Abstract

Purpose To estimate the cost-effectiveness of rufinamide relative to topiramate and lamotrigine as adjunctive treatment for children with Lennox–Gastaut Syndrome (LGS). Methods A Markov decision analytic model was developed to estimate the incremental cost-effectiveness ratio over a three-year time horizon in patients with LGS uncontrolled by up to three antiepileptic drugs. Utilities were assigned to health states, defined according to a patient's response to treatment (≥75%, ≥50% and <75%, and <50% reduction in tonic–atonic [drop attack] seizure frequency and death). Efficacy and safety estimates were made using indirect/mixed-treatment comparisons of data obtained from published literature. Outcomes included costs and quality-adjusted life-years (QALYs), allowing the incremental cost-effectiveness ratio to be estimated as cost per QALY gained. Results Over three years, the total cumulative costs for rufinamide, topiramate, and lamotrigine were £24,992, £23,360, and £21,783, respectively. Rufinamide resulted in an incremental QALY gain of 0.079 relative to topiramate and 0.021 relative to lamotrigine. The incremental costs of rufinamide were £1632 and £3209, relative to topiramate and lamotrigine, resulting in an incremental cost per QALY gained of £20,538 and £154,831, respectively. Conclusions Considering the underlying assumptions, this current economic evaluation demonstrates that rufinamide is likely to be a cost-effective alternative to topiramate as adjunctive treatment for children with LGS in the UK. In addition, when compared to lamotrigine, which is an inexpensive treatment, rufinamide should be considered as a cost-effective alternative due to the importance of patient choice and equity of access in such a rare and devastating condition.

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