Improving efficiency in RCTs using propensity scores

Propensity scores have become a popular approach for confounder adjustment in observational studies. The basic idea is to model how the probability of receiving a treatment or exposure depends on the confounders, i.e. the ‘propensity’ to be treated. To estimate the effect of exposure, outcomes are then compared between exposed and unexposed who share the same value of the propensity score. Alternatively the outcome can be regressed on exposure, weighting the observations using the propensity score. For further reading on using propensity scores in observational studies, see for example this nice paper by Peter Austin.

But the topic of this post is on the use of propensity scores in randomized controlled trials. The post was prompted by an excellent seminar recently given by my colleague Elizabeth Williamson, covering the content of her recent paper ‘Variance reduction in randomised trials by inverse probability weighting using the propensity score” (open access paper here).

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Is the two sample t-test/ANOVA really biased in RCTs?

A couple of months ago I came across this paper, “Bias, precision and statistical power of analysis of covariance in the analysis of randomized trials with baseline imbalance: a simulation study”, published in the open access online journal BMC Medical Research Methodology, by Egbewale, Lewis and Sim. Using simulation studies, as the title says, the authors investigate the bias, precision and power of three analysis methods for a randomized trial with a continuous outcome and a baseline measure of the same variable, when there is an imbalance at baseline in the baseline measure. The three methods considered are ANOVA (a two-sample t-test here), an analysis of change (CSA, change from baseline to follow-up) scores, and analysis of covariance (ANCOVA), which corresponds to fitting a linear regression model with outcome measurement as the dependent variable, with randomized treatment and baseline measure as covariates.

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Test for Alzheimer’s (allegedly) no better than a coin toss

From a tweet I just came across the following article at the UK’s NHS Choices website. It raises doubts about the predictive value of a new test for Alzheimer’s disease, published in a paper here. The model aims to predict whether those suffering from mild cognitive impairment will progress to Alzheimer’s disease (AD) in the following year.

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