Adjunctive clinical trials in epilepsy: is a placebo arm necessary?

Abstract

One of the fundamental questions that needs to be answered when evaluating an investigational drug in randomized clinical trials is whether the study drug demonstrated efficacy for the condition under evaluation. For patients with epilepsy, clinical trials are initially conducted to assess the efficacy of the investigational agent when administered as adjunctive therapy. This design is derived from ethical concerns that argue against exposing patients with epilepsy to a study drug as monotherapy without some evidence of efficacy. Once efficacy is demonstrated in adjunctive clinical trials, the study drug can be subsequently evaluated in monotherapy trials.