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Kevin Wing, Clémence Leyrat, Improving the validity of noninterventional comparative effectiveness research by basing study design on a specified existing randomized controlled trial, American Journal of Epidemiology, Volume 194, Issue 4, April 2025, Pages 913–917, https://doi.org/10.1093/aje/kwae317
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Abstract
This issue of the American Journal of Epidemiology includes 3 articles (2 reporting original analyses and 1 systematic review) in which noninterventional studies used an existing randomized controlled trial (RCT) as a reference standard to inform noninterventional study design and against which to benchmark results. This commentary provides a brief background on the challenges of noninterventional comparative effectiveness research, before elaborating on (1) the potential benefits and challenges of basing noninterventional study design on a specified existing RCT and (2) the distinction between designing analysis based upon a specified existing RCT and studies based solely upon a hypothetical target trial. Finally, several recommendations for the conduct and reporting of noninterventional studies based on existing RCTs are provided.
This article is part of a Special Collection on Pharmacoepidemiology.