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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 -
Journal of Clinical Epidemiology Jan 2020
Topics: Diagnostic Tests, Routine; Humans; Pragmatic Clinical Trials as Topic; Research Design
PubMed: 31924314
DOI: 10.1016/j.jclinepi.2019.12.004 -
Contemporary Clinical Trials Aug 2022Home-based testing for COVID-19 has potential to reduce existing health care disparities among underserved populations in the United States. However, implementation of...
BACKGROUND
Home-based testing for COVID-19 has potential to reduce existing health care disparities among underserved populations in the United States. However, implementation of home-based tests in these communities may face significant barriers. This study evaluates the acceptability, feasibility, and success of home-based testing and the potential added benefit of active support from trusted community health workers for Native Americans and Hispanic/Latino adults living in rural Montana and Washington states.
METHODS/DESIGN
The academic-community research team designed the trial to be responsive to community needs for understanding barriers and supports to home-based COVID-19 testing. The "Protecting Our Community" study is a two-arm pragmatic randomized controlled trial in which a total of 400 participants are randomized to active or passive arms. Participants of both study arms receive a commercially available home collection COVID-19 test kit, which is completed by mailing a self-collected nasal swab to a central laboratory. The primary study outcome is return of the kit to the central lab within 14 days. The cultural, social, behavioral, and economic barriers to home-based COVID-19 testing are also assessed by qualitative research methods. A survey and semi-structured interviews are conducted after the trial to evaluate perceptions and experience of home-based testing.
DISCUSSION
Implementing home-based testing in underserved populations, including among Native American and Hispanic/Latino communities, may require additional support to be successful. The Protecting Our Community trial examines the effect of trusted community health workers on use of home-based testing, which may be adaptable for community-driven models of home-based testing in other underserved populations.
Topics: COVID-19; COVID-19 Testing; Hispanic or Latino; Humans; Pragmatic Clinical Trials as Topic; Randomized Controlled Trials as Topic; SARS-CoV-2; United States; American Indian or Alaska Native
PubMed: 35691487
DOI: 10.1016/j.cct.2022.106820 -
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 -
NEJM Evidence Feb 2024Randomized controlled trials are the gold standard of clinical research for comparing therapies in well-defined groups of participants. Randomization avoids confounding...
Randomized controlled trials are the gold standard of clinical research for comparing therapies in well-defined groups of participants. Randomization avoids confounding due to unmeasured variables or to treatment selection and enables a causal interpretation of the estimated treatment effect. It has long been recognized, however, that standard explanatory clinical trials are slow, costly, and subject to participant selection. To preserve the strengths of randomized trials while mitigating their weaknesses, randomized clinical trials emerged; these trials aim to facilitate decision-making rather than explicate a mechanism of action and enroll a diverse set of participants using existing structures and data sources..
Topics: Humans; Research Design; Patient Selection; Registries; Causality; Randomized Controlled Trials as Topic
PubMed: 38320494
DOI: 10.1056/EVIDe2300310 -
Trials Dec 2019All clinical trial investigators have ethical and regulatory obligations to monitor participant safety and trial integrity. Specific procedures for meeting these...
BACKGROUND
All clinical trial investigators have ethical and regulatory obligations to monitor participant safety and trial integrity. Specific procedures for meeting these obligations, however, may differ substantially between pragmatic trials and traditional explanatory clinical trials.
METHODS/RESULTS
Appropriate monitoring of clinical trials typically includes assessing rate of recruitment or enrollment; monitoring safe and effective delivery of study treatments; assuring that study staff act to minimize risks; monitoring quality and timeliness of study data; and considering interim analyses for early detection of benefit, harm, or futility. Each of these responsibilities applies to pragmatic clinical trials. Just as design of pragmatic trials typically involves specific and necessary departures from methods of explanatory clinical trials, appropriate monitoring of pragmatic trials typically requires specific departures from monitoring procedures used in explanatory clinical trials. We discuss how specific aspects of pragmatic trial design and operations influence selection of monitoring procedures and illustrate those choices using examples from three ongoing pragmatic trials conducted by the Mental Health Research Network.
CONCLUSIONS
Pragmatic trial investigators should not routinely adopt monitoring procedures used in explanatory clinical trials. Instead, investigators should consider core principles of trial monitoring and design monitoring procedures appropriate for each pragmatic trial.
Topics: Adolescent; Adult; Aged; Data Accuracy; Humans; Middle Aged; Pragmatic Clinical Trials as Topic; Research Design; Young Adult
PubMed: 31815644
DOI: 10.1186/s13063-019-3869-3 -
Mayo Clinic Proceedings Aug 2023Clinical trials have been the bedrock of research to evaluate the safety and efficacy of new medical, surgical, or other interventions. Traditional "explanatory"... (Review)
Review
Clinical trials have been the bedrock of research to evaluate the safety and efficacy of new medical, surgical, or other interventions. Traditional "explanatory" clinical trials have aimed to explain a biological cause (new treatment) and effect (patient outcome) while controlling for many factors that might impact the evaluation, such as restricted eligibility criteria, frequent follow-up visits, and multiple clinical and laboratory measures. Despite the benefits of a well-controlled clinical trial, compromises have been made that can limit who might benefit from a new intervention, can increase complexity of the conduct of a trial, or that lead to excessively long durations of trials. An alternative approach to evaluate the effectiveness of an intervention is based on "pragmatic" clinical trials, which consider how an intervention affects a patient's condition in the real world, accounting for how to optimize an intervention within the operations of busy and diverse clinical practices. Although we describe explanatory and pragmatic trial designs as separate approaches, there is a continuum of approaches that intersect. Some key points are the need to maintain scientific rigor, increase efficiency of clinical trials operations, ensure that trial results can be generalized to a broad spectrum of patients, and balance the needs of real-world clinical care. Pragmatic trials can leverage technology and telecommunication strategies of decentralized trials to further reach underrepresented and underserved patients to close the health disparity gaps.
