TY - JOUR
T1 - An FDA perspective on the regulatory implications of complex signatures to predict response to targeted therapies
AU - Beaver, Julia A.
AU - Tzou, Abraham
AU - Blumenthal, Gideon M.
AU - McKee, Amy E.
AU - Kim, Geoffrey
AU - Pazdur, Richard
AU - Philip, Reena
N1 - Publisher Copyright:
© 2016 American Association for Cancer Research.
PY - 2017/3/15
Y1 - 2017/3/15
N2 - As technologies evolve, and diagnostics move from detection of single biomarkers toward complex signatures, an increase in the clinical use and regulatory submission of complex signatures is anticipated. However, to date, no complex signatures have been approved as companion diagnostics. In this article, we will describe the potential benefit of complex signatures and their unique regulatory challenges, including analytic performance validation, complex signature simulation, and clinical performance evaluation. We also will review the potential regulatory pathways for clearance, approval, or acceptance of complex signatures by the FDA. These regulatory pathways include regulations applicable to in vitro diagnostic devices, including companion diagnostic devices, the potential for labeling as a complementary diagnostic, and the biomarker qualification program.
AB - As technologies evolve, and diagnostics move from detection of single biomarkers toward complex signatures, an increase in the clinical use and regulatory submission of complex signatures is anticipated. However, to date, no complex signatures have been approved as companion diagnostics. In this article, we will describe the potential benefit of complex signatures and their unique regulatory challenges, including analytic performance validation, complex signature simulation, and clinical performance evaluation. We also will review the potential regulatory pathways for clearance, approval, or acceptance of complex signatures by the FDA. These regulatory pathways include regulations applicable to in vitro diagnostic devices, including companion diagnostic devices, the potential for labeling as a complementary diagnostic, and the biomarker qualification program.
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U2 - 10.1158/1078-0432.CCR-16-1098
DO - 10.1158/1078-0432.CCR-16-1098
M3 - Review article
C2 - 27993967
AN - SCOPUS:85016158215
SN - 1078-0432
VL - 23
SP - 1368
EP - 1372
JO - Clinical Cancer Research
JF - Clinical Cancer Research
IS - 6
ER -