FDA Will Defend Pediatric Drug Rule in Court

June 1, 2002

WASHINGTON-The Bush Administration has abandoned plans to suspend the Food and Drug Administration’s "pediatric rule," which requires pharmaceutical companies to study the use of drugs in pediatric patients when seeking new drug applications.

WASHINGTON—The Bush Administration has abandoned plans to suspend the Foodand Drug Administration’s "pediatric rule," which requirespharmaceutical companies to study the use of drugs in pediatric patients whenseeking new drug applications.

One month after the agency revealed its plans in court papers, Health and Human Services (HHS) Secretary Tommy G. Thompson pledged that FDA would continue to enforce the rule. Moreover, he announced a new federal initiative to ensure the safety and efficacy of drugs used to treat children and adolescents.

"We will enforce and improve the FDA’s pediatric rule as we simultaneously take additional steps made possible when President Bush signed new legislation to promote the development of pediatric drugs," Mr. Thompson said.

The pediatric rule, which became effective in April 1999, requires that companies assess the safety and efficacy of drugs and biologics in pediatric patients when seeking approval of a new active ingredient, indication, dosage form, dosing regimen, or route of administration. It also gave FDA the authority to require drug makers to develop a pediatric formulation to study in a particular pediatric group, if necessary.

However, the Association of American Physicians and Surgeons and two free-market advocacy groups—the Competitive Enterprise Institute and Consumer Alert—challenged the legality of the rule in a lawsuit filed in federal district court in 2000. The groups contend that FDA does not have the legal authority to impose or enforce the pediatric rule.

Strong Negative Reaction

In court papers filed in March, FDA said it would suspend the pediatric rule for up to 2 years as it evaluated whether the Best Pharmaceuticals for Children Act (BPCA), signed into law by President Bush on January 4, made the pediatric rule unnecessary.

The agency’s decision brought strong, negative reactions from several consumer groups and influential Democrats on Capital Hill, including Reps. John Dingell (D-Mich), Henry Waxman (D-Calif), and Sherrod Brown (D-Ohio).

In announcing the continued enforcement of the pediatric rule, Mr. Thompson said that FDA would continue to defend its legal authority under the rule in court and that it would not pursue a delay in the lawsuit, which it had sought in March. FDA is seeking comments from the public on additional steps it plans to take to ensure the adequate study of medications prescribed for pediatric use, as required by the new BPCA law.

According to HHS, the National Institutes of Health will use new contracting procedures authorized in the BPCA to request proposals later this year for studies of certain drugs that pharmaceutical companies have chosen not to do.

The initial contracts are planned for fiscal year 2003, which begins October 1. The BPCA permits funding for such studies by the Foundation for the National Institutes of Health, a tax-exempt organization chartered by Congress through which the public can donate money to support NIH research. NIH will also provide $7 million this fiscal year to fund additional research at the pediatric pharmacology research units it supports.