WASHINGTON — A pediatrician who wants to prescribe a drug for a child frequently faces a confusing and potentially dangerous dilemma.
More often than not, there is little or no information available about the correct dosage or possible adverse side effects of that drug for children.
As a result, children often either do not get access to the drugs that they need--because the medicines are not officially approved for use in that age group--or they face the risk that they could be hurt by a drug that is administered improperly.
"In many instances, you're flying by the seat of your pants," said David A. Kessler, commissioner of the Food and Drug Administration, who was a practicing pediatrician before assuming the federal post. "It's not the way to practice medicine."
So the FDA plans to propose new rules today designed to alleviate the problem by allowing drug companies, in certain situations, to extrapolate guidelines from adult studies and apply them for use in children. In doing so, they would be required to revise labels and other information available to pediatricians on how the drugs should be used in children.
The proposal amends a 1979 regulation that requires full clinical trials in children before a new drug can be approved for pediatric use. Many drug companies typically don't conduct tests of new drugs in children because their main market for a drug is usually adults, and they may be worried about testing on youngsters.
Thus, most therapies end up being approved for adults only. Yet many doctors still routinely prescribe them for children because the drugs have come into standard practice through physician experience--trial and error.
"Right now when your pediatrician prescribes a drug, chances are that the drug companies haven't done the studies in children," Kessler said in an interview. "The data are not there. An enormously high number of drugs are being prescribed for kids where pediatricians do not have this information."
The American Academy of Pediatrics estimates that about 80% of currently approved drugs have been studied and licensed for use in adults, with virtually no information available on pediatric use.
Yet such drugs routinely are being used in children to treat asthma, cancer, blood diseases, lupus, psychiatric and behavioral disorders, as well as routine ailments.
Equally troubling, some drugs that are known through practice to work in children for one condition actually are licensed only for use in adults for an entirely different ailment. This could mean that a doctor unfamiliar with standard practice either might not know of its value in treating a particular pediatric disorder or simply might be reluctant to prescribe it at all.
For example, "there is one drug that has only one indication--treatment of hypertension in adults--that is used extensively for behavioral problems in children," said Dr. Cheston Berlin Jr., who heads a special committee on drugs for the American Academy of Pediatrics.
"The profession tries very hard to be as devoted to children as it can," Berlin said. "We try very hard to provide patients with the right therapy, but we need more help in this area."
Kessler said some of the impetus for the new rules came from a meeting he had three years ago with Elizabeth Glaser, founder of the Los Angeles-based Pediatric AIDS Foundation.
Glaser, who recently died of AIDS, made a convincing case that children with AIDS deserved as much access to experimental AIDS drugs as adults, Kessler said.
"We realized something needed to be done, because drugs needed to be developed and studied in kids," Kessler said.
The changes are expected to be quickly endorsed by the pediatric medical community.