Secretary of Health and Human Services (HHS) Tommy Thompson announced last month that HHS will pursue rapid passage of legislation giving the Food and Drug Administration authority to require pharmaceutical manufacturers to conduct appropriate pediatric clinical trials on drugs and biologics.
Citing the overwhelming lack of safety and efficacy information on psychotropic medications prescribed to those under age 18, officials of both APA and the American Academy of Child and Adolescent Psychiatry (AACAP) immediately gave strong support to the proposed legislation.
That announcement came as the government decided not to appeal a federal district court finding in October that the FDA lacks the legal authority to impose certain requirements for pediatric testing in children. The court’s action was a result of a legal challenge to regulations put in place in 1998, known as the "pediatric rule," which APA, AACAP, and the AMA supported (Psychiatric News, January 19, 2001).
"The fastest and most decisive route for establishing clear authority in this area is to work with Congress for new legislation," Thompson said at a press briefing last month. "Children are a special population that need to have access to drugs that can benefit them, and these drugs need to be properly tested for pediatric use, not prescribed and sold without testing. 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."
While the FDA had strongly defended the pediatric rule, FDA Commissioner Mark B. McClellan, M.D., noted that "continued litigation is likely to take years, and its outcome is uncertain. The better course now is to work with the committees in Congress and enact new, specific legislation rapidly."
"The more we learn about serious mental illness, the more we realize that many of these disorders begin in childhood," said Lois Flaherty, M.D., chair of APA’s Council on Children, Adolescents, and Their Families and a lecturer in psychiatry at Harvard Medical School, in an interview. "This increases the urgency that we have safe and effective treatments that can improve outcomes and prevent long-term disability."
APA Trustee-at-Large David Fassler, M.D., said, "We are well aware that children are not just little adults when it comes to psychopharmacology, yet much of current clinical practice is still based on assumptions extrapolated from adult studies."
Fassler is a child and adolescent psychiatrist in Burlington, Vt., and vice chair of the APA delegation to the AMA and an AACAP delegate in the AMA’s House of Delegates. On behalf of AACAP, he introduced a resolution at the AMA’s interim meeting in New Orleans calling on Congress to authorize the FDA to require pediatric testing (see end of story on page 50). APA strongly supported the resolution, and the AMA adopted it as new policy.
"We clearly need additional research focusing on the safety and efficacy of specific medications, including those already utilized in child and adolescent treatment," Fassler told Psychiatric News. "The proposed legislation will help promote such studies, and I am confident that APA and the academy will do everything possible to support its passage."
HHS Secretary Thompson outlined principles for the new legislation, saying it should include clear authority for the FDA to
• Consult with manufacturers early in the drug-development process regarding plans for pediatric testing.
• Require pediatric data to be provided by manufacturers at the time of the submission of a New Drug Application or a firm timeline for submission of pediatric data.
• Require studies of existing approved medications in pediatric populations.
• Create a new FDA Pediatric Advisory Committee.
Thompson also outlined steps that the Bush administration is taking in implementing the Best Pharmaceuticals for Children Act (BPCA), which President Bush signed into law last year.
The BPCA reauthorized an existing economic incentive—extended protection from patent expiration—for companies that conduct pediatric studies that are requested by the FDA and created a new mechanism for the FDA to pursue studies on products that have already lost patent protection. In the latter group, the BPCA provided that the FDA develop a list of drugs designated for federally funded testing.
In cooperation with the American Academy of Pediatrics, the National Institute of Child Health and Human Development is developing the full list. Thompson announced that the first two drugs for which requests to perform proposed pediatric clinical trials under BPCA are nitroprusside, for controlled reduction in blood pressure, and lorazepam, for sedation and pediatric and infant seizures. Both medications are commonly used in pediatric and neonatal intensive care units. The full list should be published by the end of the first quarter of 2003. ▪