New FDA Draft Guidance: Pharmacogenomic Data Submissions

When finalized, this draft guidance will replace a 2005 final guidance of the same name. 

The guidance can be downloaded here.

From the introduction:

This guidance is intended to facilitate progress in the field of pharmacogenomics and the use of pharmacogenomic data in drug2 development. This document is intended to clarify 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.

 

For the purposes of this guidance, the term pharmacogenomics is defined as the study of variations of DNA and RNA characteristics as related to drug response;3 DNA or RNA variations can be germline, somatic, or microbial. Pharmacogenomics does not refer to data resulting from proteomic, metabolomic, or other -omic studies, although similar considerations in this guidance could be applicable for determining whether to submit findings and data from such studies.

 

This draft guidance is not intended to cover pharmacogenomic data associated with an investigational device exemption (IDE) application.

 

A table is provided that defines whether a synopsis or full report is needed for an IND vs NDA/BLA.  However, in reading the text, there are exceptions for when the Agency would require a full report and not a synopsis (e.g., “… genomic biomarker studies that are related to pharmacokinetics (e.g., drug metabolizing enzyme gene variants) are recommended to be submitted as a synopsis. However, if those same genomic data are also the basis for patient dosing, subject-level data, and a full report rather than only a synopsis should be submitted.”).  Two sections discuss reports for IND and NDA/BLAs. Details are also provided on the format and sections for reports. Actual data formatting is referenced to two other guide documents. Lastly, the draft guidance notes where in an NDA/BLA the reports should be located.