Congress passed the "Best Pharmaceuticals for Children Act" in 2001 to renew an incentive for pharmaceutical companies to carry out additional pediatric research. Over the past few years, this incentive has encouraged companies to increase the number of pediatric studies being conducted.

Children's Cause for Cancer Advocacy worked with Congress to ensure that the legislation addresses the needs of pediatric cancer research more directly. The bill contains provisions to broaden the mission and membership of the Food and Drug Administration's (FDA) Pediatric Subcommittee of the Oncologic Drugs Advisory Committee (ODAC). This allows greater consensus in the pediatric oncology community about priorities for testing new therapies for the treatment of childhood cancer. The Pediatric ODAC now includes representatives of the National Cancer Institute, FDA, Children's Oncology Group, pediatric oncology consortia, and others.

The legislation also includes language for the Director of the National Cancer Institute to expand, intensify, and coordinate work on preclinical models of childhood cancer. Valid and reliable preclinical models of childhood cancer are critical so that new treatments can be evaluated prior to their being tested in children. No such network or coordinated activity currently exists.

Children's Cause for Cancer Advocacy will continue working with others in the cancer community to ensure that pediatric oncology research is advanced by this legislation.

Background
Drugs that treat children with cancer are typically developed for adult use before they are evaluated for safety and efficacy in children. Pediatric oncologists typically rely on peer-reviewed studies to recommend treatments for pediatric cancer rather than on therapies approved by FDA for use in children. Historically, companies have been reluctant to develop or test drugs in children because of both the risk and the inherent cost of this research.

Passage of the Food and Drug Administration Modernization Act (FDAMA) in 1997 created an important financial incentive for pharmaceutical companies to invest in pediatric studies on approved or about to be approved drugs. This incentive offered 6 months of market exclusivity (protection from generic competition) on all uses of a drug, including adult uses, if a sponsor conducted studies that "may produce health benefits in the pediatric population."

Children's Cause for Cancer Advocacy Position
The 6-month incentive has less value for companies when a drug is new than when it is about to lose its market exclusivity. The incentive therefore has not been as effective a stimulus for companies to conduct studies in pediatric cancer as it has been for other diseases. Despite this, Children's Cause for Cancer Advocacy, along with other groups concerned with childhood cancer, has actively supported its reauthorization. The incentive is a significant tool for changing companies' perspectives about the importance of pediatric clinical studies.

To learn more about FDAMA, FDA interpretation, and pediatric drug approval, visit their web site.