Abstract
Objectives Clinical trials of new therapeutics are typically evaluated against the primary disease symptom with limited consideration of secondary effects be they positive or negative. Desirability of outcome ranking (DOOR) is a novel methodology that combines benefits and harms to rank patients with respect to their overall clinical outcome. Herein we describe the consumer-led adaptation of DOOR as an outcome measure for a clinical trial of a novel treatment for drug-resistant epilepsy.
Methods Seven patients with epilepsy were interviewed to determine and prioritise the outcomes to be included in the DOOR scale. Hierarchical levels were established for each outcome (3–5 per outcome). Patients then ranked each combination of outcomes to determine the DOOR scale for use in the trial.
Results Seizure freedom/reduction was the most important outcome, followed by adverse event profile, psychiatric comorbidities and lastly cognitive deficits. Seizure frequency (100% reduction; >50% reduction, >25% reduction, <25% reduction), adverse event profile (none, mild temporary, mild ongoing, moderate, severe) and psychiatric symptoms (improvement, no change, worsening) were combined to create a DOOR with 60 unique outcomes.
Conclusion The DOOR methodological framework can be adapted for therapeutic trials in epilepsy. Consumer-codesign is important for meaningful outcomes in clinical research.
