Cost-effectiveness of perampanel as an adjunctive treatment for uncontrolled focal seizures in pediatric patients: a Chinese perspective.

Background: The incidence of epilepsy is 41-187 per 100,000 person-year in children. The health care costs for children with uncontrolled epilepsy is a huge burden. Perampanel (PER) was effective, safety and well-tolerated as add-on therapy in pediatric patients aged 4 to <12 years with uncontrolled focal seizures. However, there is still limited evidence on cost-effectiveness of PER in pediatric patients. We aimed to evaluate the cost-effectiveness of PER as an add-on therapy for pediatric patients with uncontrolled focal seizures. Methods: A Markov model was established to conduct an analysis from the perspective of the Chinese health system and society. The incremental cost-effectiveness ratio (ICER) of patients using PER and conventional therapy versus patients using conventional therapy alone were estimated and compared. The transition probability of the response level, health state utility values, and costs were derived from clinical trials and the literature. Costs, including medical, drug, transportation and indirect costs, were calculated. We performed 1-way sensitivity analyses and probabilistic sensitivity analyses. A subgroup analysis of different ages was also conducted. Results: The base-case analysis indicated that compared to maintaining conventional therapy, adding PER as an adjuvant drug therapy had an increased cost of $3,449.85 over 5 years, with an incremental quality-adjusted life years (QALY) value of 0.40, resulting in an ICER of $8,582.58 per additional QALY. The health state utility value had the greatest effect on the ICER. The probabilistic sensitivity analyses showed that the probability of PER being cost-effective was 76.72% at a willingness-to-pay of $11,293/QALY. The ICER of the subgroup ranged from $7,167.95/QALY to $19,710.96/QALY. Conclusions: Our study demonstrated that PER is a cost-effective add-on therapy for pediatric patients.