Evidence-based medicine eats itself

There are three commonly stated principles of evidence-based research:

1. Reliance when possible on statistically significant results from randomized trials;

2. Balancing of costs, benefits, and uncertainties in decision making;

3. Treatments targeted to individuals or subsets of the population.

Unfortunately and paradoxically, the use of statistics for hypothesis testing can get in the way of the movement toward an evidence-based framework for policy analysis. This claim may come as a surprise, given that one of the meanings of evidence-based analysis is hypothesis testing based on randomized trials. The problem is that principle (1) above is in some conflict with principles (2) and (3).

The conflict with (2) is that statistical significance or non-significance is typically used at all levels to replace uncertainty with certainty—indeed, researchers are encouraged to do this and it is standard practice.

The conflict with (3) is that estimating effects for individuals or population subsets is difficult. A quick calculation finds that it takes 16 times the sample size to estimate an interaction as a main effect, and given that we are lucky if our studies are powered well enough to estimate main effects of interest, it will typically be hopeless to try to obtain the near-certainty regarding interactions. That is fine if we remember principle (2), but not so fine if our experiences with classical statistics have trained us to demand statistical significance as a prerequisite for publication and decision making.