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Summary Covariate adjustment can improve precision in analysing randomized experiments. With fully observed data, regression adjustment and propensity score weighting are asymptotically equivalent in improving efficiency over unadjusted analysis. When some outcomes are missing, we consider combining these two adjustment methods with the inverse probability of observation weighting for handling missing outcomes, and show that the equivalence between the two methods breaks down. Regression adjustment no longer ensures efficiency gain over unadjusted analysis unless the true outcome model is linear in covariates or the outcomes are missing completely at random. Propensity score weighting, in contrast, still guarantees efficiency over unadjusted analysis, and including more covariates in adjustment never harms asymptotic efficiency. Moreover, we establish the value of using partially observed covariates to secure additional efficiency by the missingness indicator method, which imputes all missing covariates by zero and uses the union of the completed covariates and corresponding missingness indicators as the new, fully observed covariates. Based on these findings, we recommend using regression adjustment in combination with the missingness indicator method if the linear outcome model or missing-completely-at-random assumption is plausible and using propensity score weighting with the missingness indicator method otherwise.
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Semi-parametric sensitivity analysis for trials with irregular and informative assessment times
ABSTRACT Many trials are designed to collect outcomes at or around pre-specified times after randomization. If there is variability in the times when participants are actually assessed, this can pose a challenge to learning the effect of treatment, since not all participants have outcome assessments at the times of interest. Furthermore, observed outcome values may not be representative of all participants’ outcomes at a given time. Methods have been developed that account for some types of such irregular and informative assessment times; however, since these methods rely on untestable assumptions, sensitivity analyses are needed. We develop a sensitivity analysis methodology that is benchmarked at the explainable assessment (EA) assumption, under which assessment and outcomes at each time are related only through data collected prior to that time. Our method uses an exponential tilting assumption, governed by a sensitivity analysis parameter, that posits deviations from the EA assumption. Our inferential strategy is based on a new influence function-based, augmented inverse intensity-weighted estimator. Our approach allows for flexible semiparametric modeling of the observed data, which is separated from specification of the sensitivity parameter. We apply our method to a randomized trial of low-income individuals with uncontrolled asthma, and we illustrate implementation of our estimation procedure in detail.
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- Award ID(s):
- 2242776
- PAR ID:
- 10596419
- Publisher / Repository:
- Biometrics
- Date Published:
- Journal Name:
- Biometrics
- Volume:
- 80
- Issue:
- 4
- ISSN:
- 0006-341X
- Format(s):
- Medium: X
- Sponsoring Org:
- National Science Foundation
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- Free Publicly Accessible Full Text
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Accepted Manuscript
- Journal Article:
- https://doi.org/10.1093/biomtc/ujae154
