The Epilepsy‐Desirability of Outcome Ranking ( DOOR ) as a Multi‐Faceted Consumer‐Informed Outcome Measure for Epilepsy Clinical Trials

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BACKGROUND: New drug trials in drug resistant epilepsy are typically powered to detect changes in seizure frequency as the primary endpoint, without integrating other treatment-associated benefits and harms. We developed Epilepsy-DOOR, a consumer-codesigned outcome measure, which combines seizure frequency with quality of life and adverse event measures. This study evaluated Epilepsy-DOOR in previously completed phase 3 clinical trials of adjunctive brivaracetam in patients with drug resistant epilepsy. METHODS: Epilepsy-DOOR was derived for each participant who completed the randomised controlled trial of three Phase 3 trials of brivaracetam (N01252, N01253, N01254). Win odds were estimated for Epilepsy-DOOR and its individual components: change in seizure frequency, quality of life, and adverse event severity for treatment with brivaracetam over placebo for each dose in each study. Odds ratio was estimated for responder rate. RESULTS: In N01252, Epilepsy-DOOR demonstrated benefit of 100 mg brivaracetam (Win odds 1.42, 95% CI 1.03, 1.97) but not smaller doses over placebo, in line with the results when using responder rate (odds ratio 1.14, 95% CI 1.02, 1.29). In studies N01253 and N01254, benefit as assessed by Epilepsy-DOOR did not attain statistical significance, despite benefits at some doses when measuring seizure responder rate. CONCLUSION: Epilepsy-DOOR showed similar effect sizes but slightly reduced power when compared with responder rate. This reduced power is due to the appropriate reflection of adverse events by Epilepsy-DOOR. Epilepsy-DOOR provides a more holistic measure of anti-seizure medication treatment effects, balancing relative benefits and harms, and has potential as a future endpoint in clinical trials in epilepsy.

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