A new Dravet entrant must beat an entrenched three-drug NHS sequence, not just clear NICE's cost bar.

NHS England Highly Specialised Services commission Dravet Syndrome management at 25 designated paediatric neurology centres, covering an estimated 400-500 UK patients. NICE has recommended two agents onto the standard pathway: cannabidiol (Epidiolex, NICE TA614, 2019) and fenfluramine (Fintepla, NICE TA808, 2022), both with confidential Patient Access Schemes. Stiripentol (Diacomit), the oldest Dravet-specific agent, has never gone through a dedicated NICE technology appraisal; it is covered under NICE clinical guideline CG137 and still anchors the backbone regimen alongside valproate and clobazam in many centres.

The NHS treatment algorithm is now fixed: stiripentol-based backbone first, cannabidiol added if seizures persist, fenfluramine added if cannabidiol response is inadequate. NHS annual cost per patient is approximately £25,000-35,000 on cannabidiol and £40,000-60,000 on fenfluramine, both post-PAS, the latter including mandatory cardiac monitoring under the Fintepla Cardiac Monitoring Scheme. A new therapy entering this space faces a structural barrier: it must demonstrate superiority or a materially better tolerability profile to dislodge an already NICE-recommended, PAS-priced two-drug sequence, not simply achieve its own positive appraisal.

The algorithm is fixed; a challenger must prove superiority, not just cost-effectiveness.

Five questions this report answers:

Q1 - What clinical evidence would justify a new NICE appraisal ahead of fenfluramine in the current sequence?

Q2 - How does NHS Highly Specialised Services commissioning gate access at the 25 designated centres?

Q3 - What role does SCN1A genomic testing play in Dravet diagnosis and trial recruitment?

Q4 - How do fenfluramine's £40,000-60,000 and cannabidiol's £25,000-35,000 annual NHS costs compare?

Q5 - Why has stiripentol never gone through a dedicated NICE technology appraisal?

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Mike || Global Pharma Commercial Marketing Head

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