REAL-WORLD EFFECTIVENESS, SAFETY, AND ECONOMIC EVALUATION OF ADJUNCT CANNABIDIOL IN PAEDIATRIC DRUG-RESISTANT EPILEPSY: EVIDENCE FROM A MULTICENTRIC INDIAN COHORT...
Author(s)
Akanksha Singh, MPH, PhD1, Jitendra Kumar Sahu, MBBS, MD, DM2, Dipika Bansal, MD3.
1PhD Scholar, National Institute of Pharmaceutical Education & Research, SAS Nagar, Sas Nagar, India, 2PGIMER, Chandigarh, India, 3National Institute of Pharmaceutical Education and Research, Mohali, India.
1PhD Scholar, National Institute of Pharmaceutical Education & Research, SAS Nagar, Sas Nagar, India, 2PGIMER, Chandigarh, India, 3National Institute of Pharmaceutical Education and Research, Mohali, India.
OBJECTIVES: Given the approval of CBD (CDSCO, February 2023) for Lennox-Gastaut syndrome, Dravet syndrome, and tuberous sclerosis complex in patients aged ≥1 year, and the limited real-world evidence from low- and middle-income countries. This study aimed to evaluate the real-world effectiveness, tolerability, and cost-effectiveness of adjunct cannabidiol (CBD) therapy in children with drug-resistant epilepsy (DRE) across six tertiary care centres in India.
METHODS: This prospective observational study was conducted across six tertiary care centres in India and enrolled 325 participants aged ≥1 year, including 220 patients in the adjunct cannabidiol (CBD) group and 105 patients in the usual care group. Data on seizure frequency, demographic characteristics, anti-seizure medication status, and treatment costs were collected between August 2024 and December 2026, with a structured follow-up period of 3 months. Economic evaluation was performed from a societal perspective using a decision tree model. Additionally, one-way sensitivity analyses were conducted to assess the impact of individual variables on the cost-effectiveness (CE) model, and the results were illustrated using a Tornado diagram.
RESULTS: CBD significantly reduced focal motor, generalized motor, focal non-motor, and generalized non-motor seizure frequencies compared to baseline and usual care (p<0.001). Approximately 53% of CBD recipients achieved ≥50% seizure reduction, with 6.4% attaining complete seizure freedom. Somnolence was the most frequently reported adverse event (24.4%); no SUDEP or hepatotoxicity was recorded. Incremental cost-effectiveness analysis revealed an additional cost of INR 3,298 yielding an 81.4% incremental seizure reduction over usual care.
CONCLUSIONS: Adjunct cannabidiol demonstrated significant seizure reduction compared to usual care in children with drug-resistant epilepsy; however, it was associated with a higher rate of adverse events, predominantly mild-to-moderate in nature.
METHODS: This prospective observational study was conducted across six tertiary care centres in India and enrolled 325 participants aged ≥1 year, including 220 patients in the adjunct cannabidiol (CBD) group and 105 patients in the usual care group. Data on seizure frequency, demographic characteristics, anti-seizure medication status, and treatment costs were collected between August 2024 and December 2026, with a structured follow-up period of 3 months. Economic evaluation was performed from a societal perspective using a decision tree model. Additionally, one-way sensitivity analyses were conducted to assess the impact of individual variables on the cost-effectiveness (CE) model, and the results were illustrated using a Tornado diagram.
RESULTS: CBD significantly reduced focal motor, generalized motor, focal non-motor, and generalized non-motor seizure frequencies compared to baseline and usual care (p<0.001). Approximately 53% of CBD recipients achieved ≥50% seizure reduction, with 6.4% attaining complete seizure freedom. Somnolence was the most frequently reported adverse event (24.4%); no SUDEP or hepatotoxicity was recorded. Incremental cost-effectiveness analysis revealed an additional cost of INR 3,298 yielding an 81.4% incremental seizure reduction over usual care.
CONCLUSIONS: Adjunct cannabidiol demonstrated significant seizure reduction compared to usual care in children with drug-resistant epilepsy; however, it was associated with a higher rate of adverse events, predominantly mild-to-moderate in nature.
Conference/Value in Health Info
2026-09, ISPOR Asia Pacific 2026, Bangkok, Thailand
Value in Health, Volume 55, Issue S1
Code
HTA3
Topic
Health Technology Assessment
Disease
SDC: Neurological Disorders, SDC: Pediatrics