Physicians’ perspectives regarding pragmatic clinical trials

Topazian, Rachel J.; Bollinger, Juli; Weinfurt, Kevin P.; Dvoskin, Rachel; Mathews, Debra J. H.; Brelsford, Kathleen M.; DeCamp, Matthew; Sugarman, Jeremy

doi:10.2217/cer-2016-0024

Cited by 12 publications

(12 citation statements)

References 18 publications

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“…Data are collected during the course of routine care, and partnerships must be built with healthcare providers to ensure that the intervention can be added into existing workflows as seamlessly and with as little burden as possible [ 42 ]. Physicians, nurses, and other clinical staff who will implement the protocol will have insight about the feasibility and sustainability of the intervention [ 43 ]. For example, the PPACT trial has an intervention that is delivered in the primary care setting where schedules are busy and space is tight.…”

Section: Results: Lessons Learnedmentioning

confidence: 99%

Pragmatic clinical trials embedded in healthcare systems: generalizable lessons from the NIH Collaboratory

Weinfurt

Hernandez

Coronado

et al. 2017

BMC Med Res Methodol

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142

118

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BackgroundThe clinical research enterprise is not producing the evidence decision makers arguably need in a timely and cost effective manner; research currently involves the use of labor-intensive parallel systems that are separate from clinical care. The emergence of pragmatic clinical trials (PCTs) poses a possible solution: these large-scale trials are embedded within routine clinical care and often involve cluster randomization of hospitals, clinics, primary care providers, etc. Interventions can be implemented by health system personnel through usual communication channels and quality improvement infrastructure, and data collected as part of routine clinical care. However, experience with these trials is nascent and best practices regarding design operational, analytic, and reporting methodologies are undeveloped.MethodsTo strengthen the national capacity to implement cost-effective, large-scale PCTs, the Common Fund of the National Institutes of Health created the Health Care Systems Research Collaboratory (Collaboratory) to support the design, execution, and dissemination of a series of demonstration projects using a pragmatic research design.ResultsIn this article, we will describe the Collaboratory, highlight some of the challenges encountered and solutions developed thus far, and discuss remaining barriers and opportunities for large-scale evidence generation using PCTs.ConclusionA planning phase is critical, and even with careful planning, new challenges arise during execution; comparisons between arms can be complicated by unanticipated changes. Early and ongoing engagement with both health care system leaders and front-line clinicians is critical for success. There is also marked uncertainty when applying existing ethical and regulatory frameworks to PCTS, and using existing electronic health records for data capture adds complexity.

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Section: Results: Lessons Learnedmentioning

confidence: 99%

Pragmatic clinical trials embedded in healthcare systems: generalizable lessons from the NIH Collaboratory

Weinfurt

Hernandez

Coronado

et al. 2017

BMC Med Res Methodol

Self Cite

142

118

View full text Add to dashboard Cite

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“…Engaging clinicians in research when they are not directly involved as investigators is a challenge that should be anticipated by researchers planning future pragmatic trials. [37][38][39] Our analysis of the variance within session duration categories suggests that for intervention facilities, the extent of intervention uptake was driven in large part by facility-level rather than patient-level factors. More work is needed to identify the relevant facility factors, which might include facility size, number of nephrologists, leadership approaches, staffing ratios and stability, