As the latest initiative in his crusade to improve health care for children, President Clinton on Wednesday ordered pharmaceutical companies to test most new drugs for safety and proper doses for children.
"The executive action that I take today simply is designed to ensure that parents and pediatricians have the safety information they need," Clinton said.
Despite abundant evidence that drugs affect children differently than adults, more than half of the drugs commonly prescribed to children have not been tested on children and lack special dosage information for them, according to the FDA.
Pediatricians often must rely on educated guesses when prescribing such drugs to treat their young patients. Because of the lack of information, doctors in many cases simply refrain from prescribing the drugs to children, possibly denying them the healing that older patients receive.
Officials say that pediatric testing poses a problem because parents are usually unwilling to give permission to allow their children to participate.
Under the proposed regulation, which will take effect after a 90-day period for public comments, manufacturers of prescription drugs likely to be used by children will be required to conduct studies on them and put information on drug labels.
The FDA estimates the regulation will cost drug companies $20 million per year. The FDA estimates that about 12 new drugs per year will need to be studied for the effects on children. Drug companies have expressed lukewarm support of the initiative.
"In view of all the ongoing testing and studying of medicines in children, we question whether a government mandate is needed," said Alan Holmer, president of Pharmaceutical Research and Manufacturers of America, which represents the leading companies in the industry. "However, we are committed to working with the president and others in the administration on a collaborative and constructive basis regarding their proposed regulation to advance the goal of better medicine for children."
Some drugs already on the market, which often are prescribed for children but lack dosage information for them, also would have to be tested for children under the proposed regulation. Decisions on which of the existing drugs must be tested will be made on a case-by-case basis, said William Schultz, deputy FDA commissioner.
The FDA compiled a list of the 10 drugs most widely prescribed for children but not tested for them. All told, these drugs were prescribed 5 million times in one year for children in age groups for which the labels carried a disclaimer or lacked adequate information on usage.
* Alupent for treatment of asthma, which was prescribed 184,000 times to patients younger than 6.
* Ritalin for treatment of attention deficit disorder, prescribed 226,000 times to children under 6.
* Ampicillin injections for treatment of infection, prescribed 639,000 times to patients younger than 12.
* Auralgan otic solution for treatment of ear pain, prescribed 600,000 times to patients under 16.
* Prozac for treatment of depression, prescribed 349,000 times to patients under 16.
Susan DeLaurentis, co-founder of the Santa Monica-based Pediatric AIDS Foundation, a not-for-profit organization dedicated to research for children with HIV, said most Americans do not realize that doctors are prescribing medicine to children without adequate studies to test safety and dosages for them.
"The public does not understand that this is a problem to begin with," said DeLaurentis, whose organization has pushed hard to get the FDA to require pharmaceutical companies to test for children.
Since only half of the HIV drugs for adults have been approved for children--including only two of the four available protease inhibitors--children with HIV are missing opportunities to extend and improve their lives, she said.
"Any child who is not afforded a lifesaving treatment is going to suffer for it," DeLaurentis said.
Elizabeth Glaser, DeLaurentis' best friend and co-founder of the organization, and Glaser's daughter, Ariel, both died of AIDS. According to DeLaurentis, the mother's health improved rapidly when she started taking AZT. But when she asked the doctor to give the drug to her daughter, whose health was declining rapidly, the doctor said it was impossible because it was not yet clear what dosages of the toxic medicine were appropriate for children.
When Ariel, who was 7 at the time, started taking AZT in 1988, she was able within weeks to talk, walk and play for the first time in months. But her immune system was so weak that she died later that year. Her mother lived until 1994.
"I believe that if Ariel had gotten [AZT] earlier, it could have had a remarkable impact on her life," said DeLaurentis.
During the White House ceremony, First Lady Hillary Rodham Clinton, who knew the Glasers, said she recalled the "heartbreaking problem" Elizabeth Glaser faced when she learned that her daughter could not take the drug that was so dramatically improving her own health.
"Too many medical treatments that are saving adult lives are slow in finding their way to children," Mrs. Clinton said.
The new FDA regulations will change this, she added.
"The plan the president is announcing today will see to it that no child or parent will have to endure this kind of agonizing uncertainty," she added.
The pharmaceutical industry reacted to the news of the mandatory regulations by stressing that it had already responded to the administration's 1994 call that it undertake the effort voluntarily.
Holmer said a 1997 survey found 146 new medicines in development for children, including 36 for cancer and nine for AIDS. All 146 had been tested for pediatric use.