Targeted Medical Therapies

For many of the rare and complex epilepsies, approved and evidencebased treatments do not exist. Thus, there is an urgent need to support high quality evidence of clinical studies and to identify and develop novel treatments targeting specific aetiologies.

What is this Working Group about?

Recognition of the epilepsies as a group of rare diseases with known aetiologies give us insight as to the underlying mechanisms involved in both development of seizure-generating networks, as well as comorbidities such as cognitive, physical, emotional and behavioural disturbances. To improve study designs and choice of relevant interventions, there is a need to collect standardized data on outcomes associated with specific treatments such as anti-seizure medications, repurposed drugs targeting underlying mechanisms, disease-modifying treatments (including immunosuppression, treatments targeting protein and gene expression), and non- pharmacological treatments (such as diets, neurostimulation, and surgical therapies) or interventions (cognitive, behavioral and physical therapies) in people with rare and complex epilepsy. To foster advancements, special interest groups (SIG) for specific epilepsies (e.g. autoimmune epilepsies, tuberous sclerosis, etc), and with focus in specific aspects of trial design (e.g. n-of 1- methodology, use of PCOMs etc.) were established to identify the particular needs of an entity to enable conduction and ultimately performance of clinical trials. The goals of this working group are to support and review initiatives in identifying unmet needs, study development and designs, provide expertise by contributing to activities of the European Consortium for Epilepsy Trials (ECET), disseminate these initiatives and study results. We also contribute to educational initiatives to improve the knowledge of novel trial designs, analysis, and outcome measures.