Pragmatic Free Trial Meta
Pragmatic Free Trail Meta is an open data platform that allows research into pragmatic trials. It collects and distributes cleaned trial data, ratings and evaluations using PRECIS-2. This allows for diverse meta-epidemiological analyses that examine the effect of treatment across trials of various levels of pragmatism.
Background
Pragmatic studies are increasingly recognized as providing real-world evidence for clinical decision-making. However, the usage of the term "pragmatic" is inconsistent and its definition and evaluation requires further clarification. Pragmatic trials are intended to guide clinical practices and policy decisions, not to confirm a physiological hypothesis or clinical hypothesis. A pragmatic trial should aim to be as similar to actual clinical practice as possible, including in the participation of participants, setting and design, the delivery and execution of the intervention, as well as the determination and analysis of outcomes as well as primary analyses. This is a significant difference between explanatory trials, as defined by Schwartz and Lellouch1 which are designed to prove a hypothesis in a more thorough way.
Studies that are truly pragmatic must be careful not to blind patients or clinicians in order to lead to bias in estimates of the effects of treatment. The trials that are pragmatic should also try to attract patients from a wide range of health care settings, so that their results are generalizable to the real world.
Additionally, 프라그마틱 공식홈페이지 should be focused on outcomes that matter to patients, such as the quality of life and functional recovery. This is especially important in trials that require the use of invasive procedures or could have dangerous adverse consequences. The CRASH trial29, for instance focused on the functional outcome to compare a 2-page case-report with an electronic system for monitoring of hospitalized patients with chronic heart failure. In addition, the catheter trial28 focused on symptomatic catheter-associated urinary tract infections as its primary outcome.
In addition to these characteristics, pragmatic trials should minimize trial procedures and data-collection requirements to reduce costs and time commitments. Furthermore pragmatic trials should try to make their findings as relevant to actual clinical practice as they can by making sure that their primary method of analysis is the intention-to-treat approach (as described in CONSORT extensions for pragmatic trials).
Despite these guidelines, many RCTs with features that challenge the concept of pragmatism have been mislabeled as pragmatic and published in journals of all kinds. This could lead to misleading claims of pragmatism and the use of the term needs to be standardized. The development of a PRECIS-2 tool that can provide an objective, standardized evaluation of the pragmatic characteristics is a first step.
Methods
In a practical study it is the intention to inform clinical or policy decisions by demonstrating how an intervention can be integrated into routine care in real-world situations. Explanatory trials test hypotheses concerning the cause-effect relation within idealized settings. In this way, pragmatic trials could have lower internal validity than studies that explain and be more susceptible to biases in their design, analysis, and conduct. Despite these limitations, pragmatic trials may be a valuable source of information for decisions in the context of healthcare.
The PRECIS-2 tool assesses the degree of pragmatism within an RCT by assessing it across 9 domains ranging from 1 (very explanatory) to 5 (very pragmatic). In this study, the recruit-ment organization, flexibility in delivery and follow-up domains received high scores, however the primary outcome and the procedure for missing data were below the pragmatic limit. This suggests that it is possible to design a trial using excellent pragmatic features without compromising the quality of its results.
It is difficult to determine the degree of pragmatism that is present in a trial because pragmatism does not have a binary attribute. Certain aspects of a study can be more pragmatic than other. Furthermore, logistical or protocol modifications during the course of a trial can change its pragmatism score. In addition, 36% of the 89 pragmatic trials identified by Koppenaal et al were placebo-controlled, or conducted prior to licensing and most were single-center. They are not in line with the usual practice, and can only be referred to as pragmatic if their sponsors accept that such trials are not blinded.
Additionally, a typical feature of pragmatic trials is that researchers try to make their results more relevant by analyzing subgroups of the trial sample. This can lead to unbalanced analyses that have lower statistical power. This increases the risk of omitting or ignoring differences in the primary outcomes. In the case of the pragmatic trials included in this meta-analysis, this was a serious issue because the secondary outcomes weren't adjusted for differences in the baseline covariates.
