FDA Guidance Calls for Pediatric Data in Oncology Drug Submissions

A new FDA draft guidance for sponsors developing cancer drugs would require NDAs and BLAs submitted after Aug. 18, 2020, that present a new active ingredient to include reports of molecularly targeted pediatric cancer investigations unless a deferral or waiver is granted by the FDA.

Applicants that are subject to the pediatric study requirements must submit an initial pediatric study plan that references studies of pediatric-related factors, such as tolerability, dosing, pharmacokinetics and activity response.

The guidance includes two lists for sponsors to reference on molecular targets relevant to pediatric cancer. One list is of molecular targets that are relevant to the growth or progression of a pediatric cancer; the other list is for targets that are not relevant to pediatric cancer studies and would warrant a waiver.