Pragmatic Free Trial Meta
Pragmatic Free Trial Meta is a non-commercial open data platform and infrastructure that supports research on pragmatic trials. It collects and distributes cleaned trial data, ratings and evaluations using PRECIS-2. This allows for diverse meta-epidemiological analyses to compare treatment effect estimates across trials of various levels of pragmatism.
Background
Pragmatic trials are increasingly acknowledged as providing evidence from the real world to support clinical decision-making. The term "pragmatic", however, is not used in a consistent manner and its definition and measurement require clarification. The purpose of pragmatic trials is to inform policy and clinical practice decisions, rather than confirm a physiological or clinical hypothesis. A pragmatic study should strive to be as close as is possible to real-world clinical practices that include recruiting participants, setting, design, delivery and implementation of interventions, determination and analysis outcomes, and primary analysis. This is a major distinction from explanation trials (as described by Schwartz and Lellouch1), which are designed to provide more complete confirmation of a hypothesis.
The trials that are truly pragmatic should be careful not to blind patients or clinicians, as this may result in bias in estimates of the effects of treatment. Pragmatic trials will also recruit patients from different health care settings to ensure that the outcomes can be compared to the real world.
Additionally the focus of pragmatic trials should be on outcomes that are important for patients, such as quality of life or functional recovery. This is particularly relevant when it comes to trials that involve the use of invasive procedures or potentially serious adverse events. The CRASH trial29, for example focused on the functional outcome to evaluate a two-page case report with an electronic system to monitor the health of hospitalized patients with chronic heart failure. In addition, the catheter trial28 focused on urinary tract infections caused by catheters as its primary outcome.
In addition to these features, pragmatic trials should minimize the procedures for conducting trials and requirements for data collection to reduce costs. Furthermore pragmatic trials should strive to make their results as relevant to actual clinical practice as they can by making sure that their primary analysis is the intention-to-treat approach (as described in CONSORT extensions for pragmatic trials).
Despite these criteria, many RCTs with features that challenge the concept of pragmatism have been mislabeled as pragmatic and published in journals of all types. This can lead to misleading claims about pragmatism, and the use of the term should be made more uniform. The creation of a PRECIS-2 tool that provides an objective, standardized evaluation of pragmatic aspects is a good start.
Methods
In a pragmatic trial, the aim is to inform policy or clinical decisions by demonstrating how the intervention can be implemented into routine care. Explanatory trials test hypotheses concerning the cause-effect relationship within idealised conditions. Consequently, pragmatic trials may have less internal validity than explanatory trials and may be more susceptible to bias in their design, conduct and analysis. Despite these limitations, pragmatic trials can provide valuable information to decisions in the context of healthcare.
The PRECIS-2 tool evaluates the degree of pragmatism in an RCT by assessing it on 9 domains that range from 1 (very explicit) to 5 (very pragmatic). In this study the areas of recruitment, organization and flexibility in delivery, flexibility in adherence, and follow-up were awarded high scores. However, the primary outcome and the method of missing data scored below the pragmatic limit. This suggests that a trial can be designed with good practical features, yet not damaging the quality.
However, it is difficult to determine how practical a particular trial really is because pragmaticity is not a definite attribute; some aspects of a trial may be more pragmatic than others. A trial's pragmatism could be affected by changes to the protocol or the logistics during the trial. In addition 36% of 89 pragmatic trials identified by Koppenaal and co. were placebo-controlled or conducted prior to licensing and most were single-center. They are not close to the standard practice, and can only be called pragmatic if their sponsors agree that these trials are not blinded.
A typical feature of pragmatic studies is that researchers try to make their findings more meaningful by analyzing subgroups within the trial sample. This can result in unbalanced analyses that have less statistical power. This increases the possibility of omitting or ignoring differences in the primary outcomes. This was the case in the meta-analysis of pragmatic trials due to the fact that secondary outcomes were not corrected for covariates that differed at the baseline.
