-->Nov. 5, 2002 -- In a decision that could have far-reaching impl
Nov. 5, 2002 -- In a decision that could have far-reaching implications for children's medication, the U.S. District Court for the District of Columbia has struck down the FDA's Pediatric Rule, which requires drug companies to test safety and efficacy of their medications in children before the new drugs are approved for marketing.
The judge in the case ruled that the agency overstepped its bounds as a regulatory agency, saying that Congress had not intended to give the FDA power to require such testing.
In January, Congress passed a bill that gives incentives to drug companies to test their products in children, such as increased patent protections, but it does not require them to do so. In cases where the company chooses not to perform the tests, the bill also set aside money so that independent clinical trials can be conducted if the FDA believes that the drug's importance to children warrants it.
The bill ostensibly applies only to drugs that are already on the market, and that is perfectly fine with Richard Gorman, MD, a practicing pediatrician and chairman of the American Academy of Pediatrics' (AAP's) committee on drugs. The Pediatric Rule was designed to take that logic a step further and get drug companies thinking about safety and efficacy for children early on in the drug development process, around the same time they begin clinical trials for adults. "It made it the expectation that drugs in development would be studied in children at the same time as they would be studied in adults. ... They would be on equal footing in terms of safety and efficacy," Gorman tells WebMD.
Surprisingly, it isn't safety that Gorman is most concerned about. It's efficacy. After the FDA began enforcing the Pediatric Rule, some 30 drugs were tested in children, and researchers found that in nearly all of the cases, the doses doctors were giving to children were too low, making the drugs less effective. "Pediatricians were really taken aback," Gorman recalls.
In the case of a seizure medication called Neurontin, the optimal dose turned out to be 30% greater than what doctors were giving. "It's a clear example of how kids were being put at a therapeutic disadvantage," Gorman says.
But the Association of American Physicians and Surgeons disagrees. Along with the Competitive Enterprise Institute and Consumer Alert, it brought the lawsuit against the FDA. "It's senseless for the FDA to require pediatric testing for drugs that expressly disclaim any use on children," says Jane M. Orient, MD, executive director of the AAP, in a statement.
The plaintiffs argue that performing clinical trials on children is risky and unnecessary and that new drugs that are not expressly intended for children will be delayed by regulatory steps.
Gorman counters by saying that clinical trials include plenty of precautions designed to protect the safety of participants. And the Pediatric Rule has corporate safety valves built in: If a drug is unlikely to be prescribed for children (if it is a treatment for Alzheimer's disease, for example), the FDA will not require testing in children. Also, if the agency believes the testing in children may significantly delay the introduction of a useful new therapy, it can issue a deferral that clears the path for immediate approval as long as the company agrees to perform the tests.
The court decision is just the latest in a series of court cases that have chipped away at the FDA's authority. The agency has been increasingly aggressive in making regulatory demands on companies, and a sentiment is growing that it may be overstepping its bounds.
The American Academy of Pediatrics hopes that Congress resolves the question -- at least with respect to children -- by passing the Pediatric Rule into law. A bill sponsored by Sens. Ted Kennedy, D-Mass., and Hillary Clinton, D-N.Y., that would accomplish just that is in the Senate and may pass before the current session is over, says Gorman. Even if it does, however, it must still go through the House of Representatives and be signed by the president. "It's a long way from becoming a law."
