Pragmatic clinical trials play a unique role in medical research, as they enable interventions to be tested and assessed under routine clinical conditions, rather than the confines of a controlled environment.
We’ve examined pragmatic trials in more detail, exploring their advantages and disadvantages, how they’re conducted, and how you can take part.
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What is a pragmatic trial?
A pragmatic clinical trial (PCT) assesses the effectiveness of a new medical intervention in a real-world setting, such as a hospital or clinic.
The treatments are delivered flexibly, reflecting how they would be administered in regular practice. The focus of the trial is on practical outcomes, and the results directly inform future decision-making about a patient's care.
PCTs typically involve a wide range of participants, representing the population who could potentially be treated.
Advantages of pragmatic clinical trials
Flexibly testing interventions on a varied and reflective sample of patients increases the relevance and applicability of the results – which in turn enhances the external validity of the trial. In addition, a wider sample of participants can be involved to more accurately represent the general population.
In addition, pragmatic trials are administered in real-world healthcare settings, so there’s no need for additional resources or infrastructures. This keeps the costs down, and can also save time as patients are already awaiting treatment.
Not only that, findings from the research can inform how participants should be treated, and interventions can promptly be put in place. Connecting research with clinical care in this way can significantly improve outcomes for patients in real time.
Limitations to pragmatic trials
Although pragmatic clinical trials offer many benefits, they also present some challenges.
The participants and clinicians aren’t usually blinded, and researchers have limited control over the study – including how the intervention is administered and how strictly the study protocol is followed. Assessing outcomes in real-world settings can also be complex, and often rely on patient feedback. This all means that the accuracy and validity of the results could potentially be compromised.
On a practical level, when interventions are applied in routine clinical practice, patient consent, data privacy, and ethical conduct can be difficult to manage.
Coordinating several different stakeholders – including healthcare systems, providers, and patients – can also be a logistical challenge.
Interested in learning more about the benefits and risks of participating in clinical trials? Read all about what to consider before applying in our comprehensive guide
How are pragmatic trials different from traditional clinical trials?
Pragmatic trials differ from traditional trials in several beneficial ways.
Traditional clinical trials are conducted in a controlled research environment, whereas pragmatic trials are undertaken in real-world environments and settings. As such, the results may be more applicable and relevant to regular clinical practice.
Pragmatic trials can also benefit from a wider and larger pool of participants, ensuring a broader sample of the population is more accurately represented. Plus the eligibility criteria are often more inclusive and flexible, making it easier for more people from the wider population to participate.
In addition, pragmatic trials allow for interventions to be administered flexibly rather than following a standardized protocol.
Finally, pragmatic trials focus on the impact the intervention has on the patient's health, quality of life, and wellbeing, whereas traditional trials often focus on biomarker or clinical endpoints.
What is randomization in pragmatic clinical trials?
As with randomization in traditional clinical trials, randomization in pragmatic trials involves placing participants into treatment groups at random. This method aims to create a broad participant sample that represents the wider patient population, with results that apply to real-life healthcare settings. Ethically, it ensures a fair selection process and avoids discrimination or favoritism.
How are pragmatic clinical trials conducted?
Similarly to other clinical trials, pragmatic trials administer medical interventions, observe the effects, collect data, and carefully analyze the outcomes.
Interventions and their delivery within a pragmatic trial
The interventions tested in pragmatic trials include medications, clinical procedures, and behavioral treatments. It’s important that they’re delivered in a consistent and systematic way that follows the study protocol to ensure accurate and reliable results – although some flexibility is usually allowed due to them being applied in real world settings. Careful planning and delivery of these interventions helps researchers gather valid and meaningful data.
Pragmatic clinical trial follow-up procedures
Follow-up procedures are vital for tracking the patient's response to the intervention, and assessing whether they’ve experienced any adverse reactions, side-effects, or complications. This might involve frequent check-ups and tests to gather data and make observations.
