Home

Section I: What Are Pragmatic Trials?

What Purpose Does Pragmatic Research Serve?

4 Reasons to Consider Pragmatic Trials

  1. Pragmatic Trials allow us to evaluate interventions under typical conditions, with typical patients vs. under ideal conditions with a homogeneous set of selected patients (Explanatory Trials).

  2. Traditional trials usually take a long time to translate into practice; it takes an average of 17 years for just 14% of research to translate into practice. 

  3. The conditions under which traditional trials are conducted are often not seen by practitioners as relevant to their practice or patient populations. 

  4. Although there are over 18,000 randomized controlled trials (RCT's) published each year, the vast majority of systematic reviews conclude that there is not enough evidence to inform clinical decisions. 

Learning Objectives:

Describe the purpose and defining characteristics of pragmatic research.

What Are Pragmatic Trials?

Overview:

There are several differences between pragmatic trials and traditional efficacy or explanatory trials. It is not that one type of trial is always better than the other, but rather that they answer different questions. Some of the key differences are that in pragmatic studies, there is more emphasis on external validity of findings. Thus, stakeholders are involved throughout the study planning, implementation and reporting phases.  Measures in pragmatic trials focus on more practical issues such as the reach and costs of an intervention and tend to use existing measures such as data available from electronic health records more than do explanatory trials.  In contrast, explanatory trials tend to focus more on investigator-defined outcomes to answer questions about theoretical mechanisms. To advance science and application, we need answers to both.

PRAGMATIC RESEARCH BY OTHER NAMES:

You may already be familiar with Pragmatic Trials, they have also been known by some of the names defined below

  • Large Simple Trials
  • Phase IIb-IV Trials
  • Community-Based Research
  • Practical Trials
  • Naturalistic Clinical Trials

Large Simple Trials

"Large Simple Trials (LSTs), usually randomized, are characterized by large sample sizes, broad entry criteria consistent with the intended target population, streamlined data collection, objectively measured endpoints (e.g., death, hospitalization), and follow-up that mimics normal clinical practice. LSTs often rely on recruitment of patients and investigators in the community setting and minimize patient visits and on-site monitoring. LSTs are ideally suited to answer many important clinical questions."

Source: Clinical Trials Transformation Initiative. "Large Simple Trials," Sept. 2013. Web. 12 Nov. 2014.
visit this site.

Phase IIIb:

Clinical trials conducted after regulatory submission of an NDA or other dossier, but prior to the medicine's approval and launch. These trials may supplement earlier trials, complete earlier trials, or may be directed toward new types of trials (e.g., quality of life, marketing) or Phase IV evaluations. This is the period between submission and approval of a regulatory dossier for marketing authorization. 

Phase IV:
Studies or trials conducted after a medicine is marketed to provide  additional details about the medicine's efficacy or safety profile. Different  formulations, dosages, durations of treatment, medicine interactions, and other medicine comparisons may be evaluated. New age groups, races, and other types of patients can be studied. 

Source: Clinical Trials Transformation Initiative. "Large Simple Trials," Sept. 2013. Web. 12 Nov. 2014.
visit this site.

Community-Based Research

A collaborative and participatory approach to research in which community organizations (schools, residents and civic leaders) partner with academic researchers (faculty, academic staff and students) to produce knowledge that solves real world problems. Community Based Research seeks community engagement at each phase of the research project. Community Based Research does not specify one model of research over another; models are selected based on what is most appropriate to the problems to be studied and solved.

 

Source: University of Wisconson-Madison/Board of Regents, College of Letters & Science. 2014. Web. 12 Nov. 2014.
visit this site.

Practical Trials

Clinical trials for which the hypothesis and study design are developed specifically to answer the questions faced by decision makers are called pragmatic or practical clinical trials (PCTs). The characteristic features of PCTs are that they (1) select clinically relevant alternative interventions to compare, (2) include a diverse population of study participants, (3) recruit participants from heterogeneous practice settings, and (4) collect data on a broad range of health outcomes. 

Source: Tunis SR, et al. Practical clinical trials: increasing the value of clinical research for decision making in clinical and health policy. JAMA, 2003;290:1624-1632. 
visit this site.

