The Scottish Medicines Consortium (SMC) has announced today [Monday, 11 June] that everolimus will be available on prescription to treat refractory epilepsy in people aged two years and older with Tuberous Sclerosis Complex (TSC) in Scotland.
Maxine Smeaton, Chief Executive of the Tuberous Sclerosis Association (TSA), the only UK charity that supports people affected by TSC, said: “We are absolutely delighted with today’s announcement.  This decision will have a tremendous impact on the physical and mental health of people living with TSC-related refractory epilepsy in Scotland, as well as improving the quality of life for their families and carers.”

TSC is a genetic condition that can lead to growths in various organs of the body, and it is estimated to affect 10,000 people across the UK. These growths may be referred to as benign tumours but they are not cancerous: when they cause problems it is mainly because of their size and where they are in the body.  The organs most commonly affected are the brain, eyes, heart, kidney, skin and lungs. 

Epilepsy is the most common neurological feature of TSC, affecting more than eight out of ten people living with the condition.  More than 50 per cent of people with TSC who have epilepsy will not respond to standard anti-epilepsy medicines and may need any alternative form of treatment.

Maxine added: “We are grateful to SMC for providing the TSA with the opportunity to speak directly to the decision-making panel along with our patient representative, Craig Ireland.  His first hand experience was key to helping the SMC make an informed decision about the benefits of taking everolimus for TSC-related refactory epilepsy.”  Father-of-one Craig, from Falkirk, represented Scotland’s TSC community at an SMC hearing in Glasgow last month.
Dr Chris Kingswood, TSA Trustee stated: “This is fantastic news and a game-changing landmark in the treatment of TSC. The TSA will now develop a plan to support the NHS to ensure all patients who need everolimus, have access and can be safely monitored.”  Dr Kingswood is a consultant nephrologist who chairs the TSC’s research committee.

Jill Booth, Area Drug and Therapeutics Committees Clinical Adviser for the Scottish Medicines Consortium, said: “We would like to thank the TSA for the time and effort taken to produce your submission and also for your participation in the PACE process. Capturing the views and experiences of patients and carers is an important part of the SMC process and plays a key role in helping our committee reach its decisions.  We hope that your patient group will welcome this decision.”

Everolimus is a holistic treatment for people living with TSC.  In addition to treating TSC-related refractory epilepsy, there are further benefits to patients including preventing high risk of kidney problems, lung problems and brain tumours (subependymal giant cell astrocytomas, or SEGAs).

The TSA is also contributing to work underway by NHS England in a bid to secure everolimus available on prescription for people with TSC-related refractory epilepsy in England.  A decision by NHS England’s Clinical Priorities Advisory Group (CPAG) is expected in the next few weeks.  “We are looking forward to what we hope will be a similarly positive decision about everolimus by NHS England,” said Maxine Smeaton.

Notes for editors
1. Maxine Smeaton and Craig Ireland are available for media interviews by contacting the TSA on 07870 210308.
2. The SMC’s advice on the adjunctive treatment of patients aged two years and older whose refractory partial-onset seizures, with or without secondary generalization, are associated with TSC can be found here:
3. NHS England consulted on draft guidance recommending the use of everolimus for refractory focal onset seizures in England during March 2018.  Details of the consultation can be found here:
4. NHS England has already issued advice to the NHS in England recommending the use of everolimus for TSC-related:
• kidney growths
• brain tumours
5. You can find more information about the TSA here: