Trials and observations: Using real world data to extend results from randomized trials in populations with acute coronary syndromes

Randomized trials do not estimate treatment effects in individuals that were eligible but did not enroll in the trial. However, guidelines established as a result of the trials apply to these populations in clinical practice. On average, around 40% of eligible individuals do not enroll in trials.

A treatment that shows no benefit in trial participants may be effective in a subpopulation not likely to enroll, such as older individuals. Differences in effectiveness must be quantified to guide policy decisions.

Extending randomized trial results to the treatment eligible population is often handled using real-world data alone, which are prone to bias.This project will propose a novel framework that uses transportability and benchmarking methods to simultaneously utilize data from the TASTE and VALIDATE randomized trials and real-world data from the SWEDEHEART registry to robustly fill the knowledge gaps left by the trials.

Specifically, we will estimate the effect of thrombus aspiration and two anticoagulant medications after myocardial infarction in the whole trial-eligible population of Sweden; test the reliability of studies that use real-world data alone to estimate treatment effects; and create tutorials, teaching materials, and open-source code to facilitate the uptake of these methodologies.

Our research will improve public health decisions in Sweden and serve as a model in the extension of findings from randomized trials to the population eligible for treatment.