Why NICE is right to be cautious about recommending medicinal cannabis for two childhood epilepsy syndromes

Last week, the UK's medical advisory body, NICE, announced it would not be recommending that cannabidiol, a medicinal cannabis in the form of Epidyolex, should be prescribed on the NHS for children with two severe forms of epilepsy. Our medical director, Professor Ley Sander, explains why he believes they are right to exercise caution.

This news will undoubtedly come as a major blow to many families who have been pinning their hopes on a new drug with the potential to reduce seizures for children with Dravet Syndrome or for those who are in the Lennox Gastaut spectrum.

Epidyolex has been trialled over several years, in the US and in Europe.  While I have always said it would never be the magic bullet some had anticipated, it nevertheless offered another treatment option for children whose seizures do not respond to seizure drugs which are currently available.

NICE - the National Institute for Clinical Health and Care Excellence - has decided not to recommend Epidyolex on account of the fact that its long-term effect remains unclear. The body also has concerns about the 'viability of the economic model' used by GW Pharma, the company that developed the drug, to establish the cost to be charged to the NHS for it.  It concluded that Epidyolex would not, at this stage, be an effective use of NHS resources.

Promising treatment

As a clinician, I am encouraged that NICE has resolved to work with the pharmaceutical company to resolve these issues. Meindert Boysen, the director of the Centre for Health Technology Evaluation at NICE said: "The often distressing and life-limiting nature of these very difficult to control epilepsies mean that we should all welcome new treatment options. Cannabidiol is a promising treatment for people with these types of epilepsies.

“Even though the committee accepted that the evidence shows that cannabidiol with clobazam reduces seizure frequency, its long-term efficacy is unknown, and the committee was not convinced about the way the company had modelled the effect on people living longer or having a better quality of life.

“However, we are committed to working with the company to resolve the economic modelling issues identified by the committee, and to help them understand what they may need to do to mitigate the cost of cannabidiol to the NHS. Patients, carers and their families deserve no less.” I could not agree more.

Proper evaluation

Caution is always wise with any new medication but particularly so with medicinal cannabis where proper evaluation has been hijacked by emotional headlines and social media.

After several decades of treating people with epilepsy, there is nothing I would like more than to be able to tell those I see in clinic that at last, there is hope on the horizon in the form of a brand new medication.

Every day I see people who have had a lifetime of struggling with their epilepsy, whose seizures are not controlled with the 26 drugs already licensed in the UK, and who aren't candidates for epilepsy surgery as we cannot identify the focal point of their seizures in the brain.

But the last thing I would want to deliver is the false hope of something which has not been thoroughly evaluated and could have long term implications. I would not feel comfortable with a system that felt emotionally compelled to rush through a drug, bowing to the pressure of a cauldron of posts, likes and shares.

Another drug for Dravet syndrome

It is interesting to note that another new drug is currently being  tested for Dravet syndrome -Fintepla, a low dose fenfluramine drug which has been developed by Zogenix.

The drug has just been rejected by the US Food and Drug Administration on account of issues with data reporting and the need for further toxicology data.

But nobody is excited, outraged, frustrated. There have been no headlines, no campaigns. But then it's not cannabis.

As clinicians, as regulators, as advisory bodies -and as healthcare professionals who really care about the people we see, we must remain level headed in our judgement but sanguine in any setbacks. We must think long-term and not chase short-term headlines that could be replaced  with'blunders', 'mistakes', 'ill-advised' in not too many years from now.

Which is why I believe NICE has got it right and why I welcome their decision not to instantly recommend Epidyolex for the treatment of Dravet syndrome and those on the Lennox Gastaut spectrum. Rather to seek further clarification.

Have your say

The recommended guidelines are still only draft and the consultation closes on 16 September. So there is plenty of time for you to have your say and let them know what you think. I shall be doing the same.

All comments received will be considered by NICE and final guidance is likely to be published in November. Make sure you have your say.

Draft guidance on use of cannabidiol in the treatment of Dravet syndrome

Draft guidance on use of cannabidiol in the treatment of Lennox-Gastaut spectrum