Lennox-Gastaut syndrome (LGS) is a catastrophic childhood form of
epilepsy. The syndrome is characterized by mental impairment, frequent
seizures of multiple types that are particularly resistant to treatment, and high rates of seizure-related injury. With the introduction of newer, but more costly,
antiepileptic drugs (AEDs), it is important that decision makers are able to assess their value in the management of this rare and difficult-to-treat condition. To evaluate the cost effectiveness, from the UK NHS perspective, of
rufinamide in patients with LGS. An individual patient-simulation model was developed to estimate the total treatment-related costs and clinical benefits of
rufinamide compared with
topiramate and
lamotrigine over a 3-year time horizon. The model examines the treatment scenarios of adding
rufinamide,
lamotrigine or
topiramate to older AEDs (standard
therapy), or standard
therapy alone within a primary-care or community setting. Three placebo-controlled clinical trials of adjunctive AED treatment for children with LGS were analysed. There are no head-to-head comparator studies. Between 98 and 139 patients were randomized in each study and the mean age in each study was 10, 11 and 14 years. A mixed-treatment comparison using a random-effects model was carried out on the number of patients in each response category, using the placebo arms of the respective trials. The primary outcome measure was the percentage of successfully treated patients, defined as >50% reduction in the frequency of total
seizures and
drop attacks. The hypothesis being tested was formulated after data collection. Costs ( pound, year 2006/07 values) of patient monitoring, switching treatments, hospitalization due to seizure, treatment of adverse effects, and personal and social services were included in the analysis. Results of 10,000 Monte Carlo simulations were bootstrapped to conduct probabilistic sensitivity analysis. Over 3 years, adjunctive
rufinamide resulted in higher total costs than
topiramate and
lamotrigine; however, with more patients being treated successfully, this leads to acceptable incremental cost-effectiveness ratios. If society is prepared to pay at least 250 pounds for a 1% increase in the number of successfully treated LGS patients, in terms of a 50% reduction in the frequency of
drop attacks, the probability of the treatment with
rufinamide being cost effective is >80%. This cost-effectiveness analysis suggests that
rufinamide results in more LGS patients being treated successfully at a reasonable cost from a UK NHS perspective.