Utility of adaptive strategy and adaptive design for biomarker-facilitated patient selection in pharmacogenomic or pharmacogenetic clinical development program

Journal of the Formosan Medical Association = Taiwan Yi Zhi
Sue-Jane Wang

Abstract

In the early to late phases of conventional clinical trials, improvement of disease status at study baseline is the anchor of an effective treatment measured by therapeutic response. These population-based clinical trials do not formally account for disease-associated marker genotype or genome-associated therapeutic response. We discuss alternative study designs in pharmacogenomic or pharmacogenetic clinical trials for genomic or genetic biomarker development, and for formally assessing the clinical utility of genomic or genetic (composite) biomarkers. A two-stage adaptive strategy from completed, ongoing or prospectively planned pharmacogenomic or pharmacogenetic clinical trials is described for development of a genomic or genetic biomarker. We present two types of adaptive design: (1) the genomic biomarker is developed external to the clinical trial, which is designed for treatment effect inference; and (2) first-stage data are used to explore a genomic biomarker, but statistical inference of treatment effect in the genomically or genetically defined biomarker subset is only performed at the second stage of the same trial. When the null hypothesis of no treatment effect in all randomized patients and the genomic patient subse...Continue Reading

References

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Citations

Apr 10, 2009·Biometrical Journal. Biometrische Zeitschrift·Sue-Jane WangRobert T O'Neill
Jul 3, 2010·Clinical Trials : Journal of the Society for Clinical Trials·Sue-Jane WangHm James Hung
Feb 26, 2016·PloS One·Miranta AntoniouRuwanthi Kolamunnage-Dona
Oct 19, 2019·The Pharmacogenomics Journal·Ruben P A van EijkMichael A van Es

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