NHS to offer licensed cannabis-based medicine to treat rare genetic condition

The NHS will be able to prescribe cannabidiol to patients with a rare, seizure-causing genetic disorder in England from today (Tuesday) after the National Institute for Health and Care Excellence (NICE) issued final guidance recommending its use.

Around one in every 6,000 people suffer from tuberous sclerosis complex (TSC) that causes seizures, which severely affect their quality of life from a very young age, as well as their families and carers.

Around 1,000 patients could benefit from the treatment, with clinical trials showing that when used alongside standard patient care such as typical antiseizure medications, cannabidiol (also known as Epidyolex®) reduces the frequency of seizures by almost a third (30%), increasing the number of days patients can go without a seizure compared with placebo, and lowering the risk of sudden death.

As a result of fewer, less severe and more predictable seizures, patients and families feel more confident when leaving the house and better enabling activities such as attending school.

It will become the fifth indication for which a cannabis treatment approved by regulators is offered to NHS patients in England, alongside treatments for people with multiple sclerosis, severe epilepsies known as Dravet and Lennox-Gastaut Syndrome, and for adults experiencing nausea caused by chemotherapy.

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.”

One patient benefitting from cannabidiol is 16-year-old Marcus, who was diagnosed with tuberous sclerosis complex (TSC) at 10 weeks of age as well as Lennox-Gastaut Syndrome, and currently attends a local special needs school.

Marcus’ mother and full-time carer, Sarah, says that there had been limited long-term success with other medications after the initial “honeymoon period” had passed, but this new treatment has changed all that more than two years on, after initially receiving it for Lennox-Gastaut Syndrome.

Sarah said: “With cannabidiol being such a new medication, I read up as much as I could about it and agreed to give it a go. My theory has always been ‘If you do not try, you will never know’, after all this could be ‘the one’… which I have hoped for with every new medication he has tried, and you cannot give up.

“Marcus started cannabidiol in October 2020, as always on a low dose and we gradually increased to his current dose. Marcus’ neurologist suggested giving clobozam on a daily basis, as reports had shown that cannabidiol and clobozam work very well together.

“It worked! Marcus has gone from around 6-10 seizures a day to 1-2 in a week! I am absolutely thrilled!

“As a parent of a child with TSC the seizures are very difficult to control. I have always been aware the ‘best we can get them’ is the reality. The combination of clobozam and cannabidiol has been phenomenal for us, but if we had not been given the opportunity to try it I dread to think where we would be now.

“I have been doing this for 16 years so after that amount of time, trying all kinds of different medications and having my son being the best he has ever been is just amazing and I thank my lucky stars every day.”

There is some evidence that cannabidiol can improve behaviour and reduce anxiety, with half of people with TSC also suffering from learning disabilities.

The treatment may also reduce the number of people hospitalised due to seizures.

Dr Pooja Takhar, Joint Chief Executive of Tuberous Sclerosis Association, said: “We’re thrilled that people with TSC in England will now have access to cannabidiol, a potentially life-changing medicine for the eight in 10 people in the UK who have TSC and also difficult to treat TSC-related epilepsy.

“Epilepsy can have a massive impact on overall quality of life for individuals and entire families, meaning that this approval could have a huge benefit to many people with TSC-related epilepsy. We worked tirelessly to make sure that NICE came to the right decision. Although this is a big victory, our work doesn’t stop and we continue to advocate and campaign for the TSC community in all areas.”

Patients will have needed to have had limited or no success with two other anti-seizure medications to be eligible for cannabidiol, before specialist consultants will decide if it is clinically appropriate to be prescribed to a patient.

The NHS is committed to rolling out the latest and most innovative treatments at a price affordable for taxpayers, following assessment by NICE, and recently became the first country in Europe to fast-track darolutamide to patients whose prostate cancer has spread to other parts of the body.

The health service also agreed deals for other breakthrough drugs last year, including the lifesaving gene therapy, Libmeldy® for children with the rare condition metachromatic leukodystrophy, and last week concluded a deal that secures long-term access to personalised CAR T cancer treatment for the treatment of an aggressive lymphoma.