In any given month, an estimated 48 percent of Americans take at least one prescription drug. Prescription drugs are crucial for preventing and treating diseases and improving the public’s health, but they can also have unintended harmful effects. Often, their benefits and risks cannot be fully identified until after a drug has been used by a large, diverse group of patients over time, mainly because clinical trials conducted before approval may be too small or too short to detect all possible risks. The passage of the Food and Drug Administration Act in 2007 provides the Food and Drug Administration (FDA) with additional postmarketing regulatory tools to better protect the health of the public, including the authority to require manufacturers to continue studying drugs that are being marketed.
To help determine when it is appropriate to require a postmarketing study, which types of studies to require, how to best protect the rights and interests of patients who participate in research, and how to use research in making regulatory decisions, the FDA asked the IOM to evaluate the scientific and ethical aspects of conducting safety studies for approved drugs. The IOM concludes that the FDA’s current approach to drug oversight in the postmarket setting is not sufficiently systematic and does not ensure that it assesses the benefits and risks of drugs consistently over the drug’s life cycle. Adopting a regulatory framework that is standardized across all drugs, yet flexible enough to adapt to regulatory decisions of differing complexity, could help make the agency’s decision-making process more predictable, transparent, and proactive. These changes could allow the FDA to better anticipate post-approval research needs and improve drug safety for all Americans.