Congress gives FDA authority to require drug testing in children

Some physicians call the legislation "misguided" and question the ethics of recruiting pediatric patients.

By Tanya Albert, amednews staff. Dec. 8, 2003.

Pediatricians say a newly passed bill ensures that they will finally get the information they need to make the best possible decisions when they prescribe medications to children.

The Pediatric Research Equity Act, which President Bush is expected to sign, gives the Food and Drug Administration the authority to require pharmaceutical companies to test specific medications for use in children. The House passed the American Academy of Pediatrics-backed bill last month, after unanimous Senate approval in July.

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Drug companies were not required to test prescription medications on children until the FDA adopted the Pediatric Rule in 1998. Many physicians were happy to finally see scientific data on the appropriate dosages for medications used in children, rather than relying on educated guesses derived from studies done in adults. In the vast majority of cases, the initial phases of a study of a new medication were done in adults to ensure it was relatively safe before testing was done in children, according to the AAP.

But in October 2002, a federal court struck down the rule on the grounds that the FDA didn't have the authority to require companies to test medications in children. Since then, more than 110 new medications and biologics have gone on the market without testing in children, the AAP said.

The Pediatric Research Equity Act would give the FDA the authority it needs to require testing in children and would thus allow the agency to restore the Pediatric Rule. For pediatricians, that would bring a sense of relief by eliminating much of the guess work when prescribing medication to children.

"When a drug hits the market, we will be able to prescribe as effectively as we would for an adult," said Richard Gorman, MD, an Ellicott City, Md., pediatrician who serves on the AAP's Committee on Drugs.

Since October 2002, more than 110 drugs and biologics have gone on the market without testing in children.

Kansas City, Mo., clinical pharmacologist Ralph Kauffman, MD, said the new knowledge would prevent physicians from potentially unknowingly undertreating or overtreating children.

"This is a big public health care issue, and [the legislation] will have a major impact on safety and effectiveness of medication in children," he said.

Studies of children who received medications based on guidelines derived from adult studies found that the dosage children received sometimes wasn't proper, Dr. Gorman said. Other studies showed that drugs sometimes produce side effects in children but not in adults, and it is hard to predict common side effects in children.

The Pediatric Rule will serve in conjunction with the Best Pharmaceuticals for Children Act, which gives financial incentives to pharmaceutical manufacturers that voluntarily test medication in children. The Pediatric Rule covers medicines that don't fall under that law.

"Every parent can appreciate the importance of this legislation," said Rep Anna G. Eshoo (D, Calif.), one of the sponsors of the House legislation.

"It will help provide doctors and parents the information they need to give children the best and safest medical treatment possible, without any guess work."

Rep. Deborah Pryce (R, Ohio), another bill sponsor, agreed. "Many people wrongly assume that children's bodies are smaller versions of adult bodies," she said. "But simply reducing the dosage of medicine for the treatment of a child is not always effective and is definitely not always safe."

Opposition to testing

Not everyone agrees that testing in children is the proper thing to do.

The Assn. of American Physicians and Surgeons and two public interest groups, Consumer Alert and the Competitive Enterprise Institute, filed the lawsuit in U.S. District Court for the District of Columbia that halted the testing.

The AAPS opposes the new legislation. The current guidelines are a better alternative than testing pharmaceuticals in children, the group says.

"It's a very misguided law," said Jane M. Orient, MD, AAPS executive director. "The net effect will hurt children more than help them."

Adults should bear the brunt of the testing, and ethical difficulties arise with recruiting children for testing, she said.

"Doses are under physician judgment, and there are guidelines for physicians prescribing for children," Dr. Orient said. "Would you want your child to be part of a study?"

But Dr. Kauffman disagrees.

"It is even more unethical to subject children to untried and unstudied treatments," he said. "Not having studies also puts the treating physician in a difficult position -- deny potentially beneficial medication or give them a drug with unknown side effects in children."

The legislation appears to have Bush administration support.

The FDA has long recognized a need to test in order to gather information on how medications "actually work in children" and is pleased Congress passed the law, Health and Human Services Secretary Tommy G. Thompson and FDA Commissioner Mark B. McClellan, MD, PhD, said in a joint statement.

"Access to drugs that have been properly tested for pediatric use will give us all more confidence that the drugs we use to treat our children will be safe and will work as expected," they said.

ADDITIONAL INFORMATION:

Weblink

Thomas, the federal legislative information service, for bill summary, status and full text of the Pediatric Research Equity Act of 2003, (S 650 or HR 2857) (thomas.loc.gov)

American Academy of Pediatrics press release on passage of the Pediatric Research Equity Act of 2003 (www.aap.org/advocacy/washing/ped_rule_passes.htm)