PNS4 A REVIEW OF PERFOS IN FDA AND EMA LABELLING CLAIMS (2009-2019)

Abstract

Performance outcomes (PerfOs) present challenges for establishing content validity and measurement properties for drug labelling (Richardson et al., 2018). PerfOs can be broadly classified as those assessing physical, cognitive, and sensory function. The primary aim of this study was to review the distribution of PerFO types in FDA and EMA labelling claims in the past decade. The secondary aim was to review the indications for which cognitive PerfOs were included in drug labelling claims. PerfOs included in labelling claims were identified via the PROLABELS database. The approval date was refined between 2009 and 2019. The results were then categorised in terms of the type of PerfOs and the regulatory body that granted their use in drug labelling claims. A total of 146 drugs with a PerfO in the label were identified, with a total of 185 PerfOs included. The majority of PerfOs related to respiratory (57.30%), followed by physical (26.5%), sensory (8.65%), cognitive (3.78%), ADHD symptoms (2.16%), driving (1.05%), and sleep parameters (0.54%). About half of the listed PerfOs were included in EMA (51.89%) and about half in FDA labels (48.1%). Indications for which cognitive PerfOs were included in the label were Epilepsy, Major Depressive Disorder, Alzheimer Disease, and Sleep initiation and maintenance. The majority of PerfOs approved in drug labelling claims related to respiratory and physical performance, and only a small number related to cognitive performance. There was not much difference in the distribution of approval by regulatory bodies (FDA or EMA). Despite cognitive performance being pertinent for many neurological and psychiatric indications, only a small range of indications had a cognitive PerfO included in the label. Further guidance and discussions on PerfOs, particularly related to cognition, are needed to expand the number and variety of PerfO that can be used as endpoints to support drug labelling claims.