Abstract
BackgroundThe Compassionate Access Scheme (CAS) being delivered through the Queensland Children’s Hospital is designed to allow access to an investigational purified Cannabidiol oral solution to paediatric patients with severe refractory epilepsy. The objectives of this study were to conduct semi-structured interviews to:1. Understand families’ expectations and attitudes about the use of an investigational cannabinoid product for their child’s seizures;2. Understand families’ perceptions of Cannabidiol’s efficacy for their child’s seizures; and other aspects of their child’s behaviour, quality of life and/or cognition.MethodsChildren aged 2-18 years had been enrolled in, or were enrolled in a compassionate access scheme for Cannabidiol at the time of the study. Semi-structured interviews (n = 19) with parents or caregivers (n = 23) of children diagnosed with refractory epilepsy were voice-recorded, transcribed and analysed to generate common themes.ResultsKey themes emerged relating to seizure activity, family and school engagement, drug safety and legal access, efficacy, clinical support, social acceptance of the medication and program delivery. The use of Cannabidiol was perceived to have benefits in relation to reducing the severity and frequency of seizure activity for almost a third of patients experiencing refractory epilepsy. Participants described other benefits including improved social engagement, wakefulness and a reduction of side effects related to a reduction of conventional medication dosage.ConclusionThis study provided unique perspectives of families’ experiences managing untreatable epilepsy, their experiences with conventional and experimental pharmacological treatments and health services. Whilst families’ perceptions showed the use of Cannabidiol did not provide a therapeutic reduction in the seizure activity for all patients diagnosed with refractory epilepsy, it’s use as an additional pharmacological agent was perceived to provide other benefits by some patient families.
Highlights
The Compassionate Access Scheme (CAS) being delivered through the Queensland Children’s Hospital is designed to allow access to an investigational purified Cannabidiol oral solution to paediatric patients with severe refractory epilepsy
The burden of epilepsy disease has been calculated in disability adjusted life years (DALYs), a population measure of health loss accounting for years of life lost (YLL) and years of life lived with disability (YLD) [2]
Clinical trial staff were recruited, trained and accredited according to Good Clinical Practice (GCP) standards accepted by International regulatory bodies and pharmaceutical compliance officers operating in Australia and other countries to administer the CAS program [13]
Summary
The Compassionate Access Scheme (CAS) being delivered through the Queensland Children’s Hospital is designed to allow access to an investigational purified Cannabidiol oral solution to paediatric patients with severe refractory epilepsy. Epilepsy is commonly defined as a disorder of the brain characterised by an enduring predisposition to generate epileptic seizures with unknown origin or, as a result of infection, injury, tumour or disease [1, 2]. Refractory, or untreatable epilepsy has been defined as the “diagnosis of drug-resistant epilepsy as a result of a failure of adequate trials of two tolerated and appropriately chosen and used anti-epileptic drug (AED) schedules (whether as monotherapies or in combination) to achieve sustained seizure freedom” [3]. Whilst the burden of disease has been calculated for idiopathic presentation of epilepsy as approximately 0.5% of all DALYs for all disease, the burden of disease for untreatable epilepsy is estimated to be even higher, approximately sevenfold [7]
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