Patient Activists are Speaking Out After NHS England Decided Not to Fund a Drug to Treat TSC-Associated Epilepsy

The National Health Service (NHS) England is facing criticism following their decision not to fund the drug everolimus as a treatment for tuberous sclerosis complex associated refractory epilepsy. In comparison, NHS Scotland has announced that they will fund everolimus for this purpose. The source article can be read here, at The York Press.

Philippa’s son Thomas is six years old and suffers from tuberous sclerosis complex (TSC) and epilepsy. Philippa says that for Thomas, taking everolimus was life-changing. Before trying the drug, he would have as many as eight seizures per day and had been on several different medications to try to manage them.

Four years ago, Thomas started taking part in a clinical trial in which he was given everolimus, which Philippa says, “changed our lives.” She says that since taking the drug, her son’s seizure-control has improved, and his tumours haven’t grown. His development has also progressed, and now he’s much happier and spends his time watching YouTube and dancing in the kitchen. She says that without everolimus Thomas “wouldn’t be like this.” She adds,

“When your child has been on six different epilepsy drugs without much success, you begin to lose hope; but this medicine gave us hope.”

Thomas may be able to continue receiving everolimus as part of a clinical trial, but many other patients in England will not have this option. Maxine Smeaton, Chief Executive of the Tuberculosis Sclerosis Association, said in a statement, “NHS England’s decision to ignore the evidence base and feedback from experts treating and living with TSC is disappointing beyond words.”

According to the Tuberous Sclerosis Association’s website, epilepsy affects 80% of people with TCS, and, of these, over half do not respond to standard anti-epilepsy medicines.

Ms Smeaton says,

“The difference that everolimus makes to the lives of people with TSC who have refractory epilepsy is unprecedented, and the TS community in England will be devastated by this news.”

After reviewing the drug, NHS England decided not to include it in their budget for the coming year. Annually, the cost of supplying it to eligible people is estimated to be almost £60 million. A spokesperson for NHS England said that this decision was partly based on the view that everolimus “delivered limited benefits compared to other treatments being considered.” This decision is in contrast to that of NHS Scotland, who plan to make everolimus available.

However, NHS England’s decision may be reviewed in November 2018 at the next Clinical Priorities Advisory Group meeting.

About Tuberous Sclerosis Complex

Tuberous sclerosis complex is a genetic condition that leads to tumours developing around the body. These tumours are usually benign, and often affect the heart, eyes, kidneys, brain, lungs, and skin. People with TSC often also develop associated conditions, such as epilepsy. TSC is linked to an alteration in the TSC1 or TSC2 genes, which help to regulate cell growth. An estimated 10,000 people in the UK are born with TSC.

About Everolimus

Everolimus is a mTOR inhibitor that can block processes involved in tumour growth. It has been shown to shrink brain tumours caused by TSC and can be used to treat TSC-associated epilepsy as well.

To raise awareness for this issue and campaign for change, the Tuberous Sclerosis Association is using the hashtags #Everolimusforepilepsy #NHSEnglandwrongdecision #Wewontgiveup.

Anna Hewitt

Anna Hewitt

Anna is from England and recently finished her undergraduate degree. She has an interest in medicine and enjoys writing. In her spare time she likes to cook, hike, and hang out with cats.

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