FDARA Implementation Guidance for Pediatric Studies of Molecularly Targeted Oncology Drugs: Amendments to Sec. 505B of the FD&C Act Draft Guidance for Industry December 2019

GUIDANCE DOCUMENT

• Docket Number: FDA-2019-D-4751
• Issued by: Oncology Center of Excellence; Center for Drug Evaluation and Research; Center for Biologics Evaluation and Research

The purpose of this guidance is to provide the pharmaceutical industry, clinical investigators, and institutional review boards (IRBs) with information to facilitate pediatric studies of molecularly targeted (also referred to as “targeted” in this guidance) oncology drugs. This guidance addresses early planning for pediatric evaluation of certain molecularly targeted oncology drugs for which original New Drug Applications (NDAs) and Biologics License Applications (BLAs) are expected to be submitted to the FDA on or after August 18, 2020 in accordance with section 505B of the Federal Food, Drug, and Cosmetic Act (FD&C Act) (also referred to as the Pediatric Research Equity Act, or PREA) as amended by the FDA Reauthorization Act of 2017 23 (FDARA).

This guidance addresses the implementation of amendments made by FDARA section 504 to section 505B of the FD&C Act regarding molecularly targeted oncology drugs. This guidance does not contain a complete discussion of general requirements for development of drugs for pediatric use under PREA or section 505A of the FD&C Act (also referred to as the Best Pharmaceuticals for Children Act or BPCA).

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