FDA News: Issue 8-1 March 2023

Author: [AUTHOR] Published on 3/31/2023 6:00:00 AM

FDA Announces Availability of a Draft Guidance on Pharmacogenomic Data Submissions

On March 20, 2023, the US Food and Drug Administration (FDA) announced the availability of a draft guidance for industry entitled Pharmacogenomic Data Submissions. When finalized, this guidance will replace the 2005 final guidance of the same name. This guidance is intended to facilitate progress in the field of pharmacogenomics and the use of pharmacogenomic data in drug development. This document clarifies the contexts in which pharmacogenomic study findings and data must be included in submissions related to investigational new drug applications (INDs), new drug applications (NDAs), and biologics license applications (BLAs) based on the FDA’s regulations. In addition, this document provides recommendations to sponsors and applicants on the format and content of the pharmacogenomic data submissions.

Pharmacogenomic studies have the potential to help identify sources of interindividual variability in drug exposure or response (both effectiveness and toxicity), making it possible to optimize therapy for individuals. Currently, many pharmacogenomic biomarkers with well-accepted mechanistic and clinical significance are being integrated into drug development (e.g., enriched clinical trial designs) and clinical practice.

Sponsors and applicants submitting or holding INDs, NDAs, or BLAs are subject to FDA requirements for submitting data to the FDA that are relevant to drug safety and effectiveness. However, the regulations were developed before the advent of widespread animal or human genetic testing (e.g., high-throughput DNA sequencing) or gene expression testing and do not specifically describe the submission requirements for such data as a separate category.  

This guidance, when final, will constitute the FDA's current thinking about pharmacogenomic study results and the associated data required to be submitted in IND, NDA, or BLA submissions, as well as the FDA’s recommendations as to the level of detail and format for reporting. This guidance is intended to facilitate the generation and use of pharmacogenomic data during drug development, and the policies outlined in this guidance are intended to advance the field in a manner that will benefit both drug development programs and public health.

The “Pharmacogenomic Data Submissions” guidance is available at: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/pharmacogenomic-data-submissions-0. Please refer to the guidance for more details. FDA is publishing this draft guidance to collect additional public comments. You may submit your comments regarding the draft guidance to the docket (Docket No. FDA-2022-D-2856) 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.

The Office of Clinical Pharmacology (OCP) is pleased to offer the e-mail subscription service Clinical Pharmacology Corner. This is a free service from FDA to provide occasional updates from OCP regarding newly approved therapies, new regulatory and scholarly publications, upcoming events and other items of interest. Subscribe today or click the button below select Clinical Pharmacology Corner under Drugs.

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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