On Monday February 13, International Epilepsy Day, MedCan Support, Drug Science and Alta Flora announced the launch of a next-generation digital observational study, for children with epilepsy who are prescribed cannabis oil.
Patient group MedCan Support and scientific body Drug Science are proud to announce the launch of a next-generation, digital observational study to learn more about the signs and symptoms of rare childhood epilepsy conditions.
The study will be run using health technology company Alta Flora’s Eva Research Platform, with data submitted by caregivers and parents of children with epilepsy through the platform’s dedicated app, for analysis by Drug Science researchers.
There are over 35,000 children with refractory epilepsy in the UK, defined as epilepsy where a patient’s seizures fail to come under control with treatment.
Cannabis was made illegal in the UK in 1971 under the Misuse of Drugs Act. This change in legislation also prevented a great deal of meaningful research into the drug and its possible benefits for patients.
Yet over the last few years, there have been many prominent examples of the drugs being beneficial for children with refractory epilepsy, helping to control seizures and improving the quality of life for both patients and their families. These case studies have been reported in the United States, Canada, Australia, Israel and other countries and have spurred renewed interest from the global research community into the effects of cannabis on childhood epilepsy.
Alongside these developments, healthcare research has seen numerous developments, including a growing interest in genomics and the incorporation of data from new technologies (such as wearable devices, similar to widely-available fitness trackers) into medical studies. Furthermore, the COVID-19 pandemic has required researchers to adapt to different ways of conducting clinical trials and prompted international regulators to acknowledge that inclusivity in healthcare research needs to be improved.
The Eva Research Platform fits neatly into these developments. It is a patient-centric research platform that enables innovative data collection through a mobile app. Alta Flora’s company mission is to make healthcare research more open and inclusive; and designers believe that research into childhood epilepsy epitomises the type of research the platform was designed to facilitate.
Epilepsy is a disease which impacts over 600,000 people in the UK, but research investment has been lacking, with only £12.8m of government funding invested (vs. an annual cost to the NHS of £1.5B). There has been a paucity of research into new treatment options and quality of life improvements. The Alta Flora platform will allow researchers to generate high-quality, longitudinal evidence on epilepsy.
Following campaigns led by parents whose children responded well to whole-plant medical cannabis extracts (but who had failed on conventional antiepileptic drugs (AEDs) and other mainstream treatments), medicinal cannabis was made legal and reintroduced as a medicine in the UK in 2018.
One of those parents, Hannah Deacon, is a founding Director of MedCan Support. Another is Matt Hughes, who brought a legal case against the National Institute of Clinical Excellence (NICE), leading to a change of guidance around when it is appropriate for Consultant Paediatric Neurologists to prescribe medical cannabis for children with epilepsy.
Despite these efforts – and others by campaigning parents of children with epilepsy — this vulnerable group of patients has struggled to find doctors willing to prescribe medicinal cannabis, because of a lack of high quality Randomised Controlled Trial (RCT) data on their safety and effectiveness. In fact, only 3 prescriptions have received NHS funding since the drugs were made legal, leaving many families having to pay for their medical cannabis prescriptions through private clinics and dispensaries.
MedCan Support and Drug Science are seeking to address perceived lack of high-quality evidence through the launch of a new digital observational study to collect Real World Evidence on the impact that medicinal cannabis can have.
Real World Evidence
Real World Evidence (RWE) has emerged as a powerful means by which investigators and regulators can support research objectives and inform clinical guidance. In June this year, NICE published its guidance on RWE, stating:
“We want to use real-world data to resolve gaps in knowledge and drive forward access to innovations for patients.”
Given the gaps in RCT evidence for childhood epilepsy syndromes, it appears that RWE could play a crucial role as NICE acknowledged in their guidance:
Randomised trials may not be available for several reasons, including:
● randomisation is considered unethical or unfeasible (for instance, for some rare or severe diseases with unmet need)
● technical challenges make randomisation impractical, which is most common for medical devices and interventional procedures
● funding is not available for a trial (for example, when the intervention is already used in routine practice).
Real-world data could be used more routinely to fill evidence gaps and speed up patient access.
NICE further elaborated on the attributes of RWE that they expect to see:
● ensure data is of good provenance, relevant and of sufficient quality to answer the research question
● generate evidence in a transparent way and with integrity from study planning through to study conduct and reporting
● use analytical methods that minimise the risk of bias and characterise uncertainty
To capture RWE, Drug Science and MedCan Support have worked with health technology company Alta Flora to leverage its platform for data collection by parents and caregivers of children with epilepsy. Researchers will gather data via validated clinical questionnaires delivered through a parent or caregivers mobile app on:
– Seizure frequency and intensity
– Prescribed and administered dosages of the drug
– Physiological and behavioural effects of the drug
– Financial costs
The data which will be collected through this observational study is in accordance with the NICE criteria for acceptance as high-quality RWE, and is expected to inform future clinical trial design and data collection methodology.
The study launches today and will run for at least 12 months.
Hannah Deacon, co-founder of MedCan Support said: “We are pleased to announce the launch of this next generation digital observational study. I know only too well the importance of securing treatments that work for your child when they are suffering severe seizures. We hope that collecting this data and presenting it will go some way to bring confidence to prescribers and ensure safe and effective treatments are available to vulnerable children as quickly as possible.”
David Badcock, CEO of Drug Science, said: “Drug Science has been at the forefront of the research effort into cannabis in the UK for several years, and this observational study will build on the work that our research teams have done in childhood epilepsy since cannabis was reintroduced to the British pharmacopoeia in 2018. We are pleased to work on this pioneering project with MedCan Support and Alta Flora and hope that the longitudinal Real World Evidence that we are now able to collect will advance the case for wide access to these transformational medicines.”