StiripentolDIACOMIT
Adjunctive therapy for severe myoclonic epilepsy in infancy (SMEI, also known as Dravet syndrome) with primary generalised clonic and tonic-clonic seizures in patients not adequately controlled by valproate and a benzodiazepine (usually clobazam).
Decision on record
- Meeting Mar 2020 Recommended Severe myoclonic epilepsy in infancy (SMEI) (also known as Dravet syndrome)
Access path
- TGA registered · DIACOMIT
TGA label narrower than the PBS population
- Mar 2020Recommended · restricted
vs placebo (plus valproate and clobazam)
- PBS listing · Authority Required
From the public summary
The PBAC recommended the Authority Required (Streamlined) listing of stiripentol (STP) for the treatment of patients with generalised tonic-clonic and clonic seizures associated with severe myoclonic epilepsy of infancy (SMEI, also known as Dravet syndrome) who are not adequately controlled by prior lines of therapy.PSD · Mar 2020
The PBAC was satisfied that STP provides, for some patients, an improvement in efficacy over the current standard of care, consisting of a range of other AEDs and treatments as adjuvant therapy to valproate (VAL) and a benzodiazepine. 21PSD · Mar 2020
The submission presented a stepped economic evaluation based on the direct randomised trials (STICLO-France and STICLO-Italy). The type of economic evaluation was a cost-utility analysis to estimate the incremental costs and benefits of STP (plus VAL and CLB), versus placebo (plus VAL and CLB). Given the proposed listing of STP may replace or displace other AEDs for SMEI including TOP and LEV, the ESC 12PSD · Mar 2020
Table 7 summarises the key component of the economic evaluation.PSD · Mar 2020
The submission described STP (as adjunctive treatment to VAL plus CLB) as superior in terms of efficacy but inferior in terms of safety compared to placebo (plus VAL and CLB), based on results in the STICLO trials.PSD · Mar 2020
The submission did not present any data or estimates with respect to this issue. With respect to safety, significantly more patients treated with STP (plus VAL and CLB) experienced AEs compared to placebo (plus VAL and CLB). AEs can be severe leading to withdrawals however appeared to be reversible upon dose adjustments of concomitant AEDs.PSD · Mar 2020
Cost-effectiveness
ICER values are redacted in the public summary document (marked as '***' in pricing table).
Decision context
PopulationChildren aged 3–18 years with severe myoclonic epilepsy in infancy (SMEI/Dravet syndrome) characterised by primary generalised clonic and tonic-clonic seizures, not adequately controlled by valproate (30 mg/kg/day or less) and clobazam (0.5 mg/kg/day, max. 20 mg/day).
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2020 | Recommended · restricted | placebo (plus valproate and clobazam) | — | RCT · responder_rate_50pct |
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to under age 18 at initiation and requires neurologist initiation; TGA label has no age or specialist requirement.