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BMC Medicine Oct 2022Pragmatic trials aim to generate evidence to directly inform patient, caregiver and health-system manager policies and decisions. Heterogeneity in patient...
BACKGROUND
Pragmatic trials aim to generate evidence to directly inform patient, caregiver and health-system manager policies and decisions. Heterogeneity in patient characteristics contributes to heterogeneity in their response to the intervention. However, there are many other sources of heterogeneity in outcomes. Based on the expertise and judgements of the authors, we identify different sources of clinical and methodological heterogeneity, which translate into heterogeneity in patient responses-some we consider as desirable and some as undesirable. For each of them, we discuss and, using real-world trial examples, illustrate how heterogeneity should be managed over the whole course of the trial.
MAIN TEXT
Heterogeneity in centres and patients should be welcomed rather than limited. Interventions can be flexible or tailored and control interventions are expected to reflect usual care, avoiding use of a placebo. Co-interventions should be allowed; adherence should not be enforced. All these elements introduce heterogeneity in interventions (experimental or control), which has to be welcomed because it mimics reality. Outcomes should be objective and possibly routinely collected; standardised assessment, blinding and adjudication should be avoided as much as possible because this is not how assessment would be done outside a trial setting. The statistical analysis strategy must be guided by the objective to inform decision-making, thus favouring the intention-to-treat principle. Pragmatic trials should consider including process analyses to inform an understanding of the trial results. Needed data to conduct these analyses should be collected unobtrusively. Finally, ethical principles must be respected, even though this may seem to conflict with goals of pragmatism; consent procedures could be incorporated in the flow of care.
Topics: Humans; Pragmatic Clinical Trials as Topic; Randomized Controlled Trials as Topic; Research Design
PubMed: 36303153
DOI: 10.1186/s12916-022-02569-w -
Trials Jun 2020Traditional randomised controlled trials remain the gold standard for improving clinical care but they do have their limitations, including their associated high costs,... (Review)
Review
BACKGROUND
Traditional randomised controlled trials remain the gold standard for improving clinical care but they do have their limitations, including their associated high costs, high failure rate and limited external validity. An alternative methodology is the newly defined, prospective, registry-based randomised controlled trial (RRCT), where treatment and outcome data is collected in an existing registry. This scoping review explores the current literature regarding RRCTs to help identify the key design elements of RRCTs and the characteristics of clinical registries on which they are reliant on.
METHODS
A scoping review methodology conducted in accordance with the Joanna Briggs Institute guidelines was performed. Four databases were searched for articles published from inception to June 2018: Medline; Embase; the Cumulative Index to Nursing and Allied Health Literature and; Scopus. The search strategy included MeSH and text words related to RRCT.
RESULTS
We identified 2369 articles of which 75 were selected for full-text screening. Of these, only 17 articles satisfied our inclusion criteria. All studies were published between 1996 and 2017 and all were investigator-initiated. Study designs were mainly multi-site comparative/effectiveness studies incorporating the use of disease registries (n = 8), procedure registries (n = 8) and a health services registry (n = 1). The low cost, reduced administrative burden and enhanced external validity of RRCTs make them an attractive research methodology which can be used to address questions of public health importance. We identified that that there are variable definitions of what constituted a RRCT and that issues related to ethical conduct and data integrity, completeness, timeliness, validation and endpoint adjudication need to be carefully addressed.
CONCLUSION
RRCTs potentially have an important role to play in informing best clinical practice and health policy. There are a number of issues that need to be addressed to optimise the utility of this approach, including establishing universally accepted criteria for the definition of a RRCT.
Topics: Humans; Multicenter Studies as Topic; Pragmatic Clinical Trials as Topic; Prospective Studies; Registries; Research Design
PubMed: 32571382
DOI: 10.1186/s13063-020-04459-z -
Trials Jun 2009Randomised controlled trials are the best research design for decisions about the effect of different interventions but randomisation does not, of itself, promote the... (Review)
Review
Randomised controlled trials are the best research design for decisions about the effect of different interventions but randomisation does not, of itself, promote the applicability of a trial's results to situations other than the precise one in which the trial was done. While methodologists and trialists have rightly paid great attention to internal validity, much less has been given to applicability. This narrative review is aimed at those planning to conduct trials, and those aiming to use the information in them. It is intended to help the former group make their trials more widely useful and to help the latter group make more informed decisions about the wider use of existing trials. We review the differences between the design of most randomised trials (which have an explanatory attitude) and the design of trials more able to inform decision making (which have a pragmatic attitude) and discuss approaches used to assert applicability of trial results. If we want evidence from trials to be used in clinical practice and policy, trialists should make every effort to make their trial widely applicable, which means that more trials should be pragmatic in attitude.
