U.S. flag An official website of the United States government

U.S. Food and Drug Administration
  1. Home
  2. Regulatory Information
  3. Search for FDA Guidance Documents
  4. FDARA Implementation Guidance for Pediatric Studies of Molecularly Targeted Oncology Drugs: Amendments to Sec. 505B of the FD&C Act
  1. Search for FDA Guidance Documents

GUIDANCE DOCUMENT

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Guidance for Pediatric Studies of Molecularly Targeted Oncology Drugs: Amendments to Sec. 505B of the FD&C Act Draft Guidance for Industry December 2019

Download the Draft Guidance Document Read the Federal Register Notice
Draft

Not for implementation. Contains non-binding recommendations.

Docket Number:
FDA-2019-D-4751
Issued by:
Guidance Issuing Office
Office of Medical Products and Tobacco, Oncology Center of Excellence
Office of Medical Products and Tobacco, Center for Drug Evaluation and Research
Office of Medical Products and Tobacco, 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).

Submit Comments

Submit Comments Online

You can submit online or written comments on any guidance at any time (see 21 CFR 10.115(g)(5))

If unable to submit comments online, please mail written comments to:

Dockets Management
Food and Drug Administration
5630 Fishers Lane, Rm 1061
Rockville, MD 20852

All written comments should be identified with this document's docket number: FDA-2019-D-4751.

Questions?

Contact Point
Human Drug Information
Office of Communications
10001 New Hampshire Ave
Hillandale Building, 4th Fl
Silver Spring, MD 20993
druginfo@fda.hhs.gov
(855) 543-3784 Toll Free
(301) 796-3400
Office of Communication, Outreach and Development (OCOD)
Center for Biologics Evaluation and Research
Food and Drug Administration
10903 New Hampshire Ave WO71-3128
Silver Spring, MD 20993-0002
ocod@fda.hhs.gov
(800) 835-4709
(240) 402-8010