Senate considers Pediatric Rule

ELK GROVE VILLAGE, Ill. — Legislation introduced in the U.S. Senate would require pharmaceutical companies to test new drugs and biologics for safety, efficacy and dosing information in children.

“This bill guarantees that children will be on equal therapeutic footing with adults,” said AAP president E. Stephen Edwards, MD.

The Pediatric Research Equity Act (S. 650) restores the 1998 FDA regulation known as the Pediatric Rule that was struck down by the courts in October. At the time, a judge ruled that Congress had not given the FDA the authority to carry out such a mandate. Prior to being struck down, the Pediatric Rule helped generate more than 50 pediatric studies since 1999.

AAP intervenes

After the FDA declined to file an appeal on behalf of the government, the AAP and the Elizabeth Glaser Pediatric AIDS Foundation requested and were granted the right to intervene in the legal action. The judge noted that the two groups had been directly harmed by the loss of the regulation and that their intervention would help resolve the case.

The AAP believes that the FDA does have the statutory and administrative authority to require pediatric testing. There are strong legal precedents demonstrating that the FDA has the authority to require labeling of a medication for “usual and customary” uses. Approximately 75% of all drugs prescribed for children are not tested for their use. The Pediatric Rule, along with a federal incentive program that provides valuable patent extensions for companies that voluntarily conduct pediatric drug tests, has been responsible for at least 98 studies that have improved the safety of medicines for children by identifying proper dosing, safety and adverse events. Without the Pediatric Rule, only half of those studies would have been conducted, according to the FDA.

The bill clearly states that if children are going to be taking a medication, it first needs to be tested for their use. The legislation complements the AAP-backed Best Pharmaceuticals for Children Act (BPCA), a law that provides financial incentives to drug companies that voluntarily test medicines frequently prescribed to children. According to the AAP, both policies are needed, since the Pediatric Rule covers drugs, age groups and uses that BPCA does not. For example, S.650 covers all new drugs and biologic products for the labeled indication. The FDA could not require a manufacturer to test the medicine for an off-label use.

“Once the testing is complete, the drug’s label would indicate that it’s safe for children of certain ages, along with the proper dose,” said Richard Gorman, MD, chairman of the AAP’s Committee on Drugs. “The medicine would also come in a liquid or chewable tablet, if necessary. That’s critical when you are treating infants and toddlers.”

Manufacturers can ask for a waiver or a deferral of the requirement in a number of instances, such as when the drug is not going to be used in at least 50,000 children or if pediatric testing could delay availability of the product for adults.

When the AAP endorsed S. 650, an amendment was added and approved by a Senate committee that would allow the legislation to expire by the end of 2007. The AAP is against the amendment, saying the bill should be permanent.

The U.S. House of Representatives has not introduced its version.

Making the rule law

While the AAP pursues legal action, it is also continuing to press for federal legislation. “We are committed to pursuing all avenues for permanently reinstating the Pediatric Rule,” said Gorman. “In addition to the legal action, we continue to work on federal legislation that would make the Pediatric Rule the Pediatric Law. It is the surest and swiftest way to guarantee that the therapeutic safety net for children is restored.”

In fact, the government decided to forgo its legal appeal of the Pediatric Rule in favor of legislation.

“Congress alone can speak clearly on the authority that FDA needs and the provisions that may be appropriate for drug manufacturers when they are required to carry out these tests,” said Tommy Thompson, secretary of the U.S. Department of Health and Human Services, in a statement. Administration officials said they would work with the committees in Congress to enact specific legislation rapidly.

AAP-backed legislation that was introduced in the last Congress will have to be re-introduced in the 108th Congress. The AAP wants legislation passed that mirrors the original Pediatric Rule.