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Genomics
at FDA
Submission of Pharmacogenomic Data Decision Tree
Decision Tree also available as PDF
Submission
of Pharmacogenomics Data to an IND
Pharmacogenomic data
must be submitted to the IND under
CFR 312.23 if ANY of the
following apply:
1. The test
results are used for making decisions pertaining to a
specific clinical trial, or in a animal trial used to
support safety (e.g., the results will affect dose
selection, entry criteria into a clinical trial safety
monitoring, or subject stratification).
2. A sponsor
is using the test results to support scientific arguments
pertaining to, for example, the pharmacologic mechanism of
action, the selection of drug dosing, or the safety and
effectiveness of a drug.
3. The test results constitute a known, or
probable, valid biomarker for physiologic, pathophysiologic,
pharmacologic, toxicologic, or clinical states or outcomes
in humans, or is a known valid biomarker for a safety
outcome in animal studies. If the information on the
biomarker (example, human CYP2D6 status) is not
being used for purposes 1 or 2 above, the information can be
submitted to the IND as an abbreviated report.
Submission to an IND is NOT needed, but voluntary
submission is encouraged (i.e., information does not meet
the criteria of
CFR 312.23) if:
4. Information is from
exploratory studies or is research data, such as from
general gene expression analyses in cells/animals/humans, or
single-nucleotide polymorphism (SNP) analysis of trial
participants.
5. Information consists
of results from test systems where the validity of the
biomarker is not established.
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Submission of Pharmacogenomics Data to a New NDA,
BLA, or Supplement
1.
The sponsor will use the test results in the drug label
or as part of the scientific database being used to support
approval as complete submissions (not in the form of an
abbreviated report, synopsis, or VGDS), including information
about test procedures and complete data, in the relevant
sections of the NDA or BLA.
If the pharmacogenomic test is already approved by the
FDA or is the subject of an application filed with the Agency,
information on the test itself can be provided by cross
reference.
The following examples would fit
this category:
-
–pharmacogenomic
test results that are being used to support scientific
arguments made by the sponsor about drug dosing, safety,
patient selection, or effectiveness;
-
–pharmacogenomic
test results that the sponsor proposes to describe in the drug
label ; or
-
–pharmacogenomic
tests that are essential to achieving the dosing, safety, or
effectiveness described in the drug label.
2. The test results are known valid biomarkers for
physiologic, pathophysiologic, pharmacologic, toxicologic, or
clinical states or outcomes in the relevant species, but the
sponsor is not relying on or mentioning this in the label.
Submit to the Agency as an abbreviated report (not as a
synopsis or VGDS). If a
pharmacogenomic test of this type was conducted as part of a
larger overall study, the reporting of the pharmacogenomic test
results can be incorporated into the larger study report.
3.
3.3.
The test results represent
probable valid biomarkers for physiologic, pathophysiologic,
pharmacologic, toxicologic, or clinical states or outcomes in
the relevant species.
Submit to the Agency as an abbreviated report.
If pharmacogenomic testing of this type was conducted
as part of a larger study, the abbreviated report can be
appended to the report of the overall study.
44.Information from general exploratory or research studies,
such as broad gene expression screening, collection of sera or
tissue samples, or results of pharmacogenomic tests that are not
known or probable valid biomarkers to the NDA or BLA are not
required to be submitted.
Because the Agency does not view these studies as germane
in determining the safety or effectiveness of a drug, the
submission requirements in
CFR 314.50 or
601.2 will be satisfied
by the submission of a synopsis of the study.
However, the Agency encourages the voluntary submission
of the data from the study in a VGDS submitted to the NDA or BLA.
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Submission of Pharmacogenomics Data to an Approved
NDA, BLA, or Supplement
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Date created: March 22, 2005 |
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