Addressing drug development for rare pediatric cancers, the newly released FDA draft guidance for industry calls for creating pediatric cohorts, embedding pediatric trials in adult trials, lowering the enrollment age for adolescents, conducting tissue-agnostic studies and master protocols.
Publishing the guidance is the next step in implementation of the RACE for Children Act, passed into law in August 2017. Companies are required to comply with the RACE Act requirements beginning in August 2020.
The guidance has links to lists of relevant molecular targets for which pediatric studies are required, and those that are non-relevant to pediatric cancers.
The guidance also explains how FDA will determine whether a molecular target is “substantially relevant to the growth or progression of pediatric cancer.”
Traditionally, drug development for pediatric cancers lagged because the requirements to study new cancer drugs in children have been based on whether the cancer occurs in children — and many adult cancers rarely occur in children, said FDA’s statement. New, targeted oncology drugs being developed for adult cancers may prove effective in the treatment of some cancers occurring primarily in pediatric patients with similar molecular targets.
The RACE for Children Act, enacted as a part of the Federal Food, Drug, and Cosmetic Act made by the FDA Reauthorization Act of 2017, requires drug developers to evaluate novel cancer medicines being developed for adults for treatment of pediatric cancers.