A Cost-Utility Analysis of Add-On Cannabidiol Versus Usual Care Alone for the Treatment of Seizures Associated with Tuberous Sclerosis Complex in England and Wales.

Autor: Burke C; Lumanity, Inc., Great Suffolk Yard, 121-131 Great Suffolk Street, London, SE1 1PP, UK. Colin.Burke@lumanity.com., Crossan C; Lumanity, Inc., Great Suffolk Yard, 121-131 Great Suffolk Street, London, SE1 1PP, UK., Tyas E; Lumanity, Inc., Great Suffolk Yard, 121-131 Great Suffolk Street, London, SE1 1PP, UK., Hemstock M; Lumanity, Inc., Great Suffolk Yard, 121-131 Great Suffolk Street, London, SE1 1PP, UK., Lee D; Lumanity, Inc., Great Suffolk Yard, 121-131 Great Suffolk Street, London, SE1 1PP, UK., Bowditch S; Jazz Pharmaceuticals, Inc., 1 Cavendish Place, Marylebone, London, W1G 0QF, UK.
Jazyk: angličtina
Zdroj: PharmacoEconomics - open [Pharmacoecon Open] 2024 Jul; Vol. 8 (4), pp. 611-626. Date of Electronic Publication: 2024 Mar 05.
DOI: 10.1007/s41669-024-00474-x
Abstrakt: Objectives: The aim of this study was to evaluate the cost effectiveness of plant-derived highly purified cannabidiol (Epidyolex ® in the UK; 100 mg/mL oral solution) as an add-on treatment to usual care for the management of treatment-refractory seizures associated with tuberous sclerosis complex (TSC) in patients aged ≥ 2 years.
Methods: A cohort-based model was developed using a National Health Service perspective and lifetime horizon. Health states were based on weekly seizure frequency and seizure-free days, utilizing patient-level data from the GWPCARE6 trial (ClinicalTrials.gov identifier: NCT02544763). Two independent regression models were applied to individual patient-level data to predict seizure-free days and seizure frequency. Healthcare resource utilization data were sourced from a Delphi panel, and patient and caregiver health-related quality of life values were elicited using vignettes valued by the general public. Outcomes relating to TSC-associated neuropsychiatric disorders were modeled with costs and quality-adjusted life-years sourced from published literature.
Results: In the base case, compared with usual care alone, 12 mg/kg/day cannabidiol was associated with an incremental cost-effectiveness ratio (ICER) of £23,797. The National Institute for Health and Care Excellence disease severity modifier reduced the ICER to £19,831. Probabilities of cost effectiveness at willingness-to-pay thresholds of £20,000 and £30,000 were 30% and 52%, respectively, for the base case and 39% and 66%, respectively, for the disease severity modifier. Results were robust to sensitivity and scenario analyses.
Conclusions: At 12 mg/kg/day and an ICER threshold of £20,000-£30,000, we provide evidence for the cost effectiveness of add-on cannabidiol treatment for patients with TSC-associated seizures aged ≥ 2 years who are refractory to current treatment.
(© 2024. The Author(s).)
Databáze: MEDLINE
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