Personalized Evaluation of Biomarker Value: A Cost-benefit Perspective

For a patient who is facing a treatment decision, the added value of information provided by a biomarker depends on the individual patient’s expected response to treatment with and without the biomarker, as well as his/her tolerance of disease and treatment harm. However, individualized estimators of the value of a biomarker are lacking. We propose a new graphical tool named the subject-specific expected benefit curve for quantifying the personalized value of a biomarker in aiding a treatment decision. We develop semiparametric estimators for two general settings: i) when biomarker data are available from a randomized trial; and ii) when biomarker data are available from a cohort or a cross-sectional study, together with external information about a multiplicative treatment effect. We also develop adaptive bootstrap confidence intervals for consistent inference in the presence of non-regularity. The proposed method is used to evaluate the individualized value of the serum creatinine marker in informing treatment decisions for the prevention of renal artery stenosis