Non-inferiority tests are well developed for randomized parallel group trials where the control and experimental groups are independent. However, these tests may not be appropriate for assessing non-inferiority in correlated one-to-many matched data. We propose a new statistical test that extends Farrington-Manning’s (FM) test to the case where many (≥1) control subjects are matched to each experimental subject. We conducted a Monte Carlo simulation study to compare the size and power of the proposed test with tests developed for clustered one-to-one matched pair data and tests based on generalized estimating equations (GEE). For various correlation patterns, the sizes of tests developed for clustered matched pair data and GEE-based tests are inflated when applied to the case where many control subjects are matched to each experimental subject. The size of the proposed test, on the other hand, is close to the nominal level for a variety of correlation patterns.
There is a debate in the literature regarding whether or not statistical tests appropriate for independent samples can be used to assess the statistical significance of treatment effects in propensity-score matched studies. We used Monte Carlo simulations to examine the effect on assessing non-inferiority via risk difference when a method for independent samples (i.e. FM test) is used versus when a method for correlated matched samples is used in propensity-score one-to-many matched studies. If propensity-score matched samples are well-matched on baseline covariates and contain almost all of the experimental treated subjects, a method for correlated matched samples is preferable with respect to power and Type I error than a method for independent samples.
Sometimes there are more experimental subjects to choose from for matching than control subjects. We conducted a Monte Carlo simulation study to compare the size and power of the previously mentioned tests when many (≥1) experimental subjects are matched to each control subject. In this case, the Nam-Kwon test for clustered data performs the best in controlling the type I error rate for a variety of correlation patterns. Therefore, the appropriate non-inferiority test to use for correlated matched data depends, in part, on the sample size allocation of subjects.
| Identifer | oai:union.ndltd.org:bu.edu/oai:open.bu.edu:2144/27329 |
| Date | 23 January 2018 |
| Creators | Perez, Jeremiah |
| Source Sets | Boston University |
| Language | en_US |
| Detected Language | English |
| Type | Thesis/Dissertation |
| Rights | Attribution-NonCommercial-NoDerivatives 4.0 International, http://creativecommons.org/licenses/by-nc-nd/4.0/ |
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