Pragmatic Clinical Trials (PCTs)

Key Words

  • Pragmatic Clinical Trial (PCT)
  • Large Simple Trial
  • Low Intervention Clinical Trial (LICT)
  • Comparative Effectiveness Study
  • Type A Clinical Trial
  • Low Risk Clinical Trial
  • Regulatory Grade Real World Evidence

 

Common Questions

  • Who pays for the drug?
  • What is Needed in Terms of Safety Reporting for PCTs?
  • What are the GCP training requirements for PCTs

There has been a recent resurgence in the use of the term ‘pragmatic clinical trial’, but what are they?

According to the imi GetReal Glossary, a pragmatic clinical trial is A study comparing health interventions among a randomised, diverse population representing clinical practice, and measuring a broad range of health outcomes. To ensure generalizability, pragmatic trials should represent the intended patients to whom the treatment will be applied as best as possible. For instance, inclusion criteria would be broad (e.g. allowing co-morbidity, co-medication, wider age range, etc.), the follow-up would not be (or not much) interventional and allowing for treatment switching etc. (See also “large simple trials” and “real-world studies”).

 

Purpose of Pragmatic Clinical Trials

Pragmatic clinical trials seek to answer important questions that are applicable to everyday clinical practice (Williams et al., 2015):

  • The design of pragmatic trials aims to test an intervention in a study environment that is closer to real life in terms of study population, intervention, comparator, and outcomes.
  • Pragmatic trials must still adhere to the stringent trial methods for minimizing selection, performance, information, attrition, selective outcome reporting, and publication bias.
  • Pragmatic trials must be prospectively registered and reported fully according to the pragmatic trials extension of the CONSORT statement.
  • ThePRECIS-2 tool is one method for assessing where on the pragmatic– explanatory continuum a trial resides and which aspects are more pragmatic or explanatory.

Limitations of Pragmatic Clinical Trials

Williams et al., (2015) list the limitations of pragmatic trials:

  • Pragmatic clinical trials can cost more than explanatory trials, and may require a more complex study design.
  • The majority of clinical trials are neither entirely pragmatic nor entirely explanatory—they are part of a continuum.
  • Pragmatic trials are not suitable for early trials that seek to explore whether a new experimental intervention shows any biological effect.