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Balancing benefit and risk of medicines: a systematic review and classification of available methodologies

Mt-Isa, Shahrul, Hallgreen, Christine E., Wang, Nan, Callréus, Torbjörn, Genov, Georgy, Hirsch, Ian, Hobbiger, Stephen F., Hockley, Kimberley S., Luciani, Davide, Phillips, Lawrence D., Quartey, George, Sarac, Sinan B., Stoeckert, Isabelle, Tzoulaki, Ioanna, Micaleff, Alain and Ashby, Deborah (2014) Balancing benefit and risk of medicines: a systematic review and classification of available methodologies. Pharmacoepidemiology and Drug Safety, 23 (7). pp. 667-678. ISSN 1053-8569

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Identification Number: 10.1002/pds.3636


Background The need for formal and structured approaches for benefit–risk assessment of medicines is increasing, as is the complexity of the scientific questions addressed before making decisions on the benefit–risk balance of medicines. We systematically collected, appraised and classified available benefit–risk methodologies to facilitate and inform their future use. Methods A systematic review of publications identified benefit–risk assessment methodologies. Methodologies were appraised on their fundamental principles, features, graphical representations, assessability and accessibility. We created a taxonomy of methodologies to facilitate understanding and choice. Results We identified 49 methodologies, critically appraised and classified them into four categories: frameworks, metrics, estimation techniques and utility survey techniques. Eight frameworks describe qualitative steps in benefit–risk assessment and eight quantify benefit–risk balance. Nine metric indices include threshold indices to measure either benefit or risk; health indices measure quality-of-life over time; and trade-off indices integrate benefits and risks. Six estimation techniques support benefit–risk modelling and evidence synthesis. Four utility survey techniques elicit robust value preferences from relevant stakeholders to the benefit–risk decisions. Conclusions Methodologies to help benefit– risk assessments of medicines are diverse and each is associated with different limitations and strengths. There is not a ‘one-size-fits-all’ method, and a combination of methods may be needed for each benefit–risk assessment. The taxonomy introduced herein may guide choice of adequate methodologies. Finally, we recommend 13 of 49 methodologies for further appraisal for use in the real-life benefit–risk assessment of medicines

Item Type: Article
Official URL:
Additional Information: © 2014 John Wiley & Sons, Ltd.
Divisions: Management
Subjects: H Social Sciences > HD Industries. Land use. Labor > HD61 Risk Management
R Medicine > RS Pharmacy and materia medica
R Medicine > RZ Other systems of medicine
Date Deposited: 10 Dec 2015 10:37
Last Modified: 20 Aug 2021 03:01

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