EFFECTIVENESS, TOLERABILITY, AND COST-EFFECTIVENESS OF ADJUNCT CANNABIDIOL THERAPY IN CHILDREN WITH DRUG-RESISTANT EPILEPSY: A MULTICENTRIC REAL-WORLD STUDY IN INDIA
Author(s)
Akanksha Singh, PhD1, Dipika Bansal, MBBS, MD, DM2, Jitendra Kumar Sahu, MBBS, MD, DM3;
1National Institute of Pharmaceutical Education & Research, SAS Nagar, PhD Scholar, Sas Nagar, India, 2National Institute of Pharmaceutical Education and Research, Mohali, India, 3PGIMER, Chandigarh, Pediatric Neurology, Chandigarh, India
1National Institute of Pharmaceutical Education & Research, SAS Nagar, PhD Scholar, Sas Nagar, India, 2National Institute of Pharmaceutical Education and Research, Mohali, India, 3PGIMER, Chandigarh, Pediatric Neurology, Chandigarh, India
OBJECTIVES: Objectives: To assess the real-world effectiveness and tolerability of cannabidiol (CBD) as an adjunct to usual care compared with usual care alone in children with drug-resistant epilepsy (DRE), and to evaluate the cost-effectiveness of add-on CBD for the treatment of patients aged ≥1 year with DRE in India.
METHODS: Methods: A multicentric ambispective observational study was conducted across four tertiary-care centers. Children receiving add-on CBD were compared with those receiving usual care alone. Effectiveness was assessed by ≥50% reduction in monthly seizure frequency at 3 months. Tolerability was evaluated through adverse events and treatment discontinuation. Cost-effectiveness was assessed from a societal perspective over 1 year using quality-adjusted life years (QALYs). Incremental cost-effectiveness ratios (ICERs) and one-way sensitivity analyses were performed.
RESULTS: Results At 3 months, 54.3% of patients in the CBD group achieved ≥50% seizure reduction compared with 26.0% in the usual care group (absolute difference: 28.3%). Mean monthly seizure frequency decreased by 46.8% in the CBD group versus 18.5% with usual care. CBD was generally well tolerated; 18.6% of patients reported mild-to-moderate adverse events (most commonly somnolence and decreased appetite), and 9.3% discontinued treatment due to adverse events. The mean total 6-month cost per patient was higher in the CBD group (USD 1,114) compared with usual care (USD 504). However, the CBD group accrued greater health benefits (0.312 vs 0.248 QALYs). The resulting ICER was USD 4,087 per QALY gained, which is below commonly cited willingness-to-pay thresholds for India. Results were robust across sensitivity analyses.
CONCLUSIONS: In a real-world Indian setting, adjunct CBD therapy demonstrated superior effectiveness and acceptable tolerability compared with usual care in children with DRE. Despite higher upfront treatment costs, CBD was found to be cost-effective.
METHODS: Methods: A multicentric ambispective observational study was conducted across four tertiary-care centers. Children receiving add-on CBD were compared with those receiving usual care alone. Effectiveness was assessed by ≥50% reduction in monthly seizure frequency at 3 months. Tolerability was evaluated through adverse events and treatment discontinuation. Cost-effectiveness was assessed from a societal perspective over 1 year using quality-adjusted life years (QALYs). Incremental cost-effectiveness ratios (ICERs) and one-way sensitivity analyses were performed.
RESULTS: Results At 3 months, 54.3% of patients in the CBD group achieved ≥50% seizure reduction compared with 26.0% in the usual care group (absolute difference: 28.3%). Mean monthly seizure frequency decreased by 46.8% in the CBD group versus 18.5% with usual care. CBD was generally well tolerated; 18.6% of patients reported mild-to-moderate adverse events (most commonly somnolence and decreased appetite), and 9.3% discontinued treatment due to adverse events. The mean total 6-month cost per patient was higher in the CBD group (USD 1,114) compared with usual care (USD 504). However, the CBD group accrued greater health benefits (0.312 vs 0.248 QALYs). The resulting ICER was USD 4,087 per QALY gained, which is below commonly cited willingness-to-pay thresholds for India. Results were robust across sensitivity analyses.
CONCLUSIONS: In a real-world Indian setting, adjunct CBD therapy demonstrated superior effectiveness and acceptable tolerability compared with usual care in children with DRE. Despite higher upfront treatment costs, CBD was found to be cost-effective.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
EE348
Topic
Economic Evaluation
Disease
SDC: Neurological Disorders