Abstract
This work aimed to provide an understanding of the current use and regulatory acceptability of patient-reported outcome (PRO) measures in labeling claims for central nervous system (CNS) agents. A subset of CNS agents was identified from all New Drug Approvals and Biologic License Applications for new drugs approved in the US from January 2006 to June 2012. Clinician-reported outcomes (ClinROs) (62%) and PROs (38%) were the most widely used primary outcome measures. The PROs were frequently used in combination with ClinROs. Twelve PRO claims were granted across 41% of CNS drug approvals: 83%, symptoms; 17%, functioning. The PROs are frequently utilized as primary and secondary end points in CNS agents, and labeling claims are granted at higher levels than for non-CNS agents (41% vs 24%, respectively). These claims are granted at a lower rate than expected, given that direct patient input may lend valuable insight to treatment impacts in most CNS diseases.