Gnanasakthy Ari, DeMuro Carla, Clark Marci, Mordin Margaret, Thomas Simu
1 Novartis Pharmaceuticals, East Hanover, NJ, USA.
2 RTI Health Solutions, Research Triangle Park, NC, USA.
Ther Innov Regul Sci. 2013 Sep;47(5):613-618. doi: 10.1177/2168479013495686.
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.
这项工作旨在了解患者报告结局（PRO）指标在中枢神经系统（CNS）药物标签声明中的当前使用情况和监管可接受性。从2006年1月至2012年6月在美国获批的所有新药的新药批准申请和生物制品许可申请中，确定了一部分CNS药物。临床医生报告结局（ClinROs）（62%）和PROs（38%）是使用最广泛的主要结局指标。PROs经常与ClinROs联合使用。在41%的CNS药物批准中，有12项PRO声明获得批准：83%为症状；17%为功能。PROs经常被用作CNS药物的主要和次要终点，并且标签声明的批准率高于非CNS药物（分别为41%和24%）。鉴于直接的患者反馈可能为大多数CNS疾病的治疗影响提供有价值的见解，这些声明的批准率低于预期。
