Adaptive propensity score procedure improves matching in prospective observational trials

Randomized controlled trials are the gold-standard for clinical trials. However, randomization is not always feasible. In this article we propose a prospective and adaptive matched case-control trial design assuming that a control group already exists.

We propose and discuss an interim analysis step to estimate the matching rate using a resampling step followed by a sample size recalculation. The sample size recalculation is based on the observed mean resampling matching rate. We applied our approach in a simulation study and to a real data set to evaluate the characteristics of the proposed design and to compare the results to a naive approach.

The proposed design achieves at least 10% higher matching rate than the naive approach at final analysis, thus providing a better estimation of the true matching rate. A good choice for the interim analysis seems to be a fraction of around [Formula: see text] to [Formula: see text] of the control patients.

The proposed resampling step in a prospective matched case-control trial design leads to an improved estimate of the final matching rate and, thus, to a gain in power of the approach due to sensible sample size recalculation.