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.
