An undisputed crisis exists due to the absence of adequate labeling for pediatric oncology drug products. Almost all drugs used to treat pediatric oncology patients were approved over decades ago and knowledge of pediatric cancer has increased logarithmically since their initial approvals. Further, the drugs were never tested in pediatric patients prior to New Drug Application (“NDA”) approval by the Food and Drug Administration (“FDA”). The drugs were tested in adult patients who suffered from the same basic disease under 1960’s and 1970’s standards. At that time, the risks associated with testing in children were considered too high to ethically permit. Children absorb, distribute, metabolize, and excrete these highly toxic drugs differently than adults. Almost all are parenterally administered. Most were approved before the enactment of the Waxman-Hatch Act in 1984[1], which formalized the generic drug approval process.

With its focus on new targets for generic drugs, the formalization of the genericization process inadvertently created a practical, but not legal, impediment to private sector pediatric oncology research for these older chemotherapeutic agents. As a result, their labeling is deficient, by contemporary standards, for pediatric patients. These agents remain valuable for treating this vulnerable patient population.

Their archaic labeling, however, creates an information deficit for patients, physicians, and payors. This deficit occurs at a time of limited resources for most health care providers, and optimal value therapy is essential. This article sets forth an administrative procedure for FDA to update the labeling of these oncology drugs for pediatric use. This administrative process follows the long line of such administrative processes created by FDA to ensure that drug labeling remains up to date in terms of safety and effectiveness.