Extending Inferences from Randomized Clinical Trials to Target Populations: A Scoping Review of Transportability Methods

TL;DR

This scoping review examines methods for extending RCT findings to real-world populations, highlighting their ability to bridge controlled trial results with observational data despite variations in effect estimates.

Contribution

It provides a comprehensive overview of transportability methods applied across diverse medical fields, emphasizing their role in translating RCT results to real-world settings.

Findings

Transportability methods can effectively extend RCT findings to observational cohorts.

Estimated treatment effects in target populations sometimes differ significantly from RCTs.

Methods vary in their ability to accurately predict real-world treatment effects.

Abstract

Objective: Randomized controlled trial (RCT) results often inform clinical decision-making, but the highly curated populations of trials and the care provided during the trial are often not reflective of real-world practice. The objective of this scoping review is to identify the ability of methods to transport findings from RCTs to target populations. Study design: A scoping review was conducted on the literature focusing on the transportability of the results from RCTs to observational cohorts. Each study was assessed based on the methodology used for transportability and the extent to which the treatment effect from the RCT was estimated in the target population in observational data. Results: A total of 15 published papers were included. The research topics include cardiovascular diseases, infectious diseases, psychiatry, oncology, orthopedics, anesthesiology, and hematology. These studies show that the findings from RCTs could be translated to real-world settings, with varying degrees of effect size and precision. In some cases, the estimated treatment effect for the target population were statistically significantly different from those in RCTs. Conclusion: Despite variations in the magnitude of effects between RCTs and real-world studies, transportability methods play an important role in effectively bridging the RCTs and real-world care delivery, offering valuable insights for evidence-based medicine.