A Review of Clinical Outcome Assessment Labeling in Europe and in the United States (2013-2016)

• Published in: Value in health 2017-10, Vol.20 (9), p.A742
• Main Authors: Arnould, B, Pierson, RF, Chan, E, Savre, I, Acquadro, C
• Format: Article
• Language: English
• Subjects: Central nervous system; Clinical assessment; Clinical outcomes; Connective tissue diseases; Drugs; FDA approval; Healthy food; Mental disorders; Mental health; Neoplasia; Nervous system; Nervous system diseases; Positioning; Questionnaires; Respiratory tract diseases; Skin diseases; Tumors; Websites
• ISSN: 1098-3015; 1524-4733
• DOI: 10.1016/j.jval.2017.08.2051

OBJECTIVES: A review of approvals by the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for 2000 to 2012 showed that respectively 21% and 26% of new drugs had patient-reported outcome (PRO) labeling. Our objectives were to expand the review to all clinical outcome assessments (COAs) for 2013 to 2016, and to identify: a) the endpoint positioning for each COA used, b) the therapeutic areas with highest number of products with COA labeling, and c) the type of instruments supporting PRO labeling. METHODS: The EMA and FDA websites were systematically explored to identify all approvals from 2013 to 2016. The PROLABELS database was used for COA labeling identification. All corresponding labels and reviews were reviewed. RESULTS: From 2013 to 2016, 407 and 244 new drugs were approved by the FDA and the EMA respectively. 37% of the FDA approvals had a COA labeling compared to 44% of the EMA approvals. 85 FDA drugs (21%) and 83 EMA drugs (34%) had a PRO labeling. Products (FDA/EMA) with other COA labeling were distributed as follows: ClinROs (67/52), PerfOs (27/32) and ObsROs (11/2). PROs were used as primary endpoints in 27% of all EMA and FDA products with a COA labeling, ClinROs in 48%, PerfOS in 59% and ObsROs in 40%. The highest number of products with a COA labeling were found in: respiratory tract diseases (n=43), nervous system diseases (n=34) and mental health disorders (n=26). Skin and connective tissue diseases were in the fourth position with 25 products and neoplasms at the fifth position with 24 products. PRO instruments used were often "legacy" instruments, i.e., SF-36 (22 products), St George's Respiratory Questionnaire (15 products), and Health Assessment Questionnaire (14 products). CONCLUSIONS: From 2013 to 2016, COAs played an important role in the evaluation of treatment benefit.