Topics: Attitude of Health Personnel; Evidence-Based Medicine; Health Knowledge, Attitudes, Practice; Humans; Randomized Controlled Trials as Topic; Reproducibility of Results; Research Design; Treatment Outcome
PubMed: 19493350
DOI: 10.1186/1745-6215-10-37 -
Clinical Research in Cardiology :... Jan 2017Electronic health records (EHRs) provide opportunities to enhance patient care, embed performance measures in clinical practice, and facilitate clinical research.... (Review)
Review
Electronic health records (EHRs) provide opportunities to enhance patient care, embed performance measures in clinical practice, and facilitate clinical research. Concerns have been raised about the increasing recruitment challenges in trials, burdensome and obtrusive data collection, and uncertain generalizability of the results. Leveraging electronic health records to counterbalance these trends is an area of intense interest. The initial applications of electronic health records, as the primary data source is envisioned for observational studies, embedded pragmatic or post-marketing registry-based randomized studies, or comparative effectiveness studies. Advancing this approach to randomized clinical trials, electronic health records may potentially be used to assess study feasibility, to facilitate patient recruitment, and streamline data collection at baseline and follow-up. Ensuring data security and privacy, overcoming the challenges associated with linking diverse systems and maintaining infrastructure for repeat use of high quality data, are some of the challenges associated with using electronic health records in clinical research. Collaboration between academia, industry, regulatory bodies, policy makers, patients, and electronic health record vendors is critical for the greater use of electronic health records in clinical research. This manuscript identifies the key steps required to advance the role of electronic health records in cardiovascular clinical research.
Topics: Access to Information; Cardiovascular Diseases; Clinical Trials as Topic; Comparative Effectiveness Research; Confidentiality; Data Accuracy; Data Mining; Electronic Health Records; Humans; Medical Record Linkage; Research Design; Systems Integration
PubMed: 27557678
DOI: 10.1007/s00392-016-1025-6 -
Clinical Trials (London, England) Oct 2015The need for high-quality evidence to support decision making about health and health care by patients, physicians, care providers, and policy-makers is well documented....
The need for high-quality evidence to support decision making about health and health care by patients, physicians, care providers, and policy-makers is well documented. However, serious shortcomings in evidence persist. Pragmatic clinical trials that use novel techniques including emerging information and communication technologies to explore important research questions rapidly and at a fraction of the cost incurred by more "traditional" research methods promise to help close this gap. Nevertheless, while pragmatic clinical trials can bridge clinical practice and research, they may also raise difficult ethical and regulatory challenges. In this article, the authors briefly survey the current state of evidence that is available to inform clinical care and other health-related decisions and discuss the potential for pragmatic clinical trials to improve this state of affairs. They then propose a new working definition for pragmatic research that centers upon fitness for informing decisions about health and health care. Finally, they introduce a project, jointly undertaken by the National Institutes of Health Health Care Systems Research Collaboratory and the National Patient-Centered Clinical Research Network (PCORnet), which addresses 11 key aspects of current systems for regulatory and ethical oversight of clinical research that pose challenges to conducting pragmatic clinical trials. In the series of articles commissioned on this topic published in this issue of Clinical Trials, each of these aspects is addressed in a dedicated article, with a special focus on the interplay between ethical and regulatory considerations and pragmatic clinical research aimed at informing "real-world" choices about health and health care.
Topics: Biomedical Research; Clinical Trials as Topic; Decision Making; Humans; National Institutes of Health (U.S.); Research Design; Surveys and Questionnaires; United States
PubMed: 26374676
DOI: 10.1177/1740774515598334 -
Frontiers in Public Health 2022The effective translation of evidence-based interventions has contributed to implementing actions that impact public policies and the population's health. However, there...
Evaluating the implementation of the active life improving health behavior change program "BCP-VAMOS" in primary health care: Protocol of a pragmatic randomized controlled trial using the RE-AIM and CFIR frameworks.
INTRODUCTION
The effective translation of evidence-based interventions has contributed to implementing actions that impact public policies and the population's health. However, there is a gap in the literature regarding the factors associated with the successful implementation of these interventions. The Active Life Improving Health Behavior Change Program (BCP-VAMOS) uses behavioral strategies to promote an active and healthy lifestyle in the community. Characterized as a health innovation, it also provides health professionals with online training to implement the program in Primary Health Care (PHC). Our study describes a pragmatic trial that aims to evaluate the implementation of BCP-VAMOS, version 3.0, in PHC in southern Brazil.
