COST-UTILITY ANALYSIS OF ADD-ON CANNABIDIOL FOR THE TREATMENT OF THERAPY-RESISTANT SEIZURES ASSOCIATED WITH TUBEROUS SCLEROSIS COMPLEX IN SWEDEN
Author(s)
Anathea Cora Cristea, MSc.1, Anna Grootendorst, BSc, MSc1, Miranda Harrison, BSc2, Nils-Peter Ipland, MSc3, Timothy John Mann, MSc2, Jamshaed Siddiqui, MMath2.
1NHTA, Stockholm, Sweden, 2Jazz Pharmaceuticals, UK Ltd., London, United Kingdom, 3Jazz Pharmaceuticals Denmark Aps., Copenhagen, Denmark.
1NHTA, Stockholm, Sweden, 2Jazz Pharmaceuticals, UK Ltd., London, United Kingdom, 3Jazz Pharmaceuticals Denmark Aps., Copenhagen, Denmark.
OBJECTIVES: This study aimed to evaluate the cost-effectiveness of add-on cannabidiol (CBD; Epidyolex®, 100 mg/mL oral solution) for the treatment of therapy-resistant seizures associated with tuberous sclerosis complex (TSC) in patients aged ≥2 years in Sweden.
METHODS: A cohort-based Markov model with a Swedish healthcare payer perspective was developed using a 50-year time horizon and a discount rate of 3% per year for both costs and effects; an average CBD maintenance dose of 12 mg/kg/day was assumed. Health states were based on seizure frequency and seizure-free days informed by GWPCARE6 trial (NCT02544763) data of CBD in patients with TSC-associated seizures. A regression-based approach was selected to predict cohort-level seizure frequency and seizure-free days. Healthcare utilisation data were sourced from a Delphi panel, and health-related quality of life values were derived from a vignette study. Costs and resource use were parameterised using Swedish DRG-tariffs, publicly available pharmaceutical prices, and other published literature; CBD pharmacy purchase price was SEK 10,002 (2023 Swedish Dental and Pharmaceutical Benefits Agency report). Multiple one-way sensitivity analyses, a probabilistic sensitivity analysis based on 5,000 simulations, and multiple scenario analyses (e.g., number of carers, time horizon) were performed.
RESULTS: In the discounted base case, patients with TSC receiving CBD plus usual care (i.e., antiseizure medicines) had additional costs of SEK 403,560 and increased quality-adjusted life years (QALY) of 0.48 compared with usual care alone over the 50-year time horizon. The resulting incremental cost-effectiveness ratio was SEK 835,251 per QALY, in line with the unofficial willingness-to-pay (WTP) threshold of SEK 1,000,000 in Sweden. Results were robust to sensitivity and scenario analyses.
CONCLUSIONS: At an average dose of 12 mg/kg/day and a WTP threshold of SEK 1,000,000, add-on CBD treatment is cost-effective when compared with usual care alone for patients with TSC-associated seizures aged ≥2 years who are refractory to current treatment in Sweden.
METHODS: A cohort-based Markov model with a Swedish healthcare payer perspective was developed using a 50-year time horizon and a discount rate of 3% per year for both costs and effects; an average CBD maintenance dose of 12 mg/kg/day was assumed. Health states were based on seizure frequency and seizure-free days informed by GWPCARE6 trial (NCT02544763) data of CBD in patients with TSC-associated seizures. A regression-based approach was selected to predict cohort-level seizure frequency and seizure-free days. Healthcare utilisation data were sourced from a Delphi panel, and health-related quality of life values were derived from a vignette study. Costs and resource use were parameterised using Swedish DRG-tariffs, publicly available pharmaceutical prices, and other published literature; CBD pharmacy purchase price was SEK 10,002 (2023 Swedish Dental and Pharmaceutical Benefits Agency report). Multiple one-way sensitivity analyses, a probabilistic sensitivity analysis based on 5,000 simulations, and multiple scenario analyses (e.g., number of carers, time horizon) were performed.
RESULTS: In the discounted base case, patients with TSC receiving CBD plus usual care (i.e., antiseizure medicines) had additional costs of SEK 403,560 and increased quality-adjusted life years (QALY) of 0.48 compared with usual care alone over the 50-year time horizon. The resulting incremental cost-effectiveness ratio was SEK 835,251 per QALY, in line with the unofficial willingness-to-pay (WTP) threshold of SEK 1,000,000 in Sweden. Results were robust to sensitivity and scenario analyses.
CONCLUSIONS: At an average dose of 12 mg/kg/day and a WTP threshold of SEK 1,000,000, add-on CBD treatment is cost-effective when compared with usual care alone for patients with TSC-associated seizures aged ≥2 years who are refractory to current treatment in Sweden.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE358
Topic
Economic Evaluation, Health Technology Assessment
Disease
Neurological Disorders, No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)