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Drug Science research predicts 96% response rate for epileptic children treated with medical cannabis


Father and daughter playing

Authors

Rayyan Zafar, Anne Katrin Schlag, Larry Phillips, David Nutt

Published

N/A

Despite medical cannabis being legalised in 2018 thanks to a campaign by parents of children diagnosed with severe epilepsy, 3 years on regulations are still blocking access to an NHS prescription for the vast majority of children affected by the same condition. However, Drug Science hopes that new clinical analysis sent to Matt Hancock could finally unlock the problem.


This new clinical analysis could end a regulatory standoff that is leaving severely epileptic children without the treatments that could help them most. Conducted by Drug Science, the analysis focuses on the use of medical cannabis for under-18s living with a range of refractory epilepsies.

The analysis sent to the Health Secretary Matt Hancock shows that:

  1. Drug Science’s audit of real-world evidence provides an alternative dataset and prediction of response to randomised controlled trials (RCTs) on a group of 21 participants with a range of rare severe childhood epilepsies.

  2. The next patient has a 96 % chance of having a significant reduction in seizures as well as a significant reduction in other health problems.

  3. The patients’ condition is so greatly improved that no other statistics are required to establish that medical cannabis is extremely effective for patients with refractory epilepsy.


"Our analysis shows that these cannabis-based medications are safe, and that a child has a 95% chance of having a significant reduction in seizures and other health problems relating to their condition, were they to be prescribed. In contrast, the best outcome of typically prescribed NHS medications, such as benzodiazepines or a drug called Epidiolex, is less than 50%. The problem now is getting decision makers to accept the evidence”

– Prof David Nutt, Founder of Drug Science

Real-world data vs randomised controlled trials


While it is possible to prescribe a pure cannabidiol medicine Epidiolex for two rare forms of childhood epilepsy in the UK (Lennox-Gastaut and Dravet syndromes), current regulations prevent the use of whole plant extract cannabidiol (medical cannabis). This is due to the absence of RCTs having proved it as effective and safe.


However, Professor Nutt states that conducting medical cannabis RCTs on these children with epilepsy “faces enormous obstacles. Not least that they would require half of participants to stop their current treatment, which would be wildly unethical, dangerous and could even prove fatal.”


‘Real-world evidence’ (RWE) is data collected by patients as they are actually use a medicine, such as the data used to reveal that established Covid-19 vaccines work against the virus’ ‘Indian’ variant. And while our case series analysis shows highly significant improvements for children being given medical cannabis, it is not currently accepted as evidence by NICE and NHS England; even though both bodies have expressed support for such forms of clinical research in the past.

“Therefore, medical cannabis for severe childhood epilepsy is effectively blocked under current NHS rules, despite convincing clinical evidence on its safety and efficacy. Moreover, a Refractory Epilepsy Specialist Clinical Advisory Service (RESCAS) set up by the NHS England to improve access to such treatments has, instead, rejected almost all applications for children whose epilepsy has already responded extremely well to private prescriptions of medical cannabis. It’s an absurd situation that cruelly affects these families and it needs to change.”

– Prof David Nutt, Founder of Drug Science

“Charlie would have upwards of 100 seizures every day. He would not be able to sleep or even function, and the standard medications that were prescribed on the NHS did very little to help. I often wonder why these drugs were so available to us, even though they had some dreadful side-effects and they had not been through RCTs themselves. The only thing that has helped Charlie has been a private prescription of medicinal cannabis.”

– Matt Hughes, whose three-year-old son Charlie was diagnosed with epilepsy when he was less than a year old.

Charlie is now given a cannabis-medication called Celixir20, which he takes as three liquid doses (each of 0.4ml) each day. The medicine is privately sourced from a company in Israel, where it is approved and licensed for use on childhood refractory epilepsy.


“He now has fewer than 20 seizures a day, and those are far less severe. He sleeps well, eats well and the improvement in his health is self-evident. Yet his medicine is still not considered to be clinically effective by UK legislators, simply because it hasn’t been studied with an RCT. In fact, our clinician has told me that he would never recommend Charlie take part in such a study, for fear it would jeopardise his newfound stability.”

– Matt Hughes, whose three-year-old son Charlie was diagnosed with epilepsy when he was less than a year old.

As a result of his family’s experiences, Hughes set up MedCan Support – helping other parents to access private prescriptions, offering information and support, and advising on how best to cover the high costs of privately-sourced medical cannabis.


“I have wiped out my own savings looking after Charlie and my story is far from unique. I regularly hear from people who have been forced to sell their house, just to help their child, or who are doing everything they can to raise funds. It’s a stressful and precarious position for parents, wondering if we’ll be able to afford the medicines that our children need.”

– Matt Hughes, whose three-year-old son Charlie was diagnosed with epilepsy when he was less than a year old.


“But what is absolutely clear to us is that these medicines work. Scientists around the world agree, and the amount of real-world evidence on the effectiveness of medical cannabis is growing. An insistence on RCTs means we will never be able to help these children so decision-makers within the NHS, the British Paediatric Neurology Association and other clinical bodies need to change tack - and accept real-world clinical analysis such as ours. Because these medicines work.”

– Prof David Nutt, Founder of Drug Science

What’s next? 

Drug Science is holding a panel discussion with live Q&A on Thursday 27th May to mark National Epilepsy Awareness Week. The webinar will cover legalisation in 2018 and shine a light on the Epilepsy patients and their parents who are still battling the health system to access medical cannabis both privately and on the NHS. It will explore the options available to patients and families now, as well as what plans are in the pipeline, and how people can help spread awareness of the issues faced by these families. Finally, the event will highlight our current deficit of prescribing doctors and the urgent need for more paediatric neurologists to come forward as prescribers of medical cannabis.


Drug Science is committed to giving children with Epilepsy access to reduced price medical cannabis through Project Twenty21, but in order for this to happen, we need the doctors to break through the stigma, learn how to prescribe medical cannabis products and encourage their clinician peers to do the same.

Drug Science has written to the Health Secretary, Matt Hancock, highlighting the results of this research and the urgent need for action.

Read the letter below:



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