Benefit–Risk Assessment in the Regulation of Pharmaceuticals

Pharmaceutical medicine is a ubiquitous technology with profound impacts on society. Regulatory decisions authorizing the marketing of pharmaceuticals require sound science and principled judgments about the meaningfulness of benefit, the acceptability of risk, and the tolerance of uncertainty. This commentary uses the U.S. Food and Drug Administration’s (FDA’s) Benefit–Risk Framework as an organizing construct to describe the foundational factors of regulatory decision making, including the degree of patient need, the strength of scientific evidence, and regulatory options to manage uncertainty and risk. It illustrates some of the more nuanced societal questions that a regulatory decision may need to address concerning heterogeneity of patients’ outcomes, complex tradeoffs, and individual autonomy. Framed for a broader scientific audience, the paper connects pharmaceutical benefit–Risk assessment to universal principles of risk governance for technologies with consequential public impact.