Naturalistic clinical trials

Prospective “noninterventional” observational studies of phenomena, or, more broadly, retrospective analyses of existing data and studies. 

Source: Institute of Translational Health Sciences. 2014. Web. 12 Nov. 2014.
visit this site.

Comparing Pragmatic Trials and Traditional Efficacy Trials

Pragmatic Trials address questions important to stakeholders.  There are often multiple stakeholders for a given issue, including patients, providers, and administrative decision makers.  Pragmatic trials include both multiple, heterogeneous settings and diverse populations that together represent typical settings and typical patients that would receive a given intervention.  They also include multiple outcomes that are important to stakeholders, including decision makers. The final important characteristic of pragmatic trials is that they include comparisons of different real world alternatives, rather than placebo or no treatment conditions.

Study outcomes include the intervention reach (percent of patients offered and completing the My Own Health Report (MOHR) assessment), effectiveness (patients reporting being asked about topics, setting change goals, and receiving assistance in early versus delayed intervention practices), contextual factors influencing outcomes, and intervention costs.

Pragmatic Study

VS.

Traditional Efficacy Trial

Stakeholder Involvement

Pragmatic Studies

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

Traditional Studies

Limited engagement, often in response to investigator ideas of study subjects.

Research Design

Pragmatic Studies

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

Traditional Studies

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

Outcomes

Pragmatic Studies

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

Traditional Studies

Efficacy, mechanism identification, component analysis.

Measures

Pragmatic Studies

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

Traditional Studies

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

Costs

Pragmatic Studies

Assessments include intervention costs and replication costs in relation to outcomes.

Traditional Studies

Often not collected or reported.

Data Source

Pragmatic Studies

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

Traditional Studies

Data generation and collection part of clinical trial.

Analyses

Pragmatic Studies

Process and outcome analyses relevant to stakeholders and from different perspectives. 

Traditional Studies

Specified a priori and typically restricted to investigator hypotheses.

Availability of Findings

Pragmatic Studies

Rapid learning and implementation. 

Traditional Studies

Delay between trial completion and availability of results. 

Source: Krist et al. Implementation Science 2013, 
View Table Source

Required

  • Important to stakeholders
  • Burden is low to moderate
  • Sensitive to change
  • Actionable

Additional

  • Broadly applicable, has norms to interpret
  • Low probability of harm
  • Addresses public health goal(s)
  • Related to theory or model
  • “Maps” to “gold standard” metric or measure

Resources

The Importance of Pragmatic Clinical Trials

Russell Glasgow, PhD
At the pragmatic trials workshop, Russell Glasgow gives an introductory presentation on the importance of pragmatic clinical trials. 

Key Takeaways : Pragmatic Trials

Click To Reveal Flipcard Answer

What do Pragmatic Trials allow us to do?

Evaluate interventions under typical conditions, with typical patients vs. Under ideal conditions with a homogeneous set of selected patients (Explanatory Trials).

Some Key Aspects of Pragmatic Trials

  • Questions from and important to stakeholders;
  • Multiple heterogeneous settings
  • Diverse populations;
  • Comparison conditions are real world alternatives;
  • Multiple outcomes important to stakeholders and policy makers.

Other names for Pragmatic Trials

  • Large Simple Trials
  • Phase IIIb-IV Trials 
  • Community-Based Research 
  • Practical Trials 
  • Naturalistic Clinical Trials 

 

Key Reasons to Consider Pragmatic Trials

  • Traditional trials usually take a long time to translate into practice: it takes an average of 17 years for just 14% of research to translate into practice.  
  • The conditions under which traditional trials are conducted are often not seen by practitioners as relevant to their practice or patient populations.  
  • there is not enough evidence to inform clinical decisions.

 

Explanatory Clinical Trials

A specialized, randomized experiment in a specialized population under optimal conditions.

Which type of Trial is Better?

It is not that one type of trial is always better than the other, but rather that they answer different questions- about results under optimal (explanatory) versus typical (pragmatic) conditions.  To advance science and application, we need answers to both.

References 

Click Each Reference to View the Abstract and Download Full Texts.