Topics: Humans; Research Design; Time Factors; Clinical Trials as Topic
PubMed: 37536808
DOI: 10.1016/j.mayocp.2023.04.013 -
PloS One 2022Child and family social workers in the UK work closely with other agencies including schools and the police, and typically they are based in local authority offices....
BACKGROUND
Child and family social workers in the UK work closely with other agencies including schools and the police, and typically they are based in local authority offices. This study will evaluate the effectiveness of placing social workers in schools (SWIS) on the need for social care interventions. SWIS was piloted in three local authorities in 2018-2020, and findings from a feasibility study of the pilots suggests SWIS may operate through three key pathways: (1) by enhancing schools' response to safeguarding issues, (2) through increased collaboration between social workers, school staff, and parents, and (3) by improving relationships between social workers and young people.
METHODS
The study is a two-arm pragmatic cluster randomised controlled trial building on three feasibility studies which found SWIS to be promising. Social workers will work within secondary schools across local authorities in England. 280 mainstream secondary schools will be randomly allocated with a 1:1 ratio to SWIS or a comparison arm, which will be schools that continue as normal, without a social worker. The primary outcome will be the rate of Child Protection (Section 47) enquiries. Secondary outcomes will comprise rate of referrals to children's social care, rate of Child in Need (Section 17) assessments, days spent in care, and educational attendance and attainment. The study also includes an economic evaluation, and an implementation and process evaluation. Social care outcomes will be measured in July 2022, and educational outcomes will be measured in July 2023. Days in care will be measured at both time points.
DISCUSSION
Findings will explore the effectiveness and cost-effectiveness of SWIS on the need for social care interventions. A final report will be published in January 2024.
TRIAL REGISTRATION
The study was registered retrospectively with the International Standard Randomised Controlled Trial Number registry on 13.11.2020 (ISRCTN90922032).
Topics: Adolescent; Child; Cost-Benefit Analysis; Feasibility Studies; Humans; Parents; Pragmatic Clinical Trials as Topic; Randomized Controlled Trials as Topic; Retrospective Studies; Schools; Social Work
PubMed: 35679281
DOI: 10.1371/journal.pone.0265354 -
Anesthesiology Jan 2020Large randomized trials provide the highest level of clinical evidence. However, enrolling large numbers of randomized patients across numerous study sites is expensive... (Review)
Review
Large randomized trials provide the highest level of clinical evidence. However, enrolling large numbers of randomized patients across numerous study sites is expensive and often takes years. There will never be enough conventional clinical trials to address the important questions in medicine. Efficient alternatives to conventional randomized trials that preserve protections against bias and confounding are thus of considerable interest. A common feature of novel trial designs is that they are pragmatic and facilitate enrollment of large numbers of patients at modest cost. This article presents trial designs including cluster designs, real-time automated enrollment, and practitioner-preference approaches. Then various adaptive designs that improve trial efficiency are presented. And finally, the article discusses the advantages of embedding randomized trials within registries.
Topics: Clinical Trials as Topic; Humans; Research Design
PubMed: 31809323
DOI: 10.1097/ALN.0000000000002989 -
Journal of Clinical Epidemiology Jan 2020Factorial designs can allow efficient evaluation of multiple treatments within a single trial. We evaluated the design, analysis, and reporting in a sample of factorial... (Review)
Review
OBJECTIVES
Factorial designs can allow efficient evaluation of multiple treatments within a single trial. We evaluated the design, analysis, and reporting in a sample of factorial trials.
STUDY DESIGN AND SETTING
Review of 2 × 2 factorial trials evaluating health-related interventions and outcomes in humans. Using Medline, we identified articles published between January 2015 and March 2018. We randomly selected 100 articles for inclusion.
RESULTS
Most trials (78%) did not provide a rationale for using a factorial design. Only 63 trials (63%) assessed the interaction for the primary outcome, and 39/63 (62%) made a further assessment for at least one secondary outcome. 12/63 trials (19%) identified a significant interaction for the primary outcome and 16/39 trials (41%) for at least one secondary outcome. Inappropriate methods of analysis to protect against potential negative effects from interactions were common, with 18 trials (18%) choosing the analysis method based on a preliminary test for interaction, and 13% (n = 10/75) of those conducting a factorial analysis including an interaction term in the model.
CONCLUSION
Reporting of factorial trials was often suboptimal, and assessment of interactions was poor. Investigators often used inappropriate methods of analysis to try to protect against adverse effects of interactions.
Topics: Clinical Trials as Topic; Data Interpretation, Statistical; Humans; Research Design
PubMed: 31585174
DOI: 10.1016/j.jclinepi.2019.09.018