The Epilepsy-Desirability of Outcome Ranking (DOOR) as a Multi-Faceted Consumer-Informed Outcome Measure for Epilepsy...

Area of Science:

• Neurology
• Clinical Trials
• Pharmacology

Background:

• Traditional epilepsy drug trials focus solely on seizure frequency.
• This overlooks other crucial patient-reported outcomes like quality of life and adverse events.
• Epilepsy-DOOR was developed as a consumer-codesigned, comprehensive outcome measure.

Purpose of the Study:

• To evaluate the efficacy of the Epilepsy-DOOR measure.
• Assessed in Phase 3 clinical trials of adjunctive brivaracetam for drug-resistant epilepsy.
• Compared Epilepsy-DOOR with traditional responder rates.

Main Methods:

• Utilized data from three randomized controlled trials (N01252, N01253, N01254) of brivaracetam.
• Calculated win odds for Epilepsy-DOOR and its components (seizure frequency, quality of life, adverse events).
• Estimated odds ratios for responder rates for comparison.

Main Results:

• Epilepsy-DOOR showed significant benefit for 100 mg brivaracetam in N01252, aligning with responder rates.
• In N01253 and N01254, Epilepsy-DOOR did not reach statistical significance despite some dose-specific responder benefits.
• Effect sizes were comparable, but Epilepsy-DOOR showed slightly reduced statistical power.

Conclusions:

• Epilepsy-DOOR provides a more holistic assessment of anti-seizure medication efficacy by integrating benefits and harms.
• The reduced power is attributed to its appropriate inclusion of adverse event data.
• Epilepsy-DOOR holds potential as a future endpoint in epilepsy clinical trials for a balanced treatment evaluation.