PragmaTrial®

Hippocrates Research New Business Unit for Pragmatic Studies

PragmaTrial® was created as a response to the growing challenges of modern clinical research.
With their methodology and application, pragmatic studies play a key role in generating Real-World Evidence (RWE) by bridging the gap between traditional Randomised Controlled Trials (RCTs) and “real life” itself.
This approach addresses the limitations of observational studies, producing more reliable, interpretable, and efficient Real-World Evidence – an increasingly essential resource for Regulatory Authorities in their decision-making processes.
The update to the ICH guideline for Good Clinical Practice (GCP) and the adoption of Annex 2 mark a paradigm shift in clinical research, moving from prescriptive rules to a more flexible approach that makes innovation more accessible and better aligned with the needs of future beneficiaries.

What is a Pragmatic Study?

A Pragmatic Study is an experimental study and, specifically, a randomised controlled trial (RCT), designed and conducted to evaluate the Effectiveness of a treatment. Since it is characterised by “external validity”, it can be considered a source of Real-World Evidence.
In contrast, classic RCTs are characterised by “internal validity”, as they investigate Efficacy for the purposes of hypothesis testing.
Pragmatic studies, which are typically open-label, therefore, provide a more comprehensive view of how a treatment works outside a laboratory setting, i.e., in real-world clinical practice conditions.

What is the purpose of a Pragmatic Study?

The purpose of a pragmatic study is to comparatively evaluate the real-world effectiveness of a treatment (Real-World Effectiveness, or Comparative Effectiveness Research) or the safety of a product, either in the marketing authorisation request phase or in the post-marketing phase.

The pragmatic approach allows for expanding clinical evaluations to more diverse populations, which are more representative of the real world, by assessing important outcomes, such as remission, recovery, or survival, through a direct comparison with the “standard of care”, in a real-world clinical practice setting. It also uses outcome measures that are useful to both patients and decision makers, including pharmacoeconomic evaluations.

Pragmatism

Pragmatism is not a rigid concept: on the contrary, it can be adapted to the specific objectives of a research study.
A study design may incorporate pragmatic aspects, for instance in terms of eligibility criteria, alongside more explanatory aspects, such as in terms of closer monitoring of treatment compliance. The modular design of pragmatic studies, therefore, is particularly important, as it:

  • Allows for balancing internal and external validity in a targeted manner;
  • Maintains the necessary rigour in key aspects while preserving generalisability, where possible;
  • Helps optimise resources, concentrating them where they are most needed.
Pragmatism should therefore be intended as a flexible tool serving the objectives of the study, not as a rigid constraint to be uniformly applied. This modular approach helps design studies that are both scientifically sound and clinically relevant.

Our multidisciplinary team

Our team is composed of experts belonging to different areas, such as Medical Directorate Personnel, Clinical Operation Specialists, Quality Managers, Market Advisors, Regulatory Compliance Managers and RWE experts.
Along with these experts, we also rely on a network of General Practitioners (GPs) and Primary Care Paediatricians (PCPs), qualified for phase III and IV studies, as well as operators for the management of data quality.

A MORE FLEXIBLE APPROACH AS COMPARED TO TRADITIONAL RCTs

FASTER TIME-TO-MARKET AND PRODUCT DISTRIBUTION

MORE SOLID EVIDENCE AS COMPARED TO OBSERVATIONAL STUDIES

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