BOX 2-1
Key Definitions

Benefit assessment and risk assessment: The gathering and analyzing of information on the nature and magnitude of potential benefits and potential harms (risks) associated with a drug and the determination of the likelihood that those benefits and harms will occur.

Benefit–risk profile: An overall evaluation of the benefits and risks associated with a drug.

Benefit–risk management: The process of identifying, evaluating, selecting, and implementing actions to increase benefits and reduce risk to human health. The goal of benefit–risk management is scientifically sound, integrated actions that increase or maintain benefits and reduce or prevent risks while taking into account social, cultural, ethical, political, and legal considerations. “A good risk management decision emerges from a decision-making process that elicits the views of those affected by the decision, so that differing technical assessments, public values, knowledge, and perceptions are considered” (Presidential/Congressional Commission on Risk Assessment and Risk Management, 1997). The process of benefit–risk management should include not only information about current regulatory actions but plans for future evaluations and regulatory actions as part of the lifecycle process.

Uncertainty: Lack or incompleteness of information. Quantitative uncertainty analysis attempts to analyze and describe the degree to which a calculated value may differ from the true value; it sometimes uses probability distributions. Uncertainty depends on the quality, quantity, and relevance of data and on the reliability and relevance of models and assumptions.

Box 1-1; the question of which study designs FDA should require is addressed in Chapter 4).

THREE-STAGE FRAMEWORK FOR REGULATORY DECISION-MAKING

Overview and Rationale

Responding in a timely and appropriate way to safety signals from already-approved drugs is among the most important and challenging public health jobs that FDA must accomplish. Permitting a drug to stay on the market that is on



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