, were censored at their last recorded day of supply. Persistence (initial CBD pharmacy fill through discontinuation/censoring) was analyzed via Kaplan-Meier method. 

We identified 19,444 new users. The overall probability of persistence at 12 months was 69.9%. The distribution of the starting dosages at ≤5, >5-10, >10-15, >15-20, >20-25, and >25 mg/kg/d were 16.8%, 53.7%, 19.7%, 5.7%, 2.3%, and 1.7%, respectively. The proportions shifted over time toward higher doses reaching 4.0%, 18.8%, 25.6%, 28.6%, 15.9%, and 6.9%, respectively, at 12 months. Cumulatively, during their initial continuous treatment period and up to 12 months, 66.1% of treatment-months were at doses >10; 38.7%, >15; and 15.6%, >20 mg/kg/d. The risk for discontinuation was highest in the second month of treatment. 

Based on US specialty pharmacy data, most patients at 12 months were taking dosages >15 mg/kg/day. Long-term persistence on CBD may be associated with dose optimization over time, leading to a balance between tolerability and seizure reduction.