Heterogeneity in pragmatic randomized trials: sources and management
October 22, 2022
IMPACT member, Monica Taljaard, PhD is among authors who identify different sources of clinical and methodological heterogeneity, which translates into heterogeneity in patient responses— some considered desirable and some undesirable. For each of them, they discuss and using real-world trial examples, illustrate how heterogeneity should be managed over the whole course of the trial.
Abstract
Background: Pragmatic trials aim to generate evidence to directly inform patient, caregiver and health-system manager policies and decisions. Heterogeneity in patient characteristics contributes to heterogeneity in their response to the intervention. However, there are many other sources of heterogeneity in outcomes. Based on the expertise and judgements of the authors, we identify different sources of clinical and methodological heterogeneity, which translate into heterogeneity in patient responses-some we consider as desirable and some as undesirable. For each of them, we discuss and, using real-world trial examples, illustrate how heterogeneity should be managed over the whole course of the trial.