SUDEP Action welcomes NICE Appeal decision on new anti-seizure medication
Hope renewed for use of anti-seizure medication for patients with Lennox-Gastaut Syndrome


Appeal against decision not to approve use of Fenfluramine upheld
The National Institute for Health Care and Excellence (NICE) has upheld an appeal against an earlier decision not to approve the use of Fenfluramine for the treatment of seizures associated with Lennox-Gastaut Syndrome.
Lennox-Gastaut Syndrome is a complex, rare and severe form of childhood-onset epilepsy in which seizures are often resistant to medication.
The Appeal Panel met virtually on 6 September to consider the appeals lodged by the Royal College of Physicians, Tuberous Sclerosis Association and UCB against final draft guidance. SUDEP Action collaborated with the appeal, along with UK Rare Epilepsies Together (UKRET) network to ensure patients and families had a strong voice. SUDEP Action’s work on the independent review into the death of Clive Treacey was one of the examples shared to show the severe harms to people and costs to the NHS when adequate investment is not made in risk prevention.
At the hearing, Jane Hanna OBE, SUDEP Action Director of Policy and Influencing, told the NICE committee that Fenfluramine could be a lifeline for some families without hope – offering up to a 50% risk reduction in seizures. The committee also heard about the experiences of the mother of a child with LGS who suffered generalized tonic seizures 4-7 times a week.
The mother said: “No one can truly understand the constant, gnawing worry of SUDEP. We’ve tried everything. Fenfluramine gives us a chance of an improved quality of life for the whole family.”
The panel upheld the appeal on a number of grounds. Critically, they found ‘standard of Care medication alone does not reflect NHS clinical practice and is not an appropriate comparator for fenfluramine.’ It was therefore unreasonable for the committee to consider standard of care alone as a comparator for fenfluramine.
Jane Hanna said: “We made the case for a flexible approach recognising the value of Fenfluramine as part of a highly individualised, person-centred treatment. There are wider benefits too, from reducing demands on the NHS and supporting unpaid carers. We hope now that after reasonable steps are taken to review the evidence, this medication will be approved.”
Dr Rhys Thomas, Consultant Neurologist, who presented to the hearing, said: “NICE is a well-respected institution that ensures drugs with the best evidence base are prescribable, at a price that makes sense to the UK taxpayer. Following enthusiastic support from epilepsy support groups and clinicians from the Royal College of Physicians, NICE has decided to consider again the evidence regarding the use of fenfluramine for treating seizures associated with a rare and severe childhood-onset epilepsy, Lennox-Gastaut syndrome. On a personal level I am indebted to SUDEP Action, The Tuberous Sclerosis Association and UKRET for their attention to detail and passionate unwavering support for people with significant unmet needs.”