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.