Background: Neuropsychiatric symptoms are common in many neurologic diseases. Psychosis, depression, agitation, anxiety, irritability, and apathy occur in degenerative, traumatic, vascular, and demyelinating brain diseases.
Design: Regulatory policies regarding approval of psychotropic agents for treatment of neuropsychiatric symptoms in neurologic disorders are reviewed.
Results: Psychotropic agents previously received broad labeling such as treatment for psychosis or depression. Food and Drug Administration (FDA) policy now requires that labeling be limited to the population included in clinical trials providing the basis for the application. When applying for approval for use of psychotropic agents in neurologic disorders, pharmaceutical companies will be required to demonstrate efficacy in the neurologic disease of interest. Definitions of specific neuropsychiatric symptoms in neurologic diseases are required for study design. Approval will depend on demonstrating efficacy in two independent, randomized, controlled trials with symptom-focused and global outcome measures. Labeling for treatment of a nonspecific symptom (eg, agitation) can be approved if efficacy is demonstrated across several neurologic diseases.
Conclusion: Patients with neurologic diseases may have dosage ranges, side effects, or response profiles of psychotropic agents that differ from those of patients with idiopathic psychiatric disorders. FDA policy now requires demonstration of efficacy, safety, and tolerability of psychotropic agents within neurologic disorders, allowing clinicians to make more rational choices regarding treatment options.