The Use of Covariates and Random Effects in Evaluating Predictive Biomarkers Under a Potential Outcome Framework

The Annals of Applied Statistics
Zhiwei ZhangAiyi Liu

Abstract

Predictive or treatment selection biomarkers are usually evaluated in a subgroup or regression analysis with focus on the treatment-by-marker interaction. Under a potential outcome framework (Huang, Gilbert and Janes [Biometrics68 (2012) 687-696]), a predictive biomarker is considered a predictor for a desirable treatment benefit (defined by comparing potential outcomes for different treatments) and evaluated using familiar concepts in prediction and classification. However, the desired treatment benefit is un-observable because each patient can receive only one treatment in a typical study. Huang et al. overcome this problem by assuming monotonicity of potential outcomes, with one treatment dominating the other in all patients. Motivated by an HIV example that appears to violate the monotonicity assumption, we propose a different approach based on covariates and random effects for evaluating predictive biomarkers under the potential outcome framework. Under the proposed approach, the parameters of interest can be identified by assuming conditional independence of potential outcomes given observed covariates, and a sensitivity analysis can be performed by incorporating an unobserved random effect that accounts for any residual ...Continue Reading

References

Feb 28, 1997·Statistics in Medicine·E Russek-Cohen, R M Simon
Sep 14, 2000·Biometrics·G L Gadbury, H K Iyer
Dec 14, 2004·The New England Journal of Medicine·Soonmyung PaikNorman Wolmark
Oct 24, 2008·The New England Journal of Medicine·Christos S KarapetisJohn R Zalcberg
Feb 5, 2009·Journal of Statistical Planning and Inference·Richard Simon
Jan 1, 2008·The International Journal of Biostatistics·Xiaogang SuSong Yang
Mar 27, 2010·Clinical Trials : Journal of the Society for Clinical Trials·Richard Simon
Jun 15, 2011·Annals of Statistics·Min Qian, Susan A Murphy
Aug 5, 2011·Statistics in Medicine·Jared C FosterStephen J Ruberg
May 4, 2012·Biometrics·Baqun ZhangMarie Davidian
Dec 4, 2012·The American Statistician·Robert S PoulsonDavid B Allison
Dec 17, 2014·Journal of the Royal Statistical Society. Series C, Applied Statistics·Zhiwei ZhangGuoxing Soon

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Citations

Mar 28, 2017·The International Journal of Biostatistics·Zhiwei ZhangGuoxing Soon
Sep 22, 2019·The International Journal of Biostatistics·Shujie MaMingming Liu
Nov 12, 2019·Annals of Internal Medicine·David M KentEwout W Steyerberg
Apr 30, 2020·Biometrical Journal. Biometrische Zeitschrift·Yoann BlangeroFabien Subtil
Jun 26, 2015·Journal of the National Cancer Institute·Holly JanesPatrick J Heagerty
Jan 3, 2019·Statistical Methods in Medical Research·Yoann BlangeroFabien Subtil
Feb 1, 2021·Statistics in Medicine·Lili LiuLei Liu

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