title: Patient representatives' contributions to the benefit-risk assessment tasks of the European Medicines Agency scientific committees.
abstract: In the European Medicines Agency (EMA), the involvement of patients has been increasingly recognized as valuable and necessary. Specifically in scientific committees, patients through patient representatives are actively involved in deliberations and decision making processes. These scientific committees are meant to ensure that licensed medicines have a positive benefit-risk ratio in favour of the patients and users. To investigate what the contributions are of patient representatives in benefit-risk assessment, we interviewed 15 scientific committee members, 10 of whom are/were EU-state regulatory representatives and five are/were patient representatives. We asked the participants questions related to the benefit-risk assessment tasks of their committees, the connection between patient representatives and the patient perspective, and the contribution of patient representatives in the various benefit-risk assessments tasks. We found that the contribution of patient representatives benefit-risk assessment may be a variable of the benefits and the risks involved in the drug such that the necessity of their contribution is strongly felt when both benefits and risks are high, when benefits are almost equal or are equal to risks and when both benefits and risks are low. In terms of the various benefit-risk tasks, patient representatives contribute to benefit-risk analysis by providing criteria that help define the benefit-risk picture. In benefit-risk evaluation, patient representatives aid in providing a specific basis for the values and weights given to specific benefits and risks and in decision making, they provide what may be a crucial patient perspective in terms of the acceptability of risks. Hence, patient representatives provide a specific expertise in these scientific committees.
authors.affiliation: Julius Center for Health Sciences and Primary Care, Utrecht University Medical Center, Str. 6.131, PO Box 85500, 3508GA, Utrecht, The Netherlands.
authors.forename: Rosemarie D L C
authors.initials: RD
authors.lastname: Bernabe
authors.forename: Ghislaine J M W
authors.initials: GJ
authors.lastname: van Thiel
authors.forename: Johannes
authors.initials: J
authors.lastname: van Delden
mesh_terms: D003657:Decision Making; D004358:Drug Therapy; D005062:European Union; D006801:Humans; D010344:Patient Advocacy; D018570:Risk Assessment; D012586:Science
keywords: European Medicines Agency; benefit-risk assessment; patient participation; patient representatives; scientific committees