Cost-Effectiveness of Cannabidiol Add-on Therapy Versus Placebo for the Treatment of Seizures in Tuberous Sclerosis Complex


Bowditch S1, Burke C2, Crossan C2, Hemstock M2, Tyas E2, Lee D2
1Jazz Pharmaceuticals, Inc., London, LON, UK, 2Lumanity, Sheffield, UK

OBJECTIVES: Tuberous sclerosis complex (TSC) is a rare disorder with substantial unmet need for treatment providing early, effective seizure control with an acceptable safety profile. This analysis evaluated the cost-effectiveness of add-on cannabidiol (Epidyolex®; 100 mg/mL oral solution), versus placebo, in patients aged ≥2 years with treatment-refractory TSC-associated seizures managed with usual care.

METHODS: A cohort-level cost-effectiveness model with a lifetime horizon was constructed from the National Health Service (NHS) perspective in England. The model considers the impact of treatment on focal seizures, with impairment of consciousness/awareness, and generalised seizures. Health states were based on weekly seizure frequency and seizure-free days, with health state distributions derived using regression models utilising patient-level data from the GWPCARE6 trial (GWP42003-P; NCT02544763). Base-case analysis parameters included annual healthcare resource use, effect of treatment on development of TSC-associated neuropsychiatric disorders (both informed by Delphi studies of UK clinical experts and published literature), patient and caregiver utilities (estimated from a vignette study of the general population valued using time trade-off methodology), and TSC-related excess mortality rates, including sudden unexpected death in epilepsy (from published literature). Cannabidiol discontinuation and stopping rates were calculated from GWPCARE6 and the subsequent open-label extension (NCT02544750). The impact of the new National Institute for Health and Care Excellence (NICE) disease severity modifier was also examined.

RESULTS: Compared with placebo, the incremental cost-effectiveness ratio (ICER) per quality-adjusted life year of cannabidiol was £12876 and, following application of the disease severity modifier, £10730. Results were robust to sensitivity and scenario analyses. The most influential scenario was the inclusion of social and educational care resource use, which resulted in a dominant ICER. Parametric sensitivity analysis showed the ICER was most sensitive to the stopping rule assessment rate at 6 months.

CONCLUSIONS: Despite conservative assumptions, cannabidiol is a cost-effective treatment option for TSC-associated seizures compared with placebo.

Conference/Value in Health Info

2022-11, ISPOR Europe 2022, Vienna, Austria

Value in Health, Volume 25, Issue 12S (December 2022)




Economic Evaluation

Topic Subcategory

Cost-comparison, Effectiveness, Utility, Benefit Analysis


SDC: Neurological Disorders

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