Abstract
This statement provides general principles of the American Academy of Neurology's (AAN) approach when invited to provide guidance to policymakers on a variety of types of therapies that may have neurologic treatment benefits, but for which available evidence is limited or for which there is no US Food and Drug Administration-approved indication. In these instances, the AAN recommends an approach in which the patient and neurologist carefully review all available evidence and discuss the potential risks and benefits of the therapy, including when patients are contemplating asserting their right to try.