FDA Issues Guidance on Pediatric Studies of Molecularly Targeted Oncology Drugs

FDA published final guidance on May 24, 2021 for the early planning of pediatric evaluation of molecularly targeted oncology drugs as required by section 505B(a)(1)(B) of the Federal Food, Drug, & Cosmetic Act (FD&C), as amended by the FDA Reauthorization Act of 2017 (FDARA). The guidance document addresses molecularly targeted oncology drugs where original New Drug Applications (NDAs) and Biologics License Applications (BLAs) are submitted to FDA. FDA expects early pediatric evaluation of these drugs to accelerate the creation of a pediatric development plan and development of pediatric oncology drugs.

Specifically, the document discusses regulatory considerations such as molecular target, determination of relevance, target lists, recommended studies, and rare cancers. It also addresses international collaboration in development of these drugs.