Retrospective analysis of placebo arm data in clinical epilepsy trials -Goldenholz

BACKGROUND
Epilepsy affects at least 2.2 million Americans, costs $9.5-$12.5 billion annually, and has a 10- fold increased risk of sudden death compared to the general public. Medicines currently do not help 1 out of 3 patients with this disease, and surgery either is ineffective or unavailable to many of these patients. When new medicines are tested in clinical studies, they are compared to placebo. If the medicine is stronger than placebo, then it is considered effective.

Clinical studies of epilepsy report a “placebo effect” of 4-19%. Studies in the natural course of epilepsy have found 30-60% of patients that become seizure-free from their disease temporarily or permanently. Recently a simulation using at the largest database of seizures found effects identical to the “placebo effect” without any placebo being given. Taken together, it seems possible that the response to placebo in clinical studies might actually be the natural course of epilepsy, not the placebo effect. Also, it is possible that if we understand this problem better, we may be able to better tell the difference between a “powerful” medicine, and a non-helpful one.

HOW THIS WILL HELP PATIENTS
If the studies can tell the difference between “natural” changes in seizure versus changes from “placebo effects” or medicines, then better medicines can be found faster and for less money.