FDA News: Issue 16-1, September 2022

Author: [AUTHOR] Published on 9/9/2022 6:00:00 AM

FDA Announces Availability of a Revised Draft Guidance on General Clinical Pharmacology Considerations for Pediatric Studies of Drugs, Including Biological Products

On September 8, 2022, the US Food and Drug Administration (FDA) announced the availability of a revised draft guidance for industry entitled General Clinical Pharmacology Considerations for Pediatric Studies of Drugs, Including Biological Products, replacing the December 2014 draft guidance. This guidance assists sponsors of investigational new drug applications (INDs) and applicants of new drug applications (NDAs) under section 505 of the Federal Food, Drug, and Cosmetic Act (the FD&C Act), biologics license applications (BLAs) under section 351(a) of the Public Health Service Act (PHS Act), and supplements to such applications who are planning to conduct clinical studies in pediatric populations. In addition, this guidance assists clinical investigators in the design and planning of, and Institutional Review Boards (IRBs) in the assessment of, clinical studies in pediatric populations.  > read more

Effectiveness, safety, or dose-finding studies in pediatric populations involve gathering clinical pharmacology information, such as information regarding a product’s pharmacokinetics and pharmacodynamics, to inform dose selection and individualization. This guidance addresses general clinical pharmacology considerations for conducting studies so that the dosing and safety information for drugs in pediatric populations can be sufficiently characterized, leading to well-designed trials to evaluate effectiveness. This guidance focuses on the clinical pharmacology information (e.g., exposure-response, pharmacokinetics, and pharmacodynamics) that supports findings of effectiveness and safety and helps identify appropriate doses in pediatric populations. 

This guidance also describes how quantitative approaches (i.e., pharmacometrics) can use disease and exposure-response knowledge from relevant prior clinical studies to help design and evaluate future pediatric studies.  

The “General Clinical Pharmacology Considerations for Pediatric Studies of Drugs, Including Biological Products” guidance is available at: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/general-clinical-pharmacology-considerations-pediatric-studies-drugs-including-biological-products. Please refer to the guidance for more details. FDA is publishing this revised draft guidance to collect additional public comments. You may submit your comments regarding the revised draft guidance to the docket (Docket No. FDA-2013-D-1275) available at https://www.regulations.gov up to 90 days following publication in the FEDERAL REGISTER. This guidance, when finalized, will represent the current thinking of the FDA on this topic. It does not establish any rights for any person and is not binding on FDA or the public. Your comments do make a difference and can impact the outcomes of FDA regulatory policy. Share your knowledge and experience and make your voice count.

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We always welcome your thoughts regarding the format, content, and utility of this communication. Comments may be sent via email to ocp@fda.hhs.gov.

This communication was prepared by Office of Clinical Pharmacology, Office of Translational Sciences, CDER, FDA.

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