METHODS AND ANALYSIS
A pragmatic randomized controlled trial (PRCT) of two arms comparing a group of PHC professionals who will participate in a traditional didactic approach (control group) vs. a group that will receive ongoing support (intervention group) for the implementation of BCP-VAMOS. The intervention will be available to adults (≥18 years old) registered at PHC. Program recipient's will be assessed at baseline and post-intervention (9 months after) to measure markers of physical activity and eating behavior (primary outcomes). Program's implementation process will be monitored for 12 months and will be evaluated using the RE-AIM and Consolidated Framework for Implementation Research (CFIR) frameworks.
DISCUSSIONS
The survey findings can be used widely throughout Brazil, guiding the work of health professionals, service planners and policy-makers. Also, the results may help to inform the national health promotion policy to plan interventions and improve the implementation of programs in PHC. This research results will provide practical guidance for researchers to develop similar protocols to implement and adapt public health interventions.
ETHICS AND DISSEMINATION
Ethics approval has been granted by the Human Research Ethics Committee of the Federal University of Santa Catarina (UFSC), Brazil, under no. 1394492. Results will be published in full as open access in the UFSC library and main trial results and associated papers in high-impact peer-reviewed journals.
TRIAL REGISTRATION NUMBER
RBR-2vw77q-Brazilian Registry of Clinical Trials - ReBEC (http://www.ensaiosclinicos.gov.br).
Topics: Adult; Exercise; Feeding Behavior; Health Behavior; Health Promotion; Humans; Pragmatic Clinical Trials as Topic; Primary Health Care; Randomized Controlled Trials as Topic
PubMed: 36172209
DOI: 10.3389/fpubh.2022.726021 -
Journal of General Internal Medicine May 2015Research on interventions within the standard of care has enormous potential, yet it also raises several ethical and regulatory challenges. Perhaps the most important is... (Comparative Study)
Comparative Study Review
Research on interventions within the standard of care has enormous potential, yet it also raises several ethical and regulatory challenges. Perhaps the most important is determining what consent process is needed for these "pragmatic" clinical trials. Some argue that pragmatic clinical trials need to obtain in-depth research consent. This approach ensures that patients are informed, but may introduce substantial selection bias and disruption of clinical care. Others argue that trials limited to interventions within the standard of care do not need to obtain research consent at all. While this approach avoids the problems with in-depth consent, it results in patients not knowing whether they are in research. The present manuscript proposes a way to avoid both sets of concerns. It argues that consent for research needs to supplement appropriate consent for standard care only to the extent that the research differs from standard care. Hence, pragmatic trials designed to mirror clinical care can obtain consent with only minimal additions to consent for standard care. This conclusion suggests that it may be possible for many pragmatic trials to obtain consent that is ethically appropriate, satisfies research regulations, and does not introduce substantial selection bias or clinical disruption.
Topics: Female; Humans; Informed Consent; Male; Needs Assessment; Patient Selection; Pragmatic Clinical Trials as Topic; United States
PubMed: 25586870
DOI: 10.1007/s11606-014-3169-2 -
Geriatric Nursing (New York, N.Y.) 2022Randomized controlled trials are considered the most rigorous research design in efficacy and effectiveness research; however, such trials present numerous challenges...
Randomized controlled trials are considered the most rigorous research design in efficacy and effectiveness research; however, such trials present numerous challenges that limit their applicability in real-world settings. As a consequence, pragmatic trials are increasingly viewed as a research design that overcomes some of these barriers with the potential to produce data that are more reproducible. Although pragmatic methodology in long-term care is receiving increasing attention as an approach to improve successful dissemination and implementation, pragmatic trials present complexities of their own. To address these complexities and related issues, experts with experience conducting pragmatic trials, developing nursing home policy, participating in advocacy efforts, and providing clinical care in long-term care settings participated in a virtual consensus conference funded by the National Institute on Aging in Spring 2021. Participants recommended 4 cross-cutting principles key to dissemination and implementation of pragmatic trial interventions: (1) engage stakeholders, (2) ensure diversity and inclusion, (3) assess organizational strain and readiness, and (4) learn from adaptations. Specifically related to implementation, participants provided 2 recommendations: (1) integrate interventions into existing workflows and (2) maintain agility and responsiveness. Finally, participants had 3 recommendations specific to dissemination: (1) package the message for the audience, (2) engage diverse audiences, and (3) apply dissemination and diffusion tools. Participants emphasized that implementation processes must be grounded in the perspectives of the people who will ultimately be responsible for implementing the intervention once it is proven to be effective. In addition, messaging must speak to long-term care staff and all others who have a stake in its outcomes. Although our understanding of dissemination and implementation strategies remains underdeveloped, this article is designed to guide long-term care researchers and community providers who are increasingly aware of the need for pragmatism in disseminating and implementing evidence-based care interventions.
