Authors :
Leah Burn, MPH, DrPH – Jazz Pharmaceuticals, Inc.
Rachel Faller, PhD, MPH – Target RWE
Kara Bennett, MS – Target RWE
Michael Faithe, PharmD – Jazz Pharmaceuticals, Inc.
Miranda Harrison, BSc – Jazz Pharmaceuticals, UK Ltd.
Vicki Osborne, PhD – Jazz Pharmaceuticals, UK Ltd.
David Pritchard, PhD – Target RWE
Karthik Rajasekaran, PhD – Jazz Pharmaceuticals, Inc.
Presenting Author: Arthur Sillah, PhD – Jazz Pharmaceuticals, Inc.
Rationale:
Efficacy of cannabidiol (CBD; Epidiolex®, 100 mg/mL oral solution) for treatment of seizures associated with Dravet syndrome (DS), Lennox-Gastaut syndrome (LGS), and tuberous sclerosis complex (TSC) has been established by randomized clinical trials. In patients with refractory epilepsy, inadequate seizure control or undesirable side effects may result in antiseizure medication (ASM) cycling, polypharmacy, and increased healthcare resource utilization (HCRU). Data on the real-world impact of CBD on these outcomes remain limited. This retrospective cohort study used the US Optum® Market Clarity Database to assess the real-world effectiveness of CBD on these outcomes among CBD-naive adult and pediatric patients with DS, LGS, or TSC.Methods:
Adult (aged ≥ 18 years) and pediatric (aged < 18 years) patients who newly initiated CBD between June 25, 2018, and September 30, 2023, were included. Baseline period was defined as the 12 months before CBD initiation (index event) and follow-up was ≤12 months post-initiation. An interrupted time series analysis was conducted to assess observed changes in ASM cycling (number of new maintenance ASMs per patient per year), polypharmacy burden (number of concomitant maintenance ASMs per patient per month [PPPM]), and HCRU (medical claims or records of seizure-related hospitalization, emergency department [ED] visits, or physician office visits PPPM) 12 months before and after CBD initiation relative to the expected trajectory if CBD had not been initiated. Results were stratified by epilepsy type and age group.Results:
A total of 2937 pediatric (DS, n=1362; LGS, n=1406; TSC, n=169) and 1624 adult (DS, n=468; LGS, n=1044; TSC, n=112) patients were included. Most were male (56% pediatric; 55% adult), non-Hispanic white (50%; 60%), and had intellectual disability or developmental delay (63%; 54%). At 12 months post–CBD initiation, mean reductions in ASM cycling were observed among both groups in the overall cohort (pediatric, 34%; adult, 28%) and within each indication-specific subgroup (Table 1). Reductions were also seen in polypharmacy (30%; 12%) and HCRU (hospitalizations [62%; 40%], ED visits [69%; 65%], and physician visits [28%; 8%]), in pediatric and adult groups, respectively, in the overall cohort and within the indication-specific subgroups (no change was seen in physician visits among adults with LGS) (Table 2).
Conclusions:
These real-world insights indicate overall reductions in ASM cycling, polypharmacy burden, and HCRU at 12 months post–CBD initiation in both pediatric and adult patients with DS, LGS, or TSC. An overall reduction in these parameters suggests that CBD may help reduce the clinical and economic burden of disease in both pediatric and adult patients living with these rare epilepsies.
Funding:
Jazz Pharmaceuticals, Inc.