Over the past 20 years, the Food and Drug Administration has initially approved only two drugs for pediatric cancer — Clofarabine and Erwinaze.  And, only Clofarabine is an anti-cancer agent.

The FDA is uniquely positioned to promote pediatric cancer drug development and support the role of pediatric cancer researchers.

Pediatric drug developers should have the opportunity to set priorities for research and development of pediatric cancer drugs.

Pediatric cancer researchers should have access to unapproved drugs – and unapproved drugs that have failed adult trials — when there is evidence that they merit for study in children.

The most qualified FDA reviewers with pediatric training and experience should be at the table reviewing pediatric new drug applications.

Trial designs requested by the FDA for pediatric cancer New Drug Applications should meet the rigors of FDA regulatory science and yet be as flexible as possible to reflect the particular circumstances of the pediatric cancer drug development— that families of terminally ill children have a very high tolerance for potential toxicities; that kids and families placed on randomized arms of trials know who they are, are devastated and often opt out; that it’s cruel and unethical to ask a dying child to suffer toxicities on a randomized arm when randomization could be avoided; that it’s difficult to accrue patients on large randomized trials; and that endpoint analysis is messy and there is no “gold standard,” including overall survival.

Kids v Cancer also works with pediatric cancer researchers to marry advances in information technology and explore new ways to pioneer personalized medicine strategies to bring the most effective and tailored therapies to children with cancer.