What's New
Announcement
Posted on 05.29.01
Renewal of the Pediatric Rule
By Sue Tolleson-Rinehart, PhD
Few people realize that doctors who write prescriptions for children are almost always venturing into the unknown territory of "off-label" therapeutic use.
Until recently, fully 80% of the drugs given to children had never been tested for their safety or effectiveness in children - in other words, they had not been "labeled" for use in children. The "off-label" use of drugs is perfectly legal; in fact, if doctors could not prescribe in this way, few drugs would be available to treat childhood illnesses.
There are understandable reasons for the lack of drugs labeled for pediatric use; for example, as a nation, we have resisted using children as subjects of medical research. But without controlled research on drugs in children and adolescents, doctors and parents are left guessing about the drugs' safety and effectiveness in this population.
The 1997 Food and Drug Modernization Act (FDAMA) began to change this. To accompany FDAMA, Senators Christopher Dodd and Mike DeWine sponsored a bill, the "Better Pharmaceuticals for Children Act," which offered an incentive to drug companies: if the companies tested their products for use in children, they would receive 6 additional months of patent protection in return.
Its passage led the Food and Drug Administration (FDA) to create the "Pediatric Rule" in 1998; the Rule required that studies be conducted:
- for drugs used to treat substantial numbers of children, or
- for drugs that could offer greater therapeutic benefits, and
- if the absence of adequate testing and labeling for children could pose significant risks.
One effect of the rule is very clear. Since 1998, researchers have conducted more than 300 pediatric drug studies. Only 11 such studies had been done in the 6 years before the Pediatric Rule.
The Rule takes the question of protecting children very seriously, and the FDA recently issued another interim rule that makes research safeguards even stronger.
The Pediatric Rule expires at the end of this year. Renewal of the original measure is pending. The Best Pharmaceuticals Act for Children has been passed by the House and Senate and is now awaiting the President's signature.