This paper studies theCox model with time-varying coefficients for cause-specific hazard functions when the causes of failure are subject to missingness. Inverse probability weighted and augmented inverse probability weighted estimators are investigated. The latter is considered as a two-stage estimator by directly utilizing the inverse probability weighted estimator and through modeling available auxiliary variables to improve efficiency. The asymptotic properties of the two estimators are investigated. Hypothesis testing procedures are developed to test the null hypotheses that the covariate effects are zero and that the covariate effects are constant. We conduct simulation studies to examine the finite sample properties of the proposed estimation and hypothesis testing procedures under various settings of the auxiliary variables and the percentages of the failure causes that are missing. These simulation results demonstrate that the augmented inverse probability weighted estimators are more efficient than the inverse probability weighted estimators and that the proposed testing procedures have the expected satisfactory results in sizes and powers. The proposed methods are illustrated using the Mashi clinical trial data for investigating the effect of randomization to formula-feeding versus breastfeeding plus extended infant zidovudine prophylaxis on death due to mother-to-child HIV transmission in Botswana.
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Inference under covariate‐adaptive randomization with multiple treatments
This paper studies inference in randomized controlled trials with covariate‐adaptive randomization when there are multiple treatments. More specifically, we study in this setting inference about the average effect of one or more treatments relative to other treatments or a control. As in Bugni, Canay, and Shaikh (2018), covariate‐adaptive randomization refers to randomization schemes that first stratify according to baseline covariates and then assign treatment status so as to achieve “balance” within each stratum. Importantly, in contrast to Bugni, Canay, and Shaikh (2018), we not only allow for multiple treatments, but further allow for the proportion of units being assigned to each of the treatments to vary across strata. We first study the properties of estimators derived from a “fully saturated” linear regression, that is, a linear regression of the outcome on all interactions between indicators for each of the treatments and indicators for each of the strata. We show that tests based on these estimators using the usual heteroskedasticity‐consistent estimator of the asymptotic variance are invalid in the sense that they may have limiting rejection probability under the null hypothesis strictly greater than the nominal level; on the other hand, tests based on these estimators and suitable estimators of the asymptotic variance that we provide are exact in the sense that they have limiting rejection probability under the null hypothesis equal to the nominal level. For the special case in which the target proportion of units being assigned to each of the treatments does not vary across strata, we additionally consider tests based on estimators derived from a linear regression with “strata fixed effects,” that is, a linear regression of the outcome on indicators for each of the treatments and indicators for each of the strata. We show that tests based on these estimators using the usual heteroskedasticity‐consistent estimator of the asymptotic variance are conservative in the sense that they have limiting rejection probability under the null hypothesis no greater than and typically strictly less than the nominal level, but tests based on these estimators and suitable estimators of the asymptotic variance that we provide are exact, thereby generalizing results in Bugni, Canay, and Shaikh (2018) for the case of a single treatment to multiple treatments. A simulation study and an empirical application illustrate the practical relevance of our theoretical results.
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- Award ID(s):
- 1729280
- PAR ID:
- 10180229
- Date Published:
- Journal Name:
- Quantitative Economics
- Volume:
- 10
- Issue:
- 4
- ISSN:
- 1759-7323
- Page Range / eLocation ID:
- 1747 to 1785
- Format(s):
- Medium: X
- Sponsoring Org:
- National Science Foundation
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