Quantitative Risk/Benefit Assessment: Where Are We?

Abstract

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.”