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

TY - JOUR

T1 - Balancing benefit and risk of medicines

T2 - a systematic review and classification of available methodologies

AU - Mt-Isa, Shahrul

AU - Hallgreen, Christine E

AU - Wang, Nan

AU - Callréus, Torbjörn

AU - Genov, Georgy

AU - Hirsch, Ian

AU - Hobbiger, Stephen F

AU - Hockley, Kimberley S

AU - Luciani, Davide

AU - Phillips, Lawrence D

AU - Quartey, George

AU - Sarac, Sinan B

AU - Stoeckert, Isabelle

AU - Tzoulaki, Ioanna

AU - Micaleff, Alain

AU - Ashby, Deborah

AU - IMI-PROTECT benefit-risk participants

N1 - Copyright © 2014 John Wiley & Sons, Ltd.

PY - 2014/7/1

Y1 - 2014/7/1

N2 - 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.

AB - 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.

KW - Decision Making

KW - Drug-Related Side Effects and Adverse Reactions

KW - Humans

KW - Models, Statistical

KW - Pharmaceutical Preparations

KW - Quality of Life

KW - Risk Assessment

KW - Journal Article

KW - Research Support, Non-U.S. Gov't

KW - Review

U2 - 10.1002/pds.3636

DO - 10.1002/pds.3636

M3 - Journal article

C2 - 24821575

SN - 1053-8569

VL - 23

SP - 667

EP - 678

JO - Pharmacoepidemiology and Drug Safety

JF - Pharmacoepidemiology and Drug Safety

IS - 7

ER -