Comparison of Methods to Generalize Randomized Clinical Trial Results without Individual-Level Data for the Target Population.

Comparison of Methods to Generalize Randomized Clinical Trial Results without Individual-Level Data for the Target Population. Am J Epidemiol. 2018 Oct 11;: Authors: Hong JL, Webster-Clark M, Funk MJ, Stürmer T, Dempster S, Cole SR, Herr I, LoCasale R Abstract Our study explored the application of methods to generalize randomized controlled trial results to a target population without individual-level data. We compared four methods using aggregate data for the target population to generalize the JUPITER trial results to a target population of trial-eligible patients in Clinical Practice Research Datalink (CPRD). The gold-standard method used individual data from both the trial and CPRD to predict probabilities of being sampled in the trial and reweight trial participants to reflect CPRD patient characteristics. Methods 1 and 2 used weighting methods based on simulated individual data or the method of moments, respectively. Method 3 weighted the trial subgroup-specific treatment effects to match the distribution of an effect modifier in CPRD. Method 4 calculated the expected absolute benefits in CPRD assuming homogeneous relative treatment effect. Methods based on aggregate data for the target population generally yielded results between the trial and gold-standard estimates. Methods 1 and 2 yielded estimates closest to the gold-standard when continuous effect modifiers were represented as categorical variables. Although individual d...
Source: Am J Epidemiol - Category: Epidemiology Authors: Tags: Am J Epidemiol Source Type: research