I have been studying equation 17 in this paper.  This is their recommended 
estimator of treatment effect in a randomized trial.  I just noticed something 
that I think is by no means profound but still sort of neat.  I wonder if 
anyone else has noticed this.

They advocate fitting separate outcome models on each arm of a trial, applying 
both models to the full dataset, and then using a function of the unconditional 
estimator of treatment effect and an odd looking function of the two sets of 
predictions.  Based on earlier work by Tsiatis and allies, this estimator is 
optimal in a well-defined sense among a broad and useful class of estimators.

Let R1bar be the average residual on just the treatment sample from the outcome 
model fit on that sample.  Similarly, let R0bar be the average residual on just 
the control sample from the outcome model fit on that sample. Let Pred1bar be 
the average prediction across the entire sample of the predictions formed using 
the outcome model fit on just the treatment sample.  Similarly, let Pred2bar be 
the average prediction across the entire sample of the predictions formed using 
the outcome model fit on just the treatment sample.  Then their recommended 
treatment effect is

Betahat= (R1bar-R0bar)+(Pred1bar-Pred0bar).   (17)

If it happens (as it almost always does) that R1bar=R0bar=0, then this 
simplifies to just Pred1bar-Pred0bar.

This is a very simple and intuitive description of the estimator.

It also suggests using imputation to form these predictions, and Rubin's 
formula for combining multiple imputations to estimate its variance.  Steff van 
Buuren just presented a paper with this theme at JSM in Boston.  Andrea Piesse 
presented a very similar paper back in 2010 at JSM in Vancouver.  I did not 
make the connection to Tsiatis et al when Andrea and I were working on this 
back in 2010, but this suggests that the multiple imputation procedure for 
analysis of RCTs is the optimal approach.

--Dave

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David Judkins | Principal Scientist | Abt Associates
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