Review of the FDA Pharmacogenomic Guidance for Industry:
Role of StaRT-PCR™ in Increasing Value of Pharmacogenomic Data

Summary
The promise of pharmacogenomics in facilitating development of new drugs and making individualized medicine a reality will be realized, in part, through development of known valid biomarkers comprising transcript abundance values. Gene Express, Inc. plays an integral role in this effort through its proprietary technology, StaRT-PCR. StaRT-PCR performs this role by generating transcript abundance data that are of sufficient quality to be valid biomarkers. See the Role of StaRT-PCR in Increasing Value of Pharmacogenomic Data. A power point presentation summarizing the final 2005 FDA Guidance for Pharmacogenomic Data Submissions is available. As stated in the FDA Guidance for Industry Pharmacogenomic Data Submissions:

For the purposes of this [FDA] guidance, a pharmacogenomic test result may be considered a valid biomarker if
(1) it is measured in an analytical test system with well-established performance characteristics and
(2) there is an established scientific framework or body of evidence that elucidates the physiologic, pharmacologic, toxicologic or clinical significance of the test results.

StaRT-PCR is a properly controlled transcript abundance measurement method with well-established performance characteristics that is suitable to qualify potential or probable biomarkers. Specific characteristics important for this purpose include:

1. An established scientific framework and a body of evidence supporting the excellent performance characteristics of the method
2. Generation of standardized, sensitive, biomarker data for drug development that is reproducible across institutions.

Following are specific applications for which StaRT-PCR is used by the industry to generate data that meet the criteria and goals described in the FDA pharmacogenomics submission guidance:

1. Generating transcript abundance data that meet the criteria for valid biomarkers described in the FDA Guidance.
2. Developing valid biomarkers comprising StaRT-PCR transcript abundance data from genes first identified through high-throughput screening.
3. Converting probable valid biomarkers into known valid biomarkers through multi-institutional clinical validation studies.
4. Developing valid biomarkers in the form of interactive transcript abundance indices

StaRT-PCR is suitable for generating probable valid biomarkers for the following purposes:

1. Early efficacy/safety assessment
2. Pre-clinical potency/dose selection information on human samples
3. Potential new target identification
4. Refinement of animal models
5. Potential theranostic, prognostic and diagnostic biomarkers
6. Supporting IND, NDA, and BLA submissions

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