The CBD-based drug, Epidyolex, will now be prescribed through the NHS for the treatment of seizures related to tuberous sclerosis complex (TSC).
From 1 March 2023, patients living with TSC will have access to the cannabinoid-based treatment, Epidyolex, through the NHS, following a new recommendation from the The National Institute for Health and Care Excellence (NICE).
According to an announcement from producer Jazz Pharmaceuticals on Tuesday 31 January, NICE has recommended the treatment be available for reimbursement by NHS England as an adjunctive therapy for seizures associated with TSC in patients two years of age and older.
TSC is a rare genetic condition, which is estimated to affect between 3,700 and 11,000 people in the UK and around 1 million people worldwide. It causes mostly benign tumours to grow in vital organs of the body, including the brain, skin, heart, eyes, kidneys and lungs.
Epilepsy is one of the most common neurological features and can severely impact the lives of patients, with conventional treatments unable to control seizures in up to half of cases.
While TSC can be diagnosed in infancy, many children are not diagnosed until later in childhood when seizures begin and other symptoms appear.
Epidyolex, which contains an isolated form of CBD, has now been approved for use in the UK in three rare forms of epilepsy. It has been prescribed through the NHS since 2019 for seizures associated with Lennox Gastaut syndrome or Dravet syndrome, in conjunction with clobazam, for patients two years of age and older.
In August 2021 it was approved by the UK Medicines and Healthcare Products Regulatory Agency (MHRA) for use as an adjunctive treatment of seizures associated with TSC. The Scottish Medicines Consortium, Northern Ireland’s Strategic Planning and Performance Group and the All Wales Medicines Strategy Group have all previously approved the drug for use in the condition.
Dr Pooja Takhar, joint chief executive at Tuberous Sclerosis Association (TSA), said: “The TSA is delighted with this decision, along with people living with TSC and their families. TSC is a very difficult to manage condition, with common issues including epilepsy in eight out of 10 people.
“Up to half of the people with TSC-related epilepsy are unable to manage their seizures with standard anti-seizure medication, leading to a massive unmet need for new treatment options. This underlines why we are so pleased that this medicine will now be available on the NHS in England, improving lives in the TSC community.”
Epidyolex was developed by GW Pharmaceuticals and is one of just three licensed cannabis medicines in the UK. GW Pharma was acquired by Jazz Pharmaceuticals in May 2021 for $7.2 billion.
Simon Newton, general manager at Jazz Pharmaceuticals, commented: “We welcome NICE’s recommendation which provides appropriate patients across the UK, who are living with TSC, a difficult to treat condition, access to a new treatment option. This is an important milestone not only for those living with TSC but also for their families, carers and clinicians.
“This demonstrates the importance of randomised clinical trials (RCTs) and regulatory approval in providing reimbursed access to cannabinoid-based medicines to patients who may benefit.”
A number of children with treatment-resistant epilepsy are prescribed unlicensed cannabis medicines privately in the UK, however regulators say more RCTs are needed before these can be approved for use through the NHS.
Last year NHS England and the National Institute for Health Research (NIHR) were said to be ‘working closely’ on two RCTs to compare the effects of medicines containing CBD, and CBD with THC, and a placebo. These were expected to begin in 2023 but further information is yet to be released.
NHS director of Specialised Commissioning and interim director of Commercial Medicines, John Stewart, said: “It is great news for patients that the NHS is able to offer this latest licensed cannabis treatment, which in this instance can help reduce the seizure frequency for those living with a serious genetic condition and significantly improve their quality of life.
“The NHS is committed to making innovative treatments available to patients as quickly as possible, at a fair price to taxpayers, following regulatory approval that provides patients with the knowledge that new treatments are safe and manufactured in a quality controlled environment.”
