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  • Writer's pictureAlta Flora

Launching Phytoepil - a digital observational study for childhood epilepsy

Updated: Feb 16

Today, we are announcing the launch of the Phytoepil study. Phytoepil has been developed with MedCan Support and Drug Science with the aim to generate Real-World Evidence (RWE) based on data related to symptoms, Quality of Life and health economic data from children with epilepsy who are using Cannabis-Based Medicinal Products (CBMP) on prescription.

MedCan Support is a non-profit community interest company who provide free resources and educational material on medical cannabis to support families with children with epilepsy. There are now over 600 families with children who have rare epilepsy conditions supported by the team at MedCan Support. Drug Science was formed in 2010 and is the UK’s leading body focused on developing rational policies on drugs.

Our team has worked in partnership with MedCan Support and Drug Science to develop a study based on our technology platform Eva. Eva is a mobile application designed for patients to participate in a health research study. Participants use Eva to answer questionnaires that researchers have defined in the study protocol. Eva includes customised questionnaires and Patient Reported Outcome Measures (PROMs) questionnaires displayed in a timeline with built-in reminders for participants. The questionnaires are displayed one question at a time in a user-friendly interface and researchers can access real-time data to develop RWE. The Eva mobile app facilitates communication between researchers from Imperial College and Drug Science, and the families of the children, ensuring real-time data collection on a pre-defined schedule on measures including Quality of Life in Childhood Epilepsy (QOLCE), and a custom questionnaire on medication and cost. Participants can join the study and complete the questionnaires from home at their own convenience via a link sent through email.

Since the rescheduling of CBMPs in November 2018, there has been limited progress in these medicines being prescribed to children with epilepsy in the UK. This has proved to be frustrating and costly. The NHS has failed in its stated aim of collecting data from its registry which launched in April 2021, and so private sector efforts have been the only efforts to study the impact of prescribing CBMPs to children with epilepsy.

At Alta Flora, we have been supporting efforts to undertake research in childhood epilepsy for a number of years now. This work is extremely challenging, and I hope that the data from the Phytoepil study will inform future research and policies which make the prescribing pathway easier for the children and their families.

A paper called The next generation of evidence-based medicine was published last month as part of a series in Nature called Rethinking evidence in medicine. The paper details advances in technology which have the potential to improve the drug development process and improve outcomes for patients. I particularly like the term “deep” medicine to describe taking advantage of all the new data sources which are available including wearables, genomics and environmental data. As somebody who has spent time in data-intensive organisations such as Facebook and Tesco, it has long felt that the existing framework for assessing the safety and efficacy of medicines is no longer fit for purpose.

There is a commonly accepted view in the medical establishment that the RCT is the gold standard, and that without RCT evidence of efficacy, claims of any effect of a drug can be put down to the placebo effect. This view is misguided, and is best summed up by the conclusion:

The success of future clinical trials requires a fundamental transformation in how trials are designed, conducted, monitored, adapted, reported and regulated to generate the best evidence. The status quo model is unsustainable. Instead, preventive, personalized, pragmatic and patient-participatory medicine is needed, and paradigm shifts are required to get there via sustainable growth. Silos need to be broken. Standards of care and clinical trials are currently viewed in different realms; however, the overarching goal of both is to improve health outcomes. The COVID-19 pandemic created an opportunity to observe how routine clinical care and clinical trials can work synergistically to generate evidence. Pragmatic platform trials such as the RECOVERY trial should be a model and guide for trial efficiency and real-time impact.

Current paradigms must be continuously challenged by emerging technology and by all stakeholders (the new generations of scientists, physicians, the pharma industry, regulatory authorities and, most importantly, patients). Disruptive innovation should lead to every clinical site being a research site, with all necessary quality checks and research as part of the standard of care. The healthcare system should be integrated into an intuitive RWE-generation system, with clinical research and clinical care going hand in hand.

This desire to “integrate an intuitive RWE-generation system” into clinical research and clinical care encapsulates much of what we have been doing at Alta Flora for the last few years. Thus, the primacy of the RCT is being challenged by advances in technology which enable more innovative trial methodologies. As an outsider to healthcare looking in, the RCT is more focused on the needs of industry and regulators rather than those of patients. What if trials focused on what patients thought important, rather than what well-meaning but poorly informed clinicians felt would protect their reputations? What if trials were designed by patients to improve quality of life rather than designed by industry to meet regulatory approval?

It is with this in mind - a focus on the metrics that are most meaningful to patients and caregivers - that the Phytoepil study has been developed. Like many other areas in medicine, this is a community-driven effort and we hope to shortly be able to make available the tools that we have used to develop this study to other patient communities who need this infrastructure to run patient-centric research.

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