Rufinamide (Inovelon®), a
triazole derivative, is an oral
antiepileptic drug approved in the EU as adjunctive
therapy in the treatment of
seizures associated with
Lennox-Gastaut syndrome (LGS) in patients aged ≥4 years. The efficacy of oral
rufinamide as adjunctive
therapy in patients with LGS uncontrolled on one to three concomitant
antiepileptic drugs was demonstrated in a pivotal, 12-week, randomized, double-blind trial.
Rufinamide significantly reduced the 28-day frequency of both
drop attacks and total
seizures compared with placebo, and significantly increased the proportions of patients experiencing a ≥50% reduction in each seizure frequency. A significantly higher proportion of
rufinamide than placebo recipients recorded an improvement in seizure severity at the end of treatment. Reductions in the frequency of
drop attacks and total
seizures were maintained in a long-term (up to 3 years), open-label extension study. Oral
rufinamide was generally well tolerated in patients with LGS.
Somnolence and
vomiting were the most common adverse events occurring more frequently with
rufinamide than with placebo. Two pharmacoeconomic analyses, using decision-analysis models with 3-month cycles over a time horizon of 3 years, assessed the cost effectiveness and cost utility, respectively, of
rufinamide compared with
topiramate and
lamotrigine as adjunctive
therapy in patients with LGS from the perspective of the UK NHS. The cost-effectiveness analysis suggested that
rufinamide would be associated with incremental costs of £62 (
drop attacks) or £2151 (total
seizures) per 1% increase in the number of patients achieving a >50% reduction in seizure frequency over 3 years. The cost-utility analysis predicted that the incremental cost per QALY gained for
rufinamide compared with the next less-costly and undominated
therapy would be more than 5-fold higher than the commonly accepted willingness-to-pay threshold range in the UK. In conclusion, the available pharmacoeconomic data indicate that
rufinamide is more effective, but more expensive, than alternative adjunctive
therapies approved for use in patients with LGS in the UK.
Rufinamide would appear to be a cost-effective alternative to
topiramate. Although
rufinamide exceeds conventional cost-effectiveness thresholds when compared with
lamotrigine, it may still be considered a valuable treatment option for a devastating
orphan disease such as LGS.