Federal Court Invalidates FDA’s Pediatric Rule on Drug Safety Tests

WASHINGTON—A federal court has invalidated the FDA’s "Pediatric Rule" after concluding that its requirement that drug companies test the safety of adult drugs in children is contrary to the intent of Congress.

Judge Henry H. Kennedy, Jr., of the US District Court for the District of Columbia, enjoined the FDA from enforcing the regulation. He found that the rule exceeded the wording of laws passed by Congress that provide specific incentives to the pharmaceutical industry to establish the safety of new and approved drugs in pediatric patients. The FDA can appeal Judge Kennedy’s ruling to the Circuit Court of Appeals.

The agency proposed the Pediatric Rule in 1997, stating that it did not believe "that incentives alone will result in pediatric studies of some of the drugs and biologics where the need is greatest." Oncologic drugs were one area of particular concern.

The rule became effective on April 1, 1999, and required companies filing new drug applications to provide "data adequate to assess whether the drug is safe and effective in pediatric populations." Pediatric testing could be deferred until after the FDA approved the adult usage of the drug. The rule also gave the agency the power to require companies to provide evidence to support the dosage and administration of approved drugs in some or all of the pediatric subpopulations, including neonates, infants, children, and adolescents.

"The Pediatric Rule may well be a better policy tool than the one enacted by Congress; it might reflect the most thoughtful, reasoned, balanced solution to a vexing public health problem," Judge Kennedy said in his decision. "The issue here is not the rule’s wisdom. . . . The issue is the rule’s statutory authority, and it is this that the court finds lacking."

The rule was challenged by three groups—the Association of American Physicians and Surgeons and two advocacy groups that oppose excessive government regulations, the Competitive Enterprise Institute and Consumer Alert.

In his decision, Judge Kennedy acknowledged that the Pediatric Rule sought to address a significant public health issue. He noted that because of expense and the difficulty of recruiting significant numbers of pediatric patients in clinical trials, many drugs are tested in adults only. As a result, he said, "physicians with pediatric patients often find their treatment options limited." Often the decision is made to prescribe a drug off-label at a reduced dosage, which "can expose children to various hazards."

Congress, in passing the Food and Drug Administration Modernization Act (FDAMA) in 1997, was aware of FDA’s proposed pediatric rule, Judge Kennedy noted. It chose, however, to establish a 5-year experimental program to encourage pediatric testing in which the FDA could request but not require manufacturers to test new drugs on pediatric patients. Companies that agreed to the testing received an added 6 months exclusivity on their drugs.

FDA made the Pediatric Rule final after concluding that the incentives did not increase pediatric testing. This year, Congress enacted the Best Pharmaceuticals for Children Act (BPCA), which expanded the earlier pediatric test incentives but again did not authorize FDA to require the testing of drugs in pediatric patients.

Plaintiffs’ Argument

The plaintiffs’ attorneys argued that FDA exceeded its powers in the Pediatric Rule. "FDA has no authority to require manufacturers to conduct studies of drug uses for which they do not intend to seek approval or devise formulations of the drug tailored to those uses," they said in a motion to overturn the rule.

Judge Kennedy noted that a basic tenet of administrative law holds that an agency’s power to promulgate legislative regulations is limited by the authority that Congress delegates to it. "This court must determine whether Congress intended to vest the FDA with authority to promulgate the Pediatric Rule," he wrote.

The judge also cited a 1984 Supreme Court decision that outlined a two-step process for courts to use in reviewing a federal agency’s interpretation of a statute. First, the court must ask whether Congress had addressed the precise issue in a law, and, second, if Congress had not, then the court must defer to the agency’s construction of the statute, so long as it is reasonable.

FDA’s Argument

FDA argued that, in part, its authority to order the rule derived from several sections of the Food, Drug and Cosmetic Act. One vests the agency with the power to set regulations to enforce the act. Another section requires labeling of adequate directions for drug use, and a third deems a drug misbranded if it endangers health "when used in the dosage or manner, or with the frequency or duration prescribed in the labeling."

Judge Kennedy rejected the agency’s arguments. "It is simply difficult to see how such power can be wholly derived from the FDA’s power over drug labeling," he said. "Moreover, if Congress had intended for these sections to authorize the FDA to require manufacturers to test their drugs for unclaimed use, Congress would likely have spoken more clearly."

He also ruled that the BPCA and the Pediatric Rule were incompatible. "Congress adopted an incentive scheme while the FDA adopted a command and control approach. The two schemes differ in almost every possible regard," he wrote.

He rejected FDA’s interpretation of the laws governing it as supporting the rule, and concluded that in creating marketing incentives to encourage voluntary testing, Congress had rejected requiring the testing of adult drugs to ensure their safety in pediatric patients.