How FDA Approves Drugs and Regulates Their Safety and Effectiveness Page: 2 of 31
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How FDA Approves Drugs and Regulates Their Safety and Effectiveness
The Food and Drug Administration (FDA), a regulatory agency within the Department of Health
and Human Services, regulates the safety and effectiveness of drugs sold in the United States.
FDA divides that responsibility into two phases. In the peappoval (premarket) phase, FDA
reviews manufacturers' applications to market drugs in the United States; a drug may not be sold
unless it has FDA approval. Once a drug is on the market, FDA continues its oversight of drug
safety and effectiveness. That postappoval (postmarket) phase lasts as long as the drug is on the
market. Beginning with the Food and Drugs Act of 1906, Congress has incrementally refined and
expanded FDA's responsibilities regarding drug approval and regulation.
The progression to drug approval begins before FDA involvement. First, basic scientists work in
the laboratory and with animals; second, a drug or biotechnology company develops a prototype
drug. That company must seek and receive FDA approval, by way of an investigational new drug
(IND) application, to test the product with human subjects. It carries out those tests, called
clinical trials, sequentially in Phase I, II, and III studies, which involve increasing numbers of
subj ects. The manufacturer then compiles the resulting data and analysis in a new drug
At that point, FDA reviews the NDA with three major concerns: (1) safety and effectiveness in
the drug's proposed use; (2) appropriateness of the proposed labeling; and (3) adequacy of
manufacturing methods to assure the drug's identity, strength, quality, and purity. The Federal
Food, Drug, and Cosmetic Act (FFDCA) and associated regulations detail the requirements for
each step. FDA uses a few special mechanisms to expedite drug development and the review
process when a drug might address an unmet need or a serious disease or condition. Those
mechanisms include accelerated approval, animal efficacy approval, fast track designation,
breakthrough therapy designation, and priority review.
Once FDA has approved an NDA, the drug may enter the U. S. market, but FDA continues to
address drug production, distribution, and use. Its activities, based on ensuring drug safety and
effectiveness, address product integrity, labeling, reporting of research and adverse events,
surveillance, drug studies, risk management, information dissemination, off-label use, and direct-
to-consumer advertising, all topics in which Congress has traditionally been interested.
FDA seeks to ensure product integrity through product and facility registration; inspections;
chain-of-custody documentation; and technologies to protect against counterfeit, diverted,
subpotent, adulterated, misbranded, and expired drugs. FDA's approval of an NBA includes the
drug's labeling; the agency may require changes once a drug is on the market based on new
information. It also prohibits manufacturer promotion of uses that are not specified in the
labeling. The FFDCA requires that manufacturers report to FDA adverse events related to its
drugs; clinicians and other members of the public may report adverse events to FDA. The
agency's surveillance of drug-related problems, which had primarily focused on analyses of
various adverse-event databases, is now expanding to more active uses of evolving computer
technology and links to other public and private information sources.
The FFDCA allows FDA to require a manufacturer to conduct postapproval studies of drugs. The
law specifies when FDA must attach that requirement to the NBA approval and when FDA may
issue the requirement after a drug is on the market. To manage exceptional risks of drugs, FDA
may also require patient or clinician guides and restrictions on distribution. The agency publicly
Congressional Research Service
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How FDA Approves Drugs and Regulates Their Safety and Effectiveness, report, September 24, 2013; Washington D.C.. (digital.library.unt.edu/ark:/67531/metadc815699/m1/2/: accessed October 20, 2018), University of North Texas Libraries, Digital Library, digital.library.unt.edu; crediting UNT Libraries Government Documents Department.