Abstracts

Rationale: A Core Outcome Set (COS) is a standardised list of outcomes that should be reported as a minimum in all trials. It is developed using consensus methods to ensure that it includes what is important to patients as well as healthcare professionals and researchers. In epilepsy, the choice of outcome measures varies widely among existing studies, particularly in randomised controlled trials (RCT). This diminishes opportunities for informed decision making, contributes to research waste and is a barrier to integrating findings from multiple RCTs in systematic reviews and meta analyses. COS facilitate the undertaking of trials that are relevant to patients and health services and help standardise trial methodology so more meaningful results can be obtained from systematic review and meta-analysis. We have developed an internationally derived COS specific to adult epilepsy treatment trials, in line with COMET Initiative methodology.

Methods: We have undertaken a rapid review of the qualitative literature exploring experiences of people with epilepsy PWE, to identify core concepts that map to potential measurable outcomes. This work represents the views of over 2000 people with epilepsy across 6 continents. We have also undertaken rapid review of outcomes already measured in phase 3 and phase 4 epilepsy specific RCTs registered on the clinicaltrials.gov and ISRCTN registries to generate a long list of potential outcomes to take to an international consensus process. In total, over 300 granular outcomes were identified from this process. In collaboration with the ILAE Big Data Commission and an international group of epileptologists, people with epilepsy and methodologists we have performed a grouping and rationalisation process, and taken 42 individual outcomes to a two-stage, online, Delphi consensus process. People with epilepsy, their representatives, healthcare professionals and researchers have completed the Delphi surveys in 8 languages, representing the global perspective. 

Results: The ratified COS will represent what is deemed to be critical to measure in clinical trials assessing the effectiveness of treatments for adults with epilepsy. Given that the COS is informed by a diverse group of stakeholders, it will be applicable to clinical trials performed globally. Future work should identify the most appropriate measurement instruments to capture each item in the COS.

Conclusions: The EPSET Project has identified a COS for adults with epilepsy and deriving international consensus, will ensure that meaningful outcomes are measured in future clinical trials, ensure that the results of trials are relevant to the needs of people with epilepsy, reduce research waste and ensure that the results of trials assessing the same treatment can be combined in meaningful ways.

Funding: The EPSET Project is funded by an Association of British Neurologists and Guarantors of Brain Charity Clinical Research Training Fellowship (awarded to J Mitchell).