Cost-Effectiveness of Cannabidiol (CBD) for the Treatment of Seizures in Patients with Treatment-Resistant Lennox-Gastaut Syndrome or Dravet Syndrome in the Netherlands

Author(s)

Siddiqui J1, Bowditch S2
1FIECON Ltd, London, UK, 2Jazz Pharmaceuticals, Inc., London, LON, UK

OBJECTIVES: This study estimated the cost-effectiveness of cannabidiol (CBD) plus usual care compared with usual care alone for the treatment of seizures in patients with treatment-resistant Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS) in the Netherlands.

METHODS: A cohort-based Markov model with a Dutch societal perspective was developed, with health states based on seizure frequency and seizure-free days. Patients received plant-derived highly purified CBD medicine (Epidyolex®; 100 mg/mL oral solution) plus usual care (consisting of a variety of antiseizure medications including clobazam) or usual care alone. Population characteristics, clinical inputs, and utility values were sourced from the pivotal trials of CBD for each indication (LGS: NCT02224560, NCT02224690; DS: NCT02091375, NCT02224703; open-label extension: NCT02224573) and quality of life studies. Drug acquisition, disease management, adverse events, and societal costs were included, with unit costs sourced from published literature. A 2019/2020 price year was used, with costs presented in euros. The model used a mean dose of 12 mg/kg/day; it adopted a lifetime (90-year) horizon and a cycle length of 3 months. Discount rates of 4.0% and 1.5% per annum were applied to costs and outcomes, respectively. Uncertainty was explored through deterministic and probabilistic sensitivity analyses.

RESULTS: In patients with LGS, CBD plus usual care led to additional costs of €28338 and increased quality-adjusted life years (QALYs) of 1.318 compared with usual care alone. The resulting incremental cost-effectiveness ratio (ICER) of €21493/QALY in patients with LGS was in line with the relevant reference willingness-to-pay threshold (WTP) of €80000/QALY in the Netherlands. In patients with DS, CBD plus usual care dominated usual care alone, with cost savings of €23642 and increased QALYs of 0.868.

CONCLUSIONS: Treatment with CBD plus usual care is cost-effective compared with usual care alone in patients with treatment-resistant LGS or DS in the Netherlands.

Conference/Value in Health Info

2023-11, ISPOR Europe 2023, Copenhagen, Denmark

Value in Health, Volume 26, Issue 11, S2 (December 2023)

Code

EE400

Topic

Economic Evaluation

Topic Subcategory

Budget Impact Analysis, Cost-comparison, Effectiveness, Utility, Benefit Analysis, Trial-Based Economic Evaluation

Disease

Neurological Disorders, Pediatrics, Rare & Orphan Diseases

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