Benefit‐risk Assessment of Investigational Drugs: Current Methodology, Limitations, and Alternative Approaches

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Abstract
Development of investigational drugs is a process integrated traditionally into four overlapping phases. The goal is to introduce new therapies to clinical medicine by assessing benefits and risks associated with administering the new drug. Benefit assessment is performed with respect to the disease for which the drug may comprise an effective treatment. In contrast, safety assessment is relatively standardized across many pharmacologic classes of agents. For purposes of benefit‐risk assessment, investigational drugs are developed to provide benefit in three major disease categories: acute, episodic, and chronic. Benefit assessment is the major focus of conventional methodologies. Inherent limitations of risk assessment produced by conventional approaches are illustrated by the historical inability to detect toxicities of various drugs until large patient populations have been treated, typically after the drug is marketed. Alternative approaches to overcome these limitations include (1) assessment of safety in studies specifically designed to optimize such evaluation and (2) more extensive safety testing of investigational drugs in patient subgroups at higher risk. Such approaches serve the interest of patients, physicians, and developers by facilitating the development of new therapies by providing a more complete benefit‐risk assessment prior to initial marketing of the drug.

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