FDA issues guidance on submission of pharmacogenomic data
The US Food and Drug Administration (FDA) has issued draft guidance to clarify which pharmacogenomic study findings and data should be included in regulatory submissions for investigational new drug applications (INDs), new drug applications (NDAs) and biologics license applications (BLAs).The guidance, issued on 16 March 2023, also provides recommendations to sponsors on the format and level of detail for reporting pharmacogenomic data submissions, which will vary based on how the genomic biomarkers are used and the potential risks. When finalized, this new guidance will replace final guidance for industry that FDA published in 2005.
“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and do not specifically describe the submission requirements for such data as a separate category,” FDA wrote in the draft guidance.
The draft guidance does not address when or how to submit pharmacogenomic results and data in an investigational device exemption (IDE) application or other device submissions.
IND submissions
IND sponsors must provide detailed reports to FDA when pharmacogenomic study findings support the use of a genomic biomarker in the design, conduct, or analysis of a planned clinical trial. “Detailed reports should be submitted in this context to facilitate the review of the biomarker’s clinical validity and ensure that trials making use of the biomarker are expected to meet stated objectives,” the agency wrote in the draft guidance.
These detailed reports should be submitted as part of the IND under the Previous Human Experience section, in meeting package, or in clinical study protocols or reports, according to the draft guidance.
Other pharmacogenomic study findings must be described in Annual Reports, such as studies that identify significant predictors of treatment response, findings related to pharmacokinetics, or results from completed clinical trials where the biomarker was integral to the design, conduct, or analysis and are relevant to understanding the drug’s actions.
Additionally, pharmacogenomic findings that identify predictors of adverse events with the potential to pose significant risks are required to be submitted in the IND Safety Report.
NDA, BLA submissions
NDA and BLA sponsors must generally submit the same types of pharmacogenomic data and studies as IND sponsors, though the level of detail required in submissions may vary. For example, if the genomic biomarker study results indicate substantial differences in responses related to efficacy across subgroups, a detailed report is required for NDA or BLA submissions. In contrast, a synopsis is required for IND submission.
NDA and BLA sponsors must also submit a detailed report with subject-level data when the genomic biomarker study results are proposed for inclusion in labeling. “This includes data pertaining to pharmacogenomic biomarkers that inform labeling, because the data pertain to selecting patients for clinical trials (whether enrollment is limited to or stratified by the biomarkers), determining dosing and administration, or informing drug-drug interactions (or lack thereof). Also, information not tied to specific clinical recommendations must be submitted if it relates to any narrative about the drug’s pharmacokinetic disposition or clinical trial populations in labeling,” the agency wrote.
Submission of pharmacogenomic reports and data can be discussed as part of pre-NDA and pre-BLA meetings.
Comments on the draft guidance can be submitted to regulations.gov and labeled as Docket No. FDA-2022-D-2856. The public comment period closes on 20 June 2023.
Pharmacogenomic Data Submissions Guidance
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