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    • Has all of the relevant evidence been taken into account?
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The content on this page is not current guidance and is only for the purposes of the consultation process.

1 Recommendations

1.1 Ganaxolone is not recommended, within its anticipated marketing authorisation, as an add-on treatment option for seizures caused by cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in children and young people aged 2 to 17 years and adults who turn 18 while on treatment.

1.2 This recommendation is not intended to affect treatment with ganaxolone that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop. For children and young people, this decision should be made jointly by the clinician and the child or young person, or their parents or carers.

Why the committee made these recommendations

Usual care for seizures caused by CDD includes antiseizure medications. There is no specific treatment for controlling seizures caused by CDD, so people often try several antiseizure medications and add-on treatments.

Clinical trial evidence suggests that ganaxolone plus usual care reduces seizure frequency compared with placebo plus usual care. But there are uncertainties in how well it works in the long term.

There are uncertainties in the economic model, including:

  • its reflection of CDD

  • the assumptions used to model quality of life

  • how ganaxolone affects seizure frequency and quality of life

  • if someone stops having ganaxolone, how this is modelled and how well it reflects clinical practice.

The cost-effectiveness estimates for ganaxolone are uncertain. Even when considering difficulties in collecting evidence for this rare condition, the severity of the condition, and potential benefits of ganaxolone not included in the economic model, the most likely cost-effectiveness estimates are above what NICE normally considers an acceptable use of NHS resources. So, ganaxolone is not recommended.