Pharmacogenomic Data Submissions in Drug Development and Biomarker Classification
This guidance aims to facilitate scientific progress in pharmacogenomics and its use in drug development. It provides recommendations to sponsors regarding when to submit pharmacogenomic data during drug/biological product development, what format and content to provide, and how the data will be used in regulatory decision making.
Recommended Actions
- Determine if pharmacogenomic data qualifies as known valid, probable valid or exploratory biomarker
- For required submissions:
- Submit complete reports for data used in decision-making
- Submit abbreviated reports for known/probable valid biomarkers
- Include in protocols/informed consent if collecting samples
- For voluntary submissions:
- Use VGDS process and cover sheet
- Provide sufficient detail for FDA analysis
- Mark clearly as voluntary submission
- For labeling:
- Submit complete information if data will be included
- Consider co-development of diagnostic tests if needed
- Establish processes to:
- Document test validation status
- Track biomarker qualification
- Manage data quality and analysis
- Archive raw data and results
Key Considerations
Clinical testing
- Pharmacogenomic test results used for clinical trial decisions must be submitted (e.g., dose selection, entry criteria, safety monitoring)
- Known valid biomarkers for clinical outcomes must be submitted
- Probable valid biomarkers for human safety studies must be submitted
Non-clinical testing
- Animal study results using pharmacogenomic data for safety assessment must be submitted
- GLP compliance required for non-clinical studies intended to support safety findings
Software
- Standards for transmission, processing and storage of microarray data should be defined
- Bioinformatics tools and software used should be documented
Labelling
- Pharmacogenomic data intended for drug labeling must be submitted as complete reports
- Labels may include information about dose adjustment by genotype or potential side effects in certain genotypes
Safety
- Pharmacogenomic data relevant to safety assessment must be submitted
- Known valid biomarkers for safety outcomes must be submitted
- Probable valid biomarkers for safety should be submitted
Other considerations
- Voluntary submissions encouraged for exploratory/research data
- Format and content should allow FDA to independently analyze data
- Clear distinction between known valid, probable valid and exploratory biomarkers
- Sample collection for potential analysis must be noted in protocols and informed consent
Relevant Guidances
- Electronic Records and Electronic Signatures - Scope and Application
- Content of Premarket Submissions for Device Software Functions
- Dual Use Molecular Diagnostic Instruments with FDA-Approved and Non-Approved Functions
- In Vitro Companion Diagnostic Devices: Development, Review Process, and Labeling Requirements
Related references and norms
- 21 CFR Part 58: Good Laboratory Practice for Nonclinical Laboratory Studies
- 21 CFR Parts 312, 314, 601: Regulations for IND, NDA and BLA submissions
Original guidance
- Pharmacogenomic Data Submissions in Drug Development and Biomarker Classification
- HTML / PDF
- Issue date: 2005-03-01
- Last changed date: 2020-04-27
- Status: FINAL
- Official FDA topics: Medical Devices, Good Clinical Practice (GCP), Drugs, Biologics, Administrative / Procedural
- ReguVirta summary file ID: cb7d3672d714658bdc7a5d9504def852
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