An introduction to pragmatic trials in lung cancer research: A multi-faceted approach

Abstract

Pragmatic clinical trials (PTs) are increasingly recognised as a key methodology in lung cancer research, designed to evaluate the effectiveness of interventions in routine clinical settings. In contrast to traditional randomised controlled trials (RCTs), which involve highly selected patient populations under ideal conditions, PTs enrol broader, more representative cohorts, use streamlined trial procedures, and focus on outcomes that reflect patient-centred priorities. This enhances the generalisability of the results and supports evidence-based, joint decision-making between patients and clinicians. PTs offer a more sustainable and scalable path to generate clinically meaningful evidence that improves the quality of patient care and can address longstanding evidence gaps in the management of patients with lung cancer.

This narrative review outlines the main features of PTs, highlighting the PRECIS-2 tool as a framework to design and evaluate the degree of pragmatism of a trial. Common challenges in clinical trial design — including recruitment of participants, informed consent, selection of appropriate clinical endpoints, data quality — are discussed alongside practical solutions. The emerging role of PTs in generating regulatory-grade evidence and the impact of PTs on clinical guidelines is discussed. Ongoing PTs are outlined, which demonstrate how pragmatic methodologies can be used to the evaluate screening interventions, therapeutic strategies and models of care delivery across a range of clinical settings.