of product labeling, prohibited the adulteration or misbranding of both foods and drugs, and ensured that drugs were consistent with national formulary standards.

During its 100-year history, food and drug regulation in the United States has evolved in response to a series of significant public health events. For example, the deaths of more than 100 people who had taken a new but untested sulfanilamide elixir led to the 1938 Food, Drug and Cosmetic Act, which established a requirement for premarket safety testing. When thalidomide (a sedative taken by pregnant woman to relieve morning sickness and aid sleep) caused thousands of European babies to be born with birth defects, Congress recognized the need for premarket safety and efficacy testing and passed the 1962 Kefauver-Harris Amendments. These amendments also called for the retroactive examination of all drugs introduced since 1938. Investigation of the nearly 3,500 drugs introduced between 1938 and 1962, as mandated by the Kefauver-Harris Amendments, was among the first of many increases in the scope of the FDA’s regulatory responsibility that were unfunded by Congress. More recently, the Hatch-Waxman Act, enacted in 1984 to ease the entry of generic drugs into the market and respond to concerns about drug pricing, led to an increase in the number of applications the agency had to review.

Dr. Henney commented that while necessary for preserving and advancing public health, this continual expansion of the FDA’s mandate has created financial challenges for the agency in the absence of corresponding funding. By the early 1990s, there was a substantial backlog of New Drug Applications (NDAs), and review times had increased. To quell growing industry frustration with the unpredictability of the FDA review process, as well as to meet the desperate need of AIDS patients for access to new therapies, Congress passed the Prescription Drug User Fee Act of 1992 (PDUFA).2

As noted in Chapter 1, recent public health events, such as the withdrawal of Vioxx and the association between the use of selective serotonin reuptake inhibitors (SSRIs) and increased risk of suicidal ideation in children, have renewed public concern about drug safety and the FDA’s ability to regulate it. Public outcry resulting from these events has led FDA officials and lawmakers to reexamine the agency and the current legislation that governs its role in regulating drug safety. Currently, the 110th Congress is considering the reauthorization of PDUFA, as well as several legislative proposals to improve the U.S. drug safety system.


PDUFA was enacted by Congress in 1992 and revised in 1997 and 2002. Under this program, the pharmaceutical/biotechnology industry pays fees to the FDA, and in return the FDA agrees to meet drug-review performance goals (e.g., reviewing NDAs and Biological License Applications [BLAs] within specified time periods).

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