Abstract / Summary
Epilepsy is a common chronic neurological disorder with significant clinical and economic burden, especially in children. In Iran, challenges such as limited access to new antiseizure medicines and high treatment costs highlight the need for cost-effective options. While carbamazepine is affordable, its side effects limit its use. Lacosamide offers better tolerability but is more expensive. This study compares the clinical outcomes and cost-effectiveness of carbamazepine and lacosamide in paediatric epilepsy. This randomized prospective cohort study compared the efficacy and cost-effectiveness of carbamazepine and lacosamide in children with epilepsy. The patients at Namazi Hospital and its outpatient clinics, Shiraz, were randomized to receive either drug and followed up for 6 months. Efficacy was determined by reduction in seizures per frequency and quality of life using the EQ-5D-3L questionnaire. Adverse effects were recorded using the Liverpool Adverse Events Profile. Cost data were extracted from patient interviews and medical records. Cost-effectiveness was evaluated using a decision-tree model with a 6-month time horizon in TreeAge Pro. Both the incremental cost-effectiveness ratio (ICER), expressed as the cost per unit increase in the expected clinical effectiveness score, and the incremental cost-utility ratio (ICUR), expressed as the cost per QALY gained, were estimated. Lacosamide treatment produced a small increase in health utility (QALY) from 0.41 to 0.42 and in the expected clinical effectiveness score, derived from the weighted treatment response categories in the decision-tree model, from 0.60 to 0.65, compared with carbamazepine, at an additional cost of $36 (total cost: $617 vs. $581). The ICUR was $5,220 per QALY gained, and the ICER was $720 per unit increase in the expected clinical effectiveness score. Probabilistic sensitivity analysis indicated that lacosamide was more effective but also more costly than carbamazepine. At a willingness-to-pay threshold of $15,331 per QALY, lacosamide had an 80% probability of being cost-effective. The 95% confidence intervals derived from the probabilistic sensitivity analysis ranged from $10 to $65 for the incremental cost and from 0.005 to 0.025 QALYs for the incremental utility, corresponding to an ICER range of $400 to $13,000 per QALY. Although lacosamide is expensive, its modest margins in efficacy and quality of life make it a cost-effective alternative, especially for children who are not responsive to first-line treatments. The results enable evidence-based decision-making in the treatment of paediatric epilepsy.