New Drug Rules Due on Pediatric Usage : Health: Manufacturers to be encouraged to provide dosing, other information on labels regarding how best to use the medicines for children.
The Food and Drug Administration is expected to propose new regulations today to encourage drug manufacturers to provide dosing and other information on medicine labels on how best to use the drugs in children, The Times has learned.
Currently, pediatricians are “in the dark” on how to use many medicines in children because labels fail to provide this information, said FDA Commissioner David A. Kessler. As a result, they must rely on experience or whatever is available in general medical literature, which is “an untenable situation,” he said.
“There’s a real problem out there and it puts pediatricians in a terrible bind,” Kessler said.
Kessler, a pediatrician and an attorney, is to announce the new policy in a speech today in San Francisco before the annual meeting of the American Academy of Pediatrics.
Drugs for diseases that solely afflict children are studied in children, but children are rarely included in studies for drugs that are used to treat diseases suffered by both children and adults.
Nevertheless, such drugs routinely have been used by pediatricians over the years to treat children, even in the absence of dosing and other information. This is legal, since the FDA does not have the authority to regulate the practice of medicine.
Existing regulations now require drug makers to conduct separate clinical trials for safety and efficacy in children if the drugs are to be approved for use in children--even though many of these drugs already have been in widespread pediatric use.
The proposed new rule would ease this requirement but only for certain kinds of drugs and diseases where the illness is known to take the same course in children as it does in adults--using antihistamines to treat hay fever, for example.
“In some cases, pediatric labeling may be based on adequate and well-controlled studies in adults, provided that additional information exists to show that the course of the disease--and the drugs’ effects--are sufficiently similar in children and adults to permit extrapolation from the adult data to children,” Kessler said in his prepared speech, a copy of which was obtained by The Times.
“There are instances where . . . separate controlled clinical trials in children are not necessary,” he said.
Kessler said that the final regulations will be issued after a 60- to 90-day period of public comment. He said that the new information should be on labels in six months to a year.
The prescription drug industry reacted with caution to the expected announcement.
Jeffrey C. Warren, a spokesman for the Pharmaceutical Manufacturers Assn., said that the group would not comment on the proposal until its members had an opportunity to study it. But he said that the PMA is working with the American Academy of Pediatrics “to identify existing medicines that need to be studied and approved for use in children.”
The pediatric community is expected to welcome the news.
“This is a significant advance for children,” said Dr. Cheston M. Berlin Jr., a pediatrician who is a member of the American Academy of Pediatrics committee on drugs. “We would treat it with great enthusiasm.”
Under the new proposal, drug makers will still have to do some work, including searching the medical literature for pediatric experience with a certain drug, FDA officials said. And they also will be required to conduct some research in children to determine whether the drugs show any adverse effects or toxicities, FDA officials said.
This is especially important because there can be cases of drug reactions that occur in children that are not seen in adults, Berlin said, adding that “children are not small adults, and one has to be cautious.”
Also, companies would be required to conduct studies in children to determine the proper dosage and how a drug is metabolized in children, agency officials said.
But manufacturers would not be required to start all over again from scratch to determine efficacy, FDA officials said.
Kessler emphasized that the policy will be limited only to those conditions where the adult experience is close to that of children’s. “There are many pediatric diseases where we cannot extrapolate from studies in adults,” he said.
In those instances, separate controlled clinical trials will continue to be required in children, he said.
Berlin, a pediatrician with Pennsylvania State University in Hershey, and others pointed out that more than 80% of the drugs listed in the Physician’s Desk Reference--a book that contains labeling information for all approved drugs--have no pediatric information.
Of the 21 drugs approved by the FDA in 1991 for diseases that afflict both children and adults, only five had information available for children’s use, said Dr. Ralph Kauffman, chair of the academy’s committee on drugs and a pediatrician at Wayne State University in Detroit. He called the FDA proposal “a very, very positive move.”
Kessler is also expected to urge companies to include more children in clinical trials of new drugs that are still being developed and have not yet been approved. In his speech, he notes that several AIDS drugs were studied in both children and adults and were approved simultaneously for use in both populations.
