TY - JOUR
T1 - Discussion of trial designs for biomarker identification and validation through the use of case studies
AU - Ou, Fang Shu
AU - An, Ming Wen
AU - Ruppert, Amy S.
AU - Mandrekar, Sumithra J.
N1 - Publisher Copyright:
© 2019 by American Society of Clinical Oncology.
PY - 2019
Y1 - 2019
N2 - With the launch of the National Cancer Institute's Precision Medicine Initiative in 2015, there has been a shift to trial designs that tailor health care solutions to individual patients by using a screening platform and by moving away from the one-trial/one-biomarker-at-a-time approach. To make precision medicine a reality, it is critical to identify and validate potential biomarkers to help select patients who will truly benefit from a targeted therapy. In this article, we discuss five trial designs: Enrichment, umbrella, basket, subgroup, and window of opportunity. For each trial design, we describe the design characteristics, use ongoing or completed trials as case studies, provide any recent advances to the trial design, and discuss advantages and disadvantages of each design.
AB - With the launch of the National Cancer Institute's Precision Medicine Initiative in 2015, there has been a shift to trial designs that tailor health care solutions to individual patients by using a screening platform and by moving away from the one-trial/one-biomarker-at-a-time approach. To make precision medicine a reality, it is critical to identify and validate potential biomarkers to help select patients who will truly benefit from a targeted therapy. In this article, we discuss five trial designs: Enrichment, umbrella, basket, subgroup, and window of opportunity. For each trial design, we describe the design characteristics, use ongoing or completed trials as case studies, provide any recent advances to the trial design, and discuss advantages and disadvantages of each design.
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U2 - 10.1200/PO.19.00051
DO - 10.1200/PO.19.00051
M3 - Review article
AN - SCOPUS:85080039579
SN - 2473-4284
VL - 3
JO - JCO Precision Oncology
JF - JCO Precision Oncology
ER -