The benefit–risk profiles of pharmaceuticals are constantly evolving as new data are collected throughout the life cycle of a drug. Discussions during the workshop focused on the following: (1) premarket assessment by the FDA and/or industry, during which clinical trial data are used to assess benefit and risk; (2) communication of that information to prescribing physicians and their patients; (3) healthcare decisions made by prescribing physicians and their patients; and (4) the accumulation of benefit–risk information from postmarketing experience, which feeds back into the other phases (Figure 1-1).

The workshop considered the role of multiple stakeholders in benefit–risk assessment throughout the development process, including the FDA, a key player in benefit–risk analyses and decision making; academia, where much of the early discovery takes place; industry, where most of the development process occurs and where marketing plays an ever-increasing role in benefit–risk communication; and physicians and patients, where final decisions about drug use are made.

This workshop summary is organized into 6 sections. Section 2 focuses on the regulatory assessment phase of the benefit–risk assessment process. It identifies challenges and potential solutions in premarket assessment of benefits and risks, including approaches to evaluating drug safety. It also examines methods used by other industries for assessing benefits and risks. Section 3 addresses communication issues such as how to quantitatively communicate information about risk to the public, and possible solutions for providing user-friendly information to physicians and patients. Section 4 focuses on healthcare decision making by providers and patients. It discusses how individuals acquire information and make judgments about benefits and risks, how decisions depend on subtle contextual factors, and ways to help patients make informed decisions. Section 5 discusses patient experience with drugs over time and the limitations of postmarketing surveillance. Section 6 concludes with actionable next steps identified by workshop participants.

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