IAN WHITE (MRC Biostats, Cambridge)

Randomisation-based efficacy estimators in randomised trials: when are they useful?

In randomised trials with departures from allocated treatment, it may be desirable to estimate the causal effect of treatment itself.  Intention-to-treat analysis does not do this, although it provides an unbiased comparison of treatment policies as implemented.  Per-protocol analysis is the most commonly used alternative in clinical trials, but it is subject to selection bias.  I will describe randomisation-based efficacy estimators, which avoid both these problems, and give some examples where they have led to useful conclusions.

Ian's PowerPoint slides