BOX 1-1

Some Key Milestones in FDA History

The Pure Food and Drug Act of 1906 gave FDA’s predecessor, the Bureau of Chemistry in the Department of Agriculture, its first regulatory powers. At inception, the agency’s pharmaceutical regulatory work focused largely on misbranding and adulteration of drugs. In 1937, elixir sulfanilamide caused more than 100 deaths and led to the passing of the 1938 Federal Food, Drug, and Cosmetic (FD&C) Act. The act prohibited false therapeutic claims for drugs and for the first time required premarket notification of FDA by the sponsor for all new drugs. This meant that a company submitted its New Drug Application (NDA) and, if FDA did not explicitly prohibit marketing, the company was free to market the product without any type of approval after 60 days (unless FDA extended that period to 180 days), when the NDA became “effective.” Although the FD&C Act required a manufacturer to prove a drug’s safety by conducting preclinical toxicity testing and gathering and submitting drug safety data, it did not require proof of efficacy (Swann, 1998; Stergachis and Hazlet, 2002). Some two decades later, thousands of children with birth defects were born to European mothers who had taken the popular sedative thalidomide for morning sickness. Marketing of thalidomide in the United States had been held up in the approval process and this so-called near miss led to the Drug Amendments of 1962. The drug amendments required companies to provide proof of efficacy of a drug for it to be considered for marketing approval, and the randomized controlled trial became established as the gold standard for demonstrating efficacy (Stergachis and Hazlet, 2002).

In the 1980s, the public health crisis of HIV/AIDS motivated a powerful advocacy movement whose aims included faster approval of drugs for patients with incurable disorders. Other consumer and patient advocacy groups began to call for changing the drug approval process to speed up the availability of potentially life-saving or life-sustaining drugs to patients in need of them. Consumer groups, regulators, the regulated industry, and others contributed to and Congress passed the PDUFA legislation that aimed to ensure that FDA had adequate resources to expand its drug review staff and capabilities, and so to increase the pace of drug reviews. Agreements among FDA, industry, and Congress are crystallized in a series of performance goals for FDA. These are not part of the PDUFA statute, so they lack the force of law (Tauzin, 2002) but they reflect activities the agency considers its obligations—“The letter outlines goals that the agency must meet, which help frame the basis to judge the

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