Benefit-Risk Evaluation and Decision Making: Some Practical Insights

Pharmaceutical drugs and devices are increasingly evaluated by quantitative tools that combine benefit and risk. These tools vary by their limitations and desirable properties, which may confuse the decision-making process. Experts from the Food and Drug Administration (FDA) and industry shared thei...

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Bibliographic Details
Published in:Therapeutic innovation & regulatory science 2015-05, Vol.49 (3), p.425-433
Main Authors: Colopy, Michael W., Damaraju, C. V., He, Weili, Jiang, Qi, Levitan, Bennett S., Ruan, Shiling, Yuan, Zhong
Format: Article
Language:English
Subjects:
Authorship
Decision making
Drug Safety and Pharmacovigilance
Pharmacotherapy
Pharmacy
Prescription drugs
Product development
Product Development and Innovation: Meeting Report
Regulatory approval
Stroke
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Summary:Pharmaceutical drugs and devices are increasingly evaluated by quantitative tools that combine benefit and risk. These tools vary by their limitations and desirable properties, which may confuse the decision-making process. Experts from the Food and Drug Administration (FDA) and industry shared their perspectives at the 2012 American Statistical Association (ASA) Biopharmaceutical Section FDA-Industry Statistics Workshop, and these insights are presented here. First, benefit-risk terminology is given to better understand subtle distinctions. Next, pragmatic considerations in endpoint selection are given that distinguish between benefit-risk assessment and analysis of clinical trials. Then the strengths of weighting methods, including ranking, utilities, and risk tolerance for assessing the trade-off between benefits and risks, are compared. The last topic presented is summarizing information to ease the interpretation, transparency, and ability to support decisions. Benefit-risk methods are moving towards a unified paradigm to make selection of endpoints, weights, and metrics easier and more structured. This will lead to better decision-making based on a transparent assessment and clear interpretability.
ISSN:2168-4790
2168-4804
DOI:10.1177/2168479014565469