The law requires pharmaceutical companies to assess safety, efficacy and appropriate dosing of agents in children with the intent of including such information in a product label. The law is most relevant for childhood diseases where data from adult studies can be extrapolated for children. It can be enforced when an agent can be used in "substantial" numbers of pediatric patients and when there is a "meaningful therapeutic benefit" (a significant improvement over existing therapies). Further, if the course of the disease and the effects of the agent are sufficiently similar in adults and children, the law permits the FDA to conclude that a therapy is effective for children based on data from studies in adults.

Deferral and waiver options are included in the new law so that the approval of drugs for adult use is not delayed while pediatric assessments are conducted or are deemed impractical or unsafe. FDA may grant a permanent waiver of pediatric testing if an agent may not have a meaningful therapeutic benefit.

The value of the law for childhood cancer patients remains uncertain at this time. There are about 12,400 new diagnoses of cancer each year, thereby not meeting the "substantial numbers" criterion. In addition, the similarities of childhood cancers and adult cancers are a matter of scientific debate and have been discussed at meetings of FDA’s Pediatric Subcommittee of the Oncologic Drugs Advisory Committee. The law’s emphasis on requiring pediatric information in a product label is also likely to have little immediate impact on childhood cancer treatment. Most children are treated in the context of pediatric oncology clinical trials with agents approved for adults but used off label in children.

Both the new law and the six-month marketing exclusivity provision (codified in the Best Pharmaceuticals for Children Act) may encourage more companies to permit testing of new anti-cancer agents in children. Many families and pediatric oncologists are hopeful that the pediatric rule and pediatric exclusivity together will enable children with cancer more ready access to the new generation of more effective and less toxic drugs and biologics.

Children's Cause for Cancer Advocacy Position

Children's Cause for Cancer Advocacy supports policies that encourage companies to evaluate promising anticancer therapies in children prior to their approval for adult use and urges greater efficiency in FDA's review of new agents.