Table 1 Distinguishing differences between pragmatic and traditional clinical efficacy trials

Stakeholder involvement

Engaged in all study phases including study design, conducting the study, collecting data, interpreting results, disseminating findings

Limited engagement, often in response to investigator ideas or study subjects

Research Design

Includes internal and external validity, design fidelity and local adaptation, real life settings and populations, contextual assessments

Focus on limiting threats to internal validity, typically uses randomized controlled trial, participants and settings typically homogenous

Outcomes

Reach, effectiveness, adoption, implementation, comparative effectiveness, sustainability

Efficacy, mechanism identification, component analysis

Measures

Brief, valid, actionable with rapid clinical utility, feasible in real world and low-resource settings

Validated measures that minimize bias, focus on internal consistency and theory rather than clinical relevance

Costs

Assessments include intervention costs and replication costs in relation to outcomes

Often not collected or reported

Data Source

May include existing data (electronic health records, administrative data) and brief patient reports.

Data generation and collection part of clinical trial

Analyses

Process and outcome analyses relevant to stakeholders and from different perspectives

Specified a priori and typically restricted to investigator hypotheses

Availability of findings

Rapid learning and implementation

Delay between trial completion and analytic availability