Analysis of odds, probability, and hazard ratios: From 2 by 2 tables to two-sample survival data

Abstract: Analysis of 2 by 2 tables and two-sample survival data has been widely used. Exact calculation is computational intractable for conditional likelihood inference in odds ratio models with large marginals in 2 by 2 tables, or partial likelihood inference in Cox's proportional hazards models with considerable tied event times. Approximate methods are often employed, but their statistical properties have not been formally studied while taking into account the approximation involved. We develop new methods and theory by constructing suitable estimating functions while leveraging knowledge from conditional or partial likelihood inference. We propose a weighted Mantel--Haenszel estimator in an odds ratio model such as Cox's discrete-time proportional hazards model. Moreover, we consider a probability ratio model, and derive as a consistent estimator the Breslow--Peto estimator, which has been regarded as an approximation to partial likelihood estimation in the odds ratio model. We study both model-based and model-robust variance estimation. For the Breslow--Peto estimator, our new model-based variance estimator is no greater than the commonly reported variance estimator. We present numerical studies which support the theoretical findings.