title: Role of patient-reported outcomes and other efficacy endpoints in the drug approval process in Europe (2008-2012).
abstract: The present study aimed at systematically reviewing the role and extent of patient-reported outcomes (PROs) usage within the package of scientific evidence considered for marketing authorization (MA). All regulatory information published by the European Medicines Agency (EMA) for products authorized between January 2008 and December 2012 and appearing in the European Public Assessment Report (EPAR) database was examined for efficacy endpoints. The endpoints here considered included: PROs, clinician reported outcomes (CROs), and laboratory reported outcomes (LROs). LROs were the most frequently reported endpoints. Out of the 180 products here selected, 99 (55%), 67 (37%), and 30 (17%), respectively, used LROs, CROs and PROs as primary endpoints (PEs). PROs as any endpoints were used in 82 (46%) products. Out of these, PROs were documented as PE in 30 (37%), with 27 (33%) products having used PROs both as primary and non-PEs. PRO usage was most frequently identified with nervous system and antineoplastic agents. During the study period, the use of all the three types of endpoints appeared to be static. Both the regulatory bodies and the industry should ensure complete and clear reporting of all endpoints used, including PROs, to improve transparency.
authors.affiliation: Department of Pharmacy Practice, National Institute of Pharmaceutical Science, Mohali, India. Electronic address: dipikabansal079@gmail.com.
authors.forename: Dipika
authors.initials: D
authors.lastname: Bansal
authors.affiliation: Department of Pharmacy Practice, National Institute of Pharmaceutical Science, Mohali, India. Electronic address: anilbhagat05@gmail.com.
authors.forename: Anil
authors.initials: A
authors.lastname: Bhagat
authors.affiliation: Clinical Pharmacology and Therapeutics, University of Hertfordshire, Hertfordshire, UK. Electronic address: f.schifano@herts.ac.uk.
authors.forename: Fabrizio
authors.initials: F
authors.lastname: Schifano
authors.affiliation: Department of Pharmacy Practice, National Institute of Pharmaceutical Science, Mohali, India. Electronic address: kapil.gudala@gmail.com.
authors.forename: Kapil
authors.initials: K
authors.lastname: Gudala
mesh_terms: D017277:Drug Approval; D005060:Europe; D006801:Humans; D063868:Patient Outcome Assessment; D016896:Treatment Outcome
keywords: European Medicines Agency; European Public Assessment Report; Health related quality of health; Patient reported outcomes