Temporal discontinuity trials and randomization: success rates versus design strength

TL;DR

This paper compares the effectiveness of randomized and non-randomized study designs, demonstrating conditions under which alternative designs can outperform randomization in informing treatment policies, especially with heterogeneous effects.

Contribution

It introduces formal conditions for temporal-discontinuity designs to approximate randomized trials and shows how deliberate assignment can be more efficient for policy formulation.

Findings

Temporal-discontinuity designs can approximate randomized trials under certain conditions.

Deliberate treatment assignment can outperform randomization in heterogeneous effect settings.

Alternative study designs can provide valuable evidence for treatment decisions.

Abstract

We consider the following comparative effectiveness scenario. There are two treatments for a particular medical condition: a randomized experiment has demonstrated mediocre effectiveness for the first treatment, while a non-randomized study of the second treatment reports a much higher success rate. On what grounds might one justifiably prefer the second treatment over the first treatment, given only the information from those two studies, including design details? This situation occurs in reality and warrants study. We consider a particular example involving studies of treatments for Crohn's disease. In order to help resolve these cases of asymmetric evidence, we make three contributions and apply them to our example. First, we demonstrate the potential to improve success rates above those found in a randomized trial, given heterogeneous effects. Second, we prove that deliberate treatment assignment can be more efficient than randomization when study results are to be transported to formulate an intervention policy on a wider population. Third, we provide formal conditions under which a temporal-discontinuity design approximates a randomized trial, and we introduce a novel design parameter to inform researchers about the strength of that approximation. Overall, our results indicate that while randomization certainly provides special advantages, other study designs such as temporal-discontinuity designs also have distinct advantages, and can produce valuable evidence that informs treatment decisions and intervention policy.