Recommendations for benefit-risk assessment methodologies and visual representations.
Author(s)
Type
Journal Article
Abstract
PURPOSE: The purpose of this study is to draw on the practical experience from the PROTECT BR case studies and make recommendations regarding the application of a number of methodologies and visual representations for benefit-risk assessment. METHODS: Eight case studies based on the benefit-risk balance of real medicines were used to test various methodologies that had been identified from the literature as having potential applications in benefit-risk assessment. Recommendations were drawn up based on the results of the case studies. RESULTS: A general pathway through the case studies was evident, with various classes of methodologies having roles to play at different stages. Descriptive and quantitative frameworks were widely used throughout to structure problems, with other methods such as metrics, estimation techniques and elicitation techniques providing ways to incorporate technical or numerical data from various sources. Similarly, tree diagrams and effects tables were universally adopted, with other visualisations available to suit specific methodologies or tasks as required. Every assessment was found to follow five broad stages: (i) Planning, (ii) Evidence gathering and data preparation, (iii) Analysis, (iv) Exploration and (v) Conclusion and dissemination. CONCLUSIONS: Adopting formal, structured approaches to benefit-risk assessment was feasible in real-world problems and facilitated clear, transparent decision-making. Prior to this work, no extensive practical application and appraisal of methodologies had been conducted using real-world case examples, leaving users with limited knowledge of their usefulness in the real world. The practical guidance provided here takes us one step closer to a harmonised approach to benefit-risk assessment from multiple perspectives. Copyright © 2016 John Wiley & Sons, Ltd.
Date Issued
2016-01-22
Date Acceptance
2015-12-08
Citation
Pharmacoepidemiology & Drug Safety, 2016, 25 (3), pp.251-262
ISSN
1053-8569
Publisher
Wiley
Start Page
251
End Page
262
Journal / Book Title
Pharmacoepidemiology & Drug Safety
Volume
25
Issue
3
Copyright Statement
This is the peer reviewed version of the following article: Hughes, D., Waddingham, E., Mt-Isa, S., Goginsky, A., Chan, E., Downey, G. F., Hallgreen, C. E., Hockley, K. S., Juhaeri, J., Lieftucht, A., Metcalf, M. A., Noel, R. A., Phillips, L. D., Ashby, D., Micaleff, A., and PROTECT Benefit–Risk Group (2016) Recommendations for benefit–risk assessment methodologies and visual representations. Pharmacoepidemiol Drug Saf, 25: 251–262 , which has been published in final form at https://dx.doi.org/10.1002/pds.3958. This article may be used for non-commercial purposes in accordance With Wiley Terms and Conditions for self-archiving.
Sponsor
Commission of the European Communities
Grant Number
115004
Subjects
benefit-risk
decision-making
drug development
pharmacoepidemiology
regulation
Publication Status
Published