Abstracts

Dravet syndrome is one of the most severe and medically intractable forms of childhood epilepsy. Stiripentol (STP) has been shown to have a unique efficacy in this syndrome. The manufacturer of STP had provided this medication as [quot]compassionate release[quot], however effective February 2004, families have been required to cover the costs for this medication. Third party insurance will not cover these costs, as this medication is available only through the Special Access Programme of Health Canada. This study compares the medical costs in children with Dravet syndrome, pre and post STP initiation. The medical costs of all four children (aged 18 mos to 11 yrs 11 mo) with Dravet syndrome followed in the Refractory Epilepsy clinic at the Alberta Childrens Hospital who were treated with STP were assessed over three time periods:
1. Pre-treatment: the year preceding STP initiation (or in children commencing STP prior to 1 year after seizure onset, the time from seizure onset to STP initiation)
2. Titration: the month following initiation during which titration to therapeutic dose was achieved
3. Post-treatment: the first year following the Titration period (or in children who have not yet been treated for a one year period, the total duration of follow-up available).
Costs assessed included hospitalization and clinic fees, ER visits, ambulance/EMS costs and physician billings, but medication costs were excluded. Families also completed a QOLCE for the pre and post-treatment periods.
The mean monthly medical costs for the Pre-treatment and Post-treatment periods were calculated for each patient. All 4 children showed a significant reduction in seizure frequency on STP (with benzodiazepine). Three of the four children have been treated with STP for at least one year, while the fourth has only completed 2 months of the Post-treatment period to date. The average monthly medical cost was lower post-treatment (mean of $93, range $40-160) compared to pre-treatment (mean of $2573, range $215-8415). Cost reduction was most dramatic in the 3 younger patients (ages 18 mos - 4 yrs 2 mos at initiation, range $516-8375 per month) than in the older child (11 yrs 11 mos at initiation, $55 per month). QOLCE markedly improved in 3 of 4 cases. The savings in medical costs by far exceeded the actual cost of STP (mean $135/mo, range $75-300). STP initiation was associated with a reduction in medical costs for all 4 children with Dravet syndrome, but the reduction appeared most significant for younger patients. This medication is clearly cost-effective and should be initiated early on after the clinical diagnosis of Dravet syndrome is made. In a publically funded health-care system, funding should be obtained for families who are financially unable to afford STP.