BOX 2-1

Benchmarks in the Regulation of Pediatric Drugs

1902 Biologics Control Act

1906 Pure Food and Drug Act

1938 Food, Drug, and Cosmetic Act

1962 Kefauver-Harris Amendment

1979 labeling requirement

1994 Pediatric Labeling Rule

1997 Food and Drug Administration Modernization Act (FDAMA)

1998 Pediatric Rule*

2002 Best Pharmaceuticals for Children Act (BPCA)

2003 Pediatric Research Equity Act (PREA)


*The Pediatric Rule was enjoined, or prohibited, in 2002 by a federal court, which ruled that Congress had not given the FDA authority to require extensive testing of drugs for children (Association of American Physicians and Surgeons, Inc. v. U.S. Food and Drug Administration, 226 F Supp 2d 204 [DC Cir 2002]).

SOURCE: Mathis, 2006.

thalidomide, a sleeping pill, caused severe birth defects in the offspring of European women who took it, as well as women in the United States who gained access to it as an investigational new drug. Before the amendment was passed, an FDA New Drug Application had to demonstrate only that the drug was safe. Under the amendment, an FDA New Drug Application was required to demonstrate that the drug was effective as well as safe.

Many of the incidents that inspired the above legislation involved children, but according to Dr. Mathis, the resulting laws benefited adults disproportionately. Information on the use of drugs in children was limited and remained insufficient for decades. Then in 1979, the FDA issued a requirement that labels note specifically whether safety and efficacy had been established in pediatric populations. The 1994 Pediatric Labeling Rule, another FDA regulation, requested that the pharmaceutical industry submit literature and other data providing additional information on the use of drugs in pediatric patients. However, it proved relatively ineffective. In 1997, the Food and Drug Administration Modernization Act (FDAMA) provided incentives for companies to test drugs in pediatric populations voluntarily: 6 months of additional marketing exclusivity and patent protection when studies are performed in children as

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