Defining the role of pragmatic clinical trials in cancer clinical research: outcomes of a collaborative workshop hosted by the European Organisation for Research and Treatment of Cancer

Abstract

Explanatory clinical trials, which focus on evaluating therapeutic efficacy under ideal circumstances, are crucial for learning about new therapeutic interventions; however, they also exhibit shortcomings. These include non-representative populations and frequent use of intermediate endpoints, leading to uncertainty about the applicability of study results to patients in the real-world. Moreover, these trials often do not address all clinically meaningful questions, highlighting the need for optimisation within the oncology research framework. Refinements can be partly achieved by incorporating more pragmatic elements into cancer clinical trials. At a virtual European Organisation for Research and Treatment of Cancer workshop, key stakeholders convened to discuss the methodological characteristics and value of pragmatic trials, which focus on evaluating effectiveness in routine clinical practice, and their capacity to address the efficacy-effectiveness gap. This Policy Review outlines and discusses some of the views and perspectives expressed on the role of pragmatic trials in the current framework and their ability to inform decision making, and the recommended priorities for enhancing pragmatism in cancer clinical research.