Abstracts

Rationale:


Alongside continued drug development, there is a need for refining clinical trial design to ensure that that the methods and results meet the needs of people with epilepsy, the wider epilepsy community, and regulatory agencies. A Core Outcome Set is a standardised list of outcomes that should be reported as a minimum in all trials. In epilepsy, the choice of outcomes measured varies widely among existing studies, particularly in randomised controlled trials, and we do not know if the outcomes currently being measured are relevant to the key stakeholders. Therefore, we have developed an internationally derived, patient-centric Core Outcome Set specific to adult epilepsy treatment trials.












Methods:


We performed a systematic review of the qualitative literature exploring experiences of people with epilepsy and reviewed outcomes already measured in phase 3 and 4 randomised controlled trials investigating adults with epilepsy, to generate an outcome long-list. In collaboration with the ILAE Big Data Commission and an international group of healthcare providers, researchers, and people with epilepsy we performed a grouping and rationalisation process and taken 42 individual outcomes to a two-stage, online Delphi survey followed by consensus meeting to decide which outcomes are critical to measure.








Results: 490 people with epilepsy, their representatives, healthcare professionals and researchers have completed the Delphi surveys in 7 languages, representing the global perspective. Inconclusive outcomes were discussed, and the final outcomes ratified at international online consensus meeting. The final Core Outcome Set included 10 outcomes that were deemed critical to measure by all the stakeholder groups: seizure frequency, seizure freedom, episodes of status epilepticus, health-related quality of life, overall health status, symptoms of depression and suicidality, side effects and their severity, and mortality.




Conclusions: The EPSET Project has identified a Core Outcome Set for adults with epilepsy and derived international consensus. This will ensure that meaningful outcomes are measured in future clinical trials, that the results of trials are relevant to people with epilepsy and facilitate systematic review and meta-analysis by aiming to reduce outcome measurement heterogeneity.


Funding: The EPSET project was jointly funded by the Association of British Neurologists and Guarantors of Brain charities, as part of a Clinical Research Training Fellowship.