UK Dravet Syndrome HTA Strategy Model

NICE recommended both existing Dravet syndrome therapies, cannabidiol (TA614, 2019) and fenfluramine (TA808, 2022), through the standard Technology Appraisal process rather than NHS England's Highly Specialised Technology route reserved for ultra-rare conditions. That routing choice matters more for a new entrant's submission strategy than it does for Dravet's own regulatory history: it fixes the cost-effectiveness bar at NICE's ordinary £20,000-30,000 per QALY, not the £100,000-300,000 per QALY threshold an HSS-designated technology would face. A new Dravet therapy's WAC and Patient Access Scheme have to be modelled against that lower bar from the outset, using a target WAC of £30,000-50,000 per year discounted through a 30-40% PAS to an effective NHS price of £18,000-35,000.

Two stakeholder engagements need to start well before the NICE dossier is filed. The British Paediatric Neurology Association runs a Dravet working group whose clinical guidance feeds directly into NICE's evidence review, and engaging that group 18-24 months ahead of submission is the recommended lead time for shaping how the committee reads the clinical evidence. Dravet UK, the patient charity, should be approached separately: sponsoring its patient-experience survey and NICE patient-group submission 12-18 months pre-filing, at an estimated budget of £20,000-40,000, gives the dossier a patient-voice component NICE weighs independently of the clinical data. Both engagements need to characterise the refractory population a new therapy actually targets, not the broad Dravet cohort already served by cannabidiol and fenfluramine.

NICE's epilepsy guideline CG137 mandates SUDEP risk counselling as part of routine Dravet management, and a submission that builds that counselling requirement into its value dossier, rather than treating it as a safety footnote, strengthens the clinical-benefit case alongside seizure-reduction data. The clock on all of this is set by soticlestat (Takeda/Ovid): positive Phase 3 ELEKTRA data and an expected NICE technology appraisal in 2026-2027 make it the benchmark a new entrant's own submission timeline should track, not a competitor to research once filing is already underway.

Every AXLRx HTA strategy model is built from primary sources: NICE technology appraisal and committee discussion documents, patient-charity and clinical-body guidance, and clinical-trial registration data, not secondary summaries. Every stakeholder-engagement lead time and cost figure is pressure-tested before being accepted into the model.

UK Dravet syndrome HTA strategy sources: NICE TA614 (cannabidiol) and TA808 (fenfluramine) Final Appraisal Determinations; British Paediatric Neurology Association Dravet working-group guidance; Dravet UK annual report 2023; NICE CG137 epilepsy guideline; ELEKTRA (soticlestat) ClinicalTrials.gov registration.