Additional feedback about the trial itself and the participant’s experience may also be sought, as this can help researchers shape and improve future trials.
The analysis of pragmatic trial outcomes
Once the data is gathered, researchers have the challenging task of assessing and evaluating the data, and forming conclusions about the intervention. The ultimate aim is to assess whether the treatment is safe and effective, and can be disseminated to the wider population.
This requires a detailed understanding of the trial design, the sample of patients, and the unique contexts in which the interventions were delivered. Any potential biases or confounders must also be taken into consideration.
Participating in a pragmatic clinical trial
By participating in a pragmatic trial, you can play a vital role in advancing medical research and improving healthcare for future patients – and potentially yourself.
Pragmatic trials aim to reflect real-world clinical practice, and researchers will often work with healthcare providers to find participants. As such, your medical practitioner may be aware of relevant studies.
Several online databases also promote trials that are seeking new participants, or you may see adverts online that are targeted at a specific audience.
When you’ve located an appropriate trial, check that you meet eligibility criteria. For example, you may need to be within a certain age range, or have a specific health condition in order to apply.
Before the trial begins, you will be asked to give your informed consent to confirm you understand what the trial involves, and any potential risks. As such, it’s vital that you read all the study materials in advance, and raise any concerns or queries with the research team.
During the trial, you will be given the appropriate intervention, then closely monitored and asked to share your feedback. You are also likely to undergo tests to gather relevant data.
Benefits and risks of participating in a pragmatic clinical trial
Understanding the potential risks and benefits of pragmatic clinical trials can help you make an informed decision about whether to take part.
Potential benefits
By participating in a pragmatic trial, you may be one of the first people to benefit from innovative new treatments that aren’t yet available to the general public. You might also be monitored more closely than usual, and receive more comprehensive and tailored care.
Many people take part in pragmatic trials because they also want to help others. By contributing to research in this way, you can play a crucial part in advancing medical research and developing new treatments.
Risks and considerations
As with any medical treatment, the interventions tested in pragmatic trials do present the risk of side-effects or adverse reactions. It’s vital to gain a thorough understanding of the potential risks before you take part, and consider them alongside the potential benefits.
Due to the very nature of a trial, the results are never guaranteed. As such, it’s important to manage your expectations and understand that you may not get the desired outcome.
You should also be aware that pragmatic trials might involve more frequent visits to your healthcare provider, alongside additional tests, so take these into account and ensure you can fit them into your regular routine.
Other types of clinical trial
In addition to pragmatic trials, several other studies are also used for medical research, each with its own benefits.
- Observational trials involve gathering participant data without administering any interventions. They can be incredibly helpful for monitoring the progression of a disease within the general population and identifying potential risk factors.
- Placebo-controlled trials involve administering a placebo to one group of participants, and the actual treatment to another, which helps researchers compare the effects and obtain less biased results.
- Single arm clinical studies involve one just one group of participants who all receive the same medical intervention, and are closely monitored to assess the outcomes.
- Crossover trials enable multiple interventions to be tested on a participant over a length of time. This is often regarded as a more efficient way of assessing more than one treatment.
- Adaptive trials are a flexible and efficient approach to clinical studies, enabling the trial design to change based on preliminary results.
- Medical pilot studies involve testing new drugs, procedures, or devices on a small group of patients to assess their impact. The results then inform larger and more comprehensive trials.
- Non inferiority clinical trials compare the effectiveness of a new treatment against an existing treatment to ensure it is not inferior.
Conclusion
Unlike other studies, pragmatic trials take place under regular clinical conditions, delivering results that accurately reflect how a treatment would impact patients in the real world.
They also focus on patient outcomes, which means the participant’s health is of primary concern, and people can benefit from new treatments that aren’t available to the wider population. Volunteers are also contributing to the advancement of important medical discoveries, and potentially enhancing lives.
Find out more about how to take part in a pragmatic clinical trial.