Furthermore the pragmatic trials may be a challenge in the gathering and interpretation of safety data. This is due to the fact that adverse events are usually self-reported and are susceptible to reporting delays, inaccuracies, or coding variations. It is essential to improve the quality and accuracy of the results in these trials.
Results
Although the definition of pragmatism doesn't require that clinical trials be 100% pragmatic, there are benefits to including pragmatic components in trials. These include:
By incorporating routine patients, the trial results can be more quickly translated into clinical practice. However, pragmatic trials can also have disadvantages. The right kind of heterogeneity for instance could allow a study to generalise its findings to many different patients or settings. However the wrong type of heterogeneity could decrease the sensitivity of the test and, consequently, decrease the ability of a study to detect even minor effects of treatment.
Several studies have attempted to classify pragmatic trials using a variety of definitions and scoring methods. Schwartz and Lellouch1 created a framework to distinguish between research studies that prove a clinical or physiological hypothesis as well as pragmatic trials that inform the selection of appropriate therapies in the real-world clinical setting. The framework was comprised of nine domains that were scored on a scale of 1 to 5 with 1 indicating more lucid and 5 indicating more practical. The domains included recruitment of intervention, setting up, delivery of intervention, flex compliance and primary analysis.
The original PRECIS tool3 was built on the same scale and domains. Koppenaal et al10 created an adaptation of this assessment called the Pragmascope which was more user-friendly to use in systematic reviews. They discovered that pragmatic systematic reviews had a higher average scores across all domains, with lower scores in the primary analysis domain.
This distinction in the analysis domain that is primary could be due to the fact that most pragmatic trials analyze their data in the intention to treat way however some explanation trials do not. The overall score for pragmatic systematic reviews was lower when the areas of management, flexible delivery and follow-up were merged.
It is crucial to keep in mind that a pragmatic study should not mean a low-quality trial. In fact, there are an increasing number of clinical trials that use the term 'pragmatic' either in their abstract or title (as defined by MEDLINE, but that is not precise nor sensitive). The use of these terms in abstracts and titles could indicate a greater understanding of the importance of pragmatism but it isn't clear if this is manifested in the content of the articles.
Conclusions
In recent years, pragmatic trials are gaining popularity in research as the value of real world evidence is increasingly recognized. They are randomized clinical trials that evaluate real-world alternatives to care rather than experimental treatments under development, they include patient populations that are more similar to those treated in routine care, they use comparators that are used in routine practice (e.g., existing medications), and they depend on the self-reporting of participants about outcomes. This method can help overcome the limitations of observational studies which include the limitations of relying on volunteers and limited availability and coding variability in national registry systems.
프라그마틱 공식홈페이지 have advantages, such as the ability to leverage existing data sources, and a greater probability of detecting meaningful differences than traditional trials. However, pragmatic trials may have some limitations that limit their credibility and generalizability. For example, participation rates in some trials could be lower than anticipated due to the healthy-volunteer effect and incentives to pay or compete for participants from other research studies (e.g. industry trials). A lot of pragmatic trials are limited by the need to enroll participants in a timely manner. Certain pragmatic trials lack controls to ensure that the observed variations aren't due to biases in the trial.
The authors of the Pragmatic Free Trial Meta identified RCTs that were published between 2022 and 2022 that self-described as pragmatic. They evaluated pragmatism using the PRECIS-2 tool, which consists of the domains eligibility criteria, recruitment, flexibility in adherence to intervention and follow-up. 프라그마틱 추천 found 14 trials scored highly pragmatic or pragmatic (i.e. scoring 5 or more) in at least one of these domains.
Trials that have a high pragmatism score tend to have higher eligibility criteria than traditional RCTs that have specific criteria that are not likely to be present in clinical practice, and they include populations from a wide variety of hospitals. According to the authors, may make pragmatic trials more useful and useful in the daily clinical. However they do not guarantee that a trial will be free of bias. The pragmatism characteristic is not a fixed attribute the test that doesn't have all the characteristics of an explicative study can still produce valid and useful outcomes.