Quantitative Risk/Benefit Assessment: Where Are We?
Pharmaceutical sponsors use a variety of approaches to make important benefit/risk decisions about their products internally. Benefit/risk assessment is equally important when regulators evaluate a product for marketing approval and payers evaluate it for reimbursement decision. Once a product receives marketing authorization, it is critical to communicate pertinent benefit and risk information to patients and health-care providers. All of the above can be made easier by the use of a common framework. In this paper, we review where we are in benefit/risk assessment. This includes endeavors by academic institutions, regulators, and the pharmaceutical industry. Despite concerns about quantitative benefit/risk assessment expressed by some, we argue that without a way to quantitatively incorporate the relative importance of factors impacting benefit/risk assessment, it will be hard to bring transparent decisions to questions such as “does the benefit of this product outweigh the risk.”
KeywordsBleeding Event Utility Weighting Advisory Committee Meeting Fourth Hurdle Advisory Committee Member
The author wants to thank Jon Norton for the use of Fig. 2. The author also wants to thank PhRMA BRAT, especially Bennett Levitan, Paul Coplan, Rebecca Noel, Marilyn Metcalf, and Diana Hughes, for BRAT-generated materials. In addition, the author wants to thank Leila Zelnick and Tom Fleming for their comments which have helped improve the quality of the paper.
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