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Clinical research is moving toward studies that matter in everyday care and pragmatic trials are at the forefront of that change. At their core, these trials ask a straightforward but powerful question: Does this intervention work in everyday clinical settings? That question changes everything, from how trials are designed to how their results are interpreted and used.
Unlike traditional trials that test efficacy in ideal, tightly controlled environments, pragmatic trials aim to evaluate effectiveness in the real world. They include broader, more representative populations, make use of existing care infrastructure, and focus on outcomes that genuinely matter in daily clinical decision-making like hospital admissions, treatment adherence, and overall patient well-being.
Both the FDA and EMA have recognized the value of this approach, supporting it through initiatives such as Project Pragmatica1 and ACT EU2. Still, the shift in mindset has been slow. Most clinical programs continue to default to conventional RCT models, not because the idea of pragmatic trials is flawed, but because the systems needed to support them remain underdeveloped.
The core distinction lies in intent and design. Explanatory trials ask: Can this treatment work under optimal conditions? Pragmatic trials ask: Does it work in the actual environments where care is delivered?
To answer this, researchers draw on tools like the PRECIS-2 framework3, which helps calibrate trial design decisions across nine domains — from eligibility criteria to recruitment, follow-up, and primary outcomes. Pragmatic trials tend to:
Still, the transition from concept to execution is where most efforts stumble.
Pragmatic trials bring a lot to the table, but they are not free from challenges. Here is where the toughest ones still stand.
A recent pragmatic trial conducted within the UK’s National Health Service (NHS) aimed to evaluate a chronic obstructive pulmonary disease (COPD) therapy4 in routine care settings. The design was ambitious include diverse patients, capture data across both primary care and hospital systems, and minimize disruption to everyday clinical workflows.
However, the execution exposed persistent infrastructure limitations. Clinical data was scattered across non-uniform electronic health record (EHR) systems, with inconsistent coding and limited interoperability. Sites faced delays due to incompatible platforms, manual reconciliation of clinical events, and lack of real-time visibility into trial metrics. Even with additional investment in data integration and training, the process remained slow and resource-intensive highlighting how fragmented health IT systems continue to undermine the scalability of pragmatic trial designs.
Pragmatic trials are often praised for their ability to bring research into real-world settings, but this shift comes with its own set of challenges. Community clinics, general practices, and non-research hospitals rarely have the research infrastructure found in academic medical centers. These sites may lack trained coordinators, familiarity with trial procedures, or systems for data capture and monitoring.
In the NHS COPD trial4, many sites required significant operational support just to get up and running. Protocol training had to be simplified, digital tools had to be introduced to streamline documentation, and additional staff were needed to manage patient follow-up. Without this level of hands-on support, even the best-designed trials risk stalling before enrollment begins. The trial’s experience underscores a key lesson: operational friction at the site level is one of the most underestimated barriers to pragmatic research.
One of the strongest promises of pragmatic trials is their ability to expand access, reaching people who have long been left out of traditional research. By operating in everyday care settings, these studies are better positioned to include rural patients, older adults, and underrepresented communities that rarely walk through the doors of academic trial centers.
But so far, that promise remains mostly unfulfilled. The FDA’s 2023 Drug Trials Snapshots report shows that participation by non-White populations still falls short across most therapeutic areas. Pragmatic designs could help close this gap but only if equity is treated as a core feature, not a side benefit8.
That means going beyond traditional site selection. It means investing in community-based partnerships, using mobile and remote tools that lower access barriers, and embedding diversity considerations into the trial from the ground up. Without these deliberate choices, even the most flexible designs risk repeating the same patterns of exclusion.
In 2023, the S2302 Pragmatica-Lung Study, launched by the FDA’s Oncology Center of Excellence, marked a turning point. Built on the infrastructure of the existing LUNG-MAP platform, this study removed unnecessary complexity—no biopsies, no central imaging, and only two primary endpoints: overall survival and serious adverse events.
What set this study apart was not only its simplicity but it was also how approachable it felt. By focusing on what truly mattered to patients and clinicians, it enabled faster site activation, broader participation, and alignment with routine care workflows. According to FDA officials, it’s a template for how pragmatic trials can be executed without compromising scientific credibility.
For pragmatic trials to become a staple of clinical development, the shift has to be more than philosophical—it must be infrastructural, operational, and strategic. That means:
These are not small tasks. But without them, the field will remain stuck between vision and viability.
At Maxis Clinical Sciences, the groundwork for pragmatic trial delivery is already visible in how we execute real-world oncology programs. In a recent metastatic breast cancer initiative, our teams built a multi-phase health economic model using patient-level data from diverse real-world sources. This model didn’t measure the cost of treatment, it tracked what really happens in everyday care, from changes in treatment plans to survival outcomes, capturing details that standard trials often miss.
This kind of infrastructure linking real-world data to meaningful outcomes mirrors the demands of pragmatic clinical trial design. It enables:
While not structured as a formal pragmatic RCT, the program illustrates Maxis’ ability to operationalize many of the same principles: flexibility in data sourcing, alignment with everyday care workflows, and a focus on outcomes that resonate with patients, clinicians, and payers alike.
In this way, our oncology RWE work doesn’t just complement pragmatic trials it actively helps sponsors move toward them, helping sponsors bridge feasibility with impact.
Pragmatic clinical trials have moved beyond being a new idea, they are now a critical part of how we advance medical research. They mark a meaningful shift toward studies that reflect everyday clinical practice, diverse patient populations, and the realities of care delivery. But adopting this approach isn’t just about being open to change. It takes coordination across teams, well-prepared data systems, and a thoughtful approach to simplifying trial design without compromising on quality. With growing regulatory support and better tools now available, sponsors have a clear opportunity: to lead by example and help make pragmatic trials a more routine part of clinical development. The tools exist. The frameworks are in place. What is needed now is action.
