Randomized trials are seen as the gold standard for estimating the effects of interventions because, when implemented well, they provide unbiased estimates of treatment effects in the sample at hand. However, recent years have seen an increased understanding of their limitations in providing evidence that is more broadly applicable and relevant for real-world practice, known as “generalizability.” A lack of generalizability may be a particular problem for comparative effectiveness research (CER), which aims to help clinicians and policymakers make informed decisions for individuals and populations. This chapter outlines recent advances in methods to assess and enhance the generalizability of randomized trials in CER, discussing both design and analysis strategies. A case study is provided of a weighting approach that reweights the trial sample to reflect the target population with respect to effect modifiers. Recommendations for further research and the practical use of the methods discussed are also provided.
ASJC Scopus subject areas
- Pharmacology, Toxicology and Pharmaceutics(all)