Furthermore, pragmatic studies can present challenges in the collection and interpretation safety data. This is due to the fact that adverse events are typically self-reported, and are prone to delays, inaccuracies or coding errors. Therefore, it is crucial to enhance the quality of outcomes assessment in these trials, ideally by using national registries instead of relying on participants to report adverse events in a trial's own database.
Results
While the definition of pragmatism does not require that all clinical trials be 100% pragmatic There are advantages to including pragmatic components in trials. These include:
Incorporating routine patients, the results of trials can be more quickly translated into clinical practice. However, pragmatic studies can also have disadvantages. For instance, the appropriate type of heterogeneity could help a trial to generalise its findings to a variety of settings and patients. However, the wrong type of heterogeneity could reduce assay sensitiveness and consequently decrease the ability of a study to detect even minor effects of treatment.
A variety of studies have attempted to categorize pragmatic trials, with various definitions and scoring systems. Schwartz and Lellouch1 have developed a framework that can differentiate between explanation studies that support the physiological hypothesis or clinical hypothesis and pragmatic studies that guide the selection of appropriate treatments in the real-world clinical practice. The framework was composed of nine domains that were assessed on a scale of 1-5 which indicated that 1 was more explanatory while 5 was more practical. The domains were recruitment, setting, intervention delivery with flexibility, follow-up and primary analysis.
The initial PRECIS tool3 featured similar domains and an assessment scale ranging from 1 to 5. Koppenaal et al10 devised an adaptation of this assessment dubbed the Pragmascope that was easier to use in systematic reviews. They found that pragmatic reviews scored higher on average in all domains, but scored lower in the primary analysis domain.
This difference in the analysis domain that is primary could be due to the fact that most pragmatic trials process their data in an intention to treat way while some explanation trials do not. The overall score was lower for systematic reviews that were pragmatic when the domains on the organization, flexibility of delivery and follow-up were merged.
It is important to remember that the term "pragmatic trial" does not necessarily mean a poor quality trial, and in fact there is an increasing rate of clinical trials (as defined by MEDLINE search, however this is neither specific nor sensitive) which use the word "pragmatic" in their abstract or title. The use of these words in abstracts and titles may suggest a greater awareness of the importance of pragmatism, but it isn't clear if this is reflected in the contents of the articles.
프라그마틱 공식홈페이지
In recent years, pragmatic trials are becoming more popular in research as the value of real-world evidence is becoming increasingly acknowledged. They are clinical trials that are randomized which compare real-world treatment options instead of experimental treatments in development. They involve patient populations which are more closely resembling those treated in routine care, they employ comparators which exist in routine practice (e.g. existing medications), and they rely on participant self-report of outcomes. This method is able to overcome the limitations of observational research, such as the biases associated with the reliance on volunteers, as well as the insufficient availability and coding variations in national registries.
Other advantages of pragmatic trials include the ability to use existing data sources, and a higher chance of detecting meaningful changes than traditional trials. However, pragmatic trials may have some limitations that limit their reliability and generalizability. For instance the participation rates in certain trials might be lower than expected due to the healthy-volunteer effect and incentives to pay or compete for participants from other research studies (e.g., industry trials). The requirement to recruit participants in a timely manner also limits the sample size and impact of many pragmatic trials. Some pragmatic trials also lack controls to ensure that any observed variations aren't due to biases during the trial.
The authors of the Pragmatic Free Trial Meta identified RCTs published up to 2022 that self-described as pragmatic. The PRECIS-2 tool was used to assess the degree of pragmatism. It includes domains such as eligibility criteria, recruitment flexibility as well as adherence to interventions and follow-up. They found that 14 trials scored highly pragmatic or pragmatic (i.e. scoring 5 or higher) in at least one of these domains.
Trials with high pragmatism scores tend to have more criteria for eligibility than conventional RCTs. They also contain populations from various hospitals. These characteristics, according to the authors, can make pragmatic trials more useful and useful in everyday practice. However, they don't guarantee that a trial will be free of bias. In addition, the pragmatism that is present in the trial is not a definite characteristic; a pragmatic trial that doesn't have all the characteristics of an explanatory trial can yield reliable and relevant results.
