Authors :
Arthur Sillah, PhD – Jazz Pharmaceuticals, Inc.
Leah Burn, MPH, DrPH – Jazz Pharmaceuticals, Inc.
Rachel Faller, PhD, MPH – Target RWE
Timothy Saurer, PhD – Jazz Pharmaceuticals, Inc.
Teresa Greco, PhD – Jazz Pharmaceuticals, Inc.
Sanket Shah, MBBS, PhD – Jazz Pharmaceuticals, Inc.
Kara Bennett, MS – Target RWE
Nuvan Rathnayaka, PhD – Target RWE
Presenting Author: Marco Navetta, MD – Jazz Pharmaceuticals, Inc.
Rationale:
In phase 3 clinical trials leading to approval of plant-derived, highly purified cannabidiol (CBD; Epidiolex®, 100 mg/mL oral solution) for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC), trial participants were taking a median of 3 antiseizure medications (ASMs) at baseline and had previously discontinued a median of 4–6 ASMs. The effectiveness of CBD when initiated as an early line of therapy has not been well characterized. This retrospective cohort study evaluated polypharmacy burden and healthcare resource utilization (HCRU) as surrogate markers of effectiveness in new users of CBD and in a subset for whom CBD was prescribed as an early-line therapy (ie, first-, second-, or third-line ASM) using the US Optum® Market Clarity integrated electronic health record and claims database. Methods:
Patients with LGS, DS, or TSC who initiated CBD between June 25, 2018 and September 30, 2023 were included. The baseline period was defined as the 12 months before CBD initiation (index event), and follow-up was ≤ 12 months after initiation. An interrupted time series analysis assessed changes in polypharmacy burden (number of concomitant ASMs per patient per month [PPPM]) and HCRU (medical claims or records of physician office visits, seizure-related hospitalizations, or emergency department [ED] visits, PPPM) before and after CBD initiation. Reductions in polypharmacy burden and HCRU relative to the trend prior to CBD initiation (baseline period) are reported overall and for the subgroup of early-line CBD users (Figure). Results:
In total, 4561 new CBD users were identified, of whom 177 (4%) were early-line users. Compared with all new CBD users (median [first and third quartile] age 13.0 [7.0, 22.0] years), the early-line subgroup was younger (4.0 [1.0, 12.0] years), had a higher proportion of commercial insurance (53% and 46%), and lower proportion of comorbidities and use of other medications. After CBD initiation, trends in polypharmacy and seizure-related HCRU decreased over time relative to the pre-initiation trend in both the early-line subgroup and overall population (percent decrease from pre-initiation trend at 12 months: polypharmacy, 63% and 24%; physician office visits, 64% and 24%; hospitalizations, 79% and 57%; ED visits, 85% and 68%, respectively) (Table).Conclusions:
In new CBD users with LGS, DS, or TSC, we observed decreases in polypharmacy and HCRU compared with their trends before starting CBD. In the early-line CBD subgroup, further reductions in outcomes, particularly polypharmacy burden and physician office visits, were observed. These results suggest CBD is effective across all lines of therapy, regardless of when initiated. Although the small sample size and differences in baseline characteristics between early- and all-line groups may limit interpretation of these results, future studies with larger, balanced cohorts may confirm and build on these promising results.
Funding:
Jazz Pharmaceuticals, Inc.