Evaluation of Heterogeneity of Treatment Effects in ComparativeEffectiveness Research

[摘要] Reliable knowledge of heterogeneity of treatment effects is criticalin decision making about the relative risks and benefits of competingtreatment options for individual patients and subgroups of patients.Despite their widely recognized attributes as the gold-standard forgenerating evidence relating to comparative efficacy, randomizedcontrolled trials (RCTs) are designed to provide data on treatmentefficacy on average for a target population, and thus fall short ofgenerating evidence pertinent at the individual or subgroup level.Extrapolation of results from RCTs to real-world situations is limitedby factors inherent to the design of the studies, which include stringententry criteria that exclude subgroups of interest, logistical constraintsto study broader subgroups, and other practical considerations thatimpose constraints that are inconsistent with real-world practice andadherence.