Pragmatic Approaches in Clinical Trials

Pragmatic clinical trials are designed to assess the effectiveness of interventions in routine care settings, maximising applicability to everyday practice while preserving sufficient methodological rigour. Unlike explanatory trials, which seek to determine whether an intervention can work under ideal conditions, pragmatic trials aim to establish how well it does work when delivered through standard clinical processes. Key features include broad eligibility criteria, flexible intervention delivery, use of existing health‐care infrastructure for recruitment and data collection, and outcomes that matter to patients and health‐care providers. Embedding randomisation within electronic health records or clinical registries streamlines recruitment and follow-up, reduces costs and timelines, and generates real-world evidence to inform policy and practice. These approaches have global significance in resource‐limited settings and for rapidly evolving health challenges, offering a pathway to more efficient and generalisable evidence generation.

Research from Nature Portfolio

No recent Nature Portfolio content available.

Pragmatic Approaches in Clinical Trials publication trend

The graph below shows the total number of articles in pragmatic approaches in clinical trials across all publications each year (not limited to Nature Index journals).

Technical terms

Pragmatic trial: A randomised study conducted under usual care conditions to evaluate intervention effectiveness in real-world settings.

Explanatory trial: A randomised study under tightly controlled conditions to assess an intervention’s efficacy.

PRECIS-2 tool: An instrument comprising domains that score a trial’s features from very explanatory to very pragmatic, guiding design and reporting.

Registry-based RCT (RRCT): A trial that uses existing clinical registries for participant identification, randomisation and outcome capture.

Real-world evidence: Data obtained from routine clinical practice, administrative databases or registries rather than traditional controlled research environments.

References

1. Validity and timeliness of cancer diagnosis data collected during a prospective cohort study and reported by the English and Welsh cancer registries: a retrospective, comparative analysis. The Lancet Oncology (2024).
2. Real-world evidence: How pragmatic are randomized controlled trials labeled as pragmatic?. BMC Medicine (2018).
3. The opportunities and challenges of pragmatic point-of-care randomised trials using routinely collected electronic records: evaluations of two exemplar trials. Health Technology Assessment (2014).
4. Making clinical trials more relevant: improving and validating the PRECIS tool for matching trial design decisions to trial purpose. Trials (2013).
5. Defining key design elements of registry-based randomised controlled trials: a scoping review. Trials (2020).