Topics: Humans; Long-Term Care; Nursing Homes; Pragmatic Clinical Trials as Topic
PubMed: 35219533
DOI: 10.1016/j.gerinurse.2022.02.006 -
BMJ Open Feb 2022The reduction of coercion in psychiatry is a high priority for both the WHO and many member countries. Open-door policy (ODP) is a service model for psychiatric ward...
Protocol for the Lovisenberg Open Acute Door Study (LOADS): a pragmatic randomised controlled trial to compare safety and coercion between open-door policy and usual-care services in acute psychiatric inpatients.
INTRODUCTION
The reduction of coercion in psychiatry is a high priority for both the WHO and many member countries. Open-door policy (ODP) is a service model for psychiatric ward treatment that prioritises collaborative and motivational measures to better achieve acute psychiatric safety - and treatment objectives. Keeping the ward main door open is one such measure. Evidence on the impact of ODP on coercion and violent events is mixed, and only one randomised controlled trial (RCT) has previously compared ODP to standard practice. The main objectives of the Lovisenberg Open Acute Door Study (LOADS) are to implement and evaluate a Nordic version of ODP for acute psychiatric inpatient services. The evaluation is designed as a pragmatic RCT with treatment-as-usual (TAU) control followed by a 4-year observational period.
METHODS AND ANALYSIS
In this 12-month pragmatic randomised trial, all patients referred to acute ward care will be randomly allocated to either TAU or ODP wards. The primary outcome is the proportion of patient stays with one or more coercive measures. Secondary outcomes include adverse events involving patients and/or staff, substance use and users' experiences of the treatment environment and of coercion. The main hypothesis is that ODP services will not be inferior to state-of-the art psychiatric treatment. ODP and TAU wards are determined via ward-level randomisation. Following conclusion of the RCT, a longitudinal observational phase begins designed to monitor any long-term effects of ODP.
ETHICS AND DISSEMINATION
The trial has been approved by the Regional Committees for Medical and Health Research Ethics (REC) in Norway (REC South East #29238), who granted LOADS exemption from consent requirements for all eligible, admitted patients. Data are considered highly sensitive but can be made available on request. Results will be published in peer-reviewed journals and presented at scientific conferences and meetings.
TRIAL REGISTRATION NUMBER
ISRCTN16876467.
PROTOCOL VERSION
1.4, 21 December 2021.
Topics: Coercion; Hospitalization; Humans; Inpatients; Policy; Pragmatic Clinical Trials as Topic; Psychiatric Department, Hospital; Randomized Controlled Trials as Topic
PubMed: 35173011
DOI: 10.1136/bmjopen-2021-058501 -
Contemporary Clinical Trials Aug 2022Cigarette smoking prevalence is higher among rural compared with urban adults, yet access to cessation programming is reduced. The Increasing Digital Equity and Access...
BACKGROUND
Cigarette smoking prevalence is higher among rural compared with urban adults, yet access to cessation programming is reduced. The Increasing Digital Equity and Access (IDEA) study aims to evaluate three digital access and literacy interventions for promoting engagement with an online evidence-based smoking cessation treatment (EBCT) program among rural adults.
METHODS
The pilot trial will use a pragmatic, three-arm, randomized, parallel-group design with participants recruited from a Midwest community-based health system in Minnesota, Wisconsin, and Iowa. All participants will receive an online, 12-week, EBCT program, and written materials on digital access resources. Participants will be stratified based on state of residence and randomly assigned with 1:1:1 allocation to one of three study groups: (1) Control Condition-no additional study intervention (n = 30); (2) Loaner Digital Device-Bluetooth enabled iPad with data plan coverage loaned for the study duration (n = 30); (3) Loaner Digital Device + Coaching Support-loaner device plus up to six, 15-20 min motivational interviewing-based coaching calls to enhance participants' digital access and literacy (n = 30). All participants will complete study assessments at baseline and 4- and 12-weeks post-randomization. Outcomes are cessation program and trial engagement, biochemically confirmed smoking abstinence, and patient experience.
RESULTS
A rural community advisory committee was formed that fostered co-design of the study protocol for relevance to rural populations, including the trial design and interventions.
CONCLUSION
Study findings, processes, and resources may have relevance to other health systems aiming to foster digital inclusion in smoking cessation and chronic disease management programs and clinical trials in rural communities.
Topics: Adult; Humans; Minnesota; Pilot Projects; Pragmatic Clinical Trials as Topic; Randomized Controlled Trials as Topic; Rural Population; Smoking; Smoking Cessation
PubMed: 35760340
DOI: 10.1016/j.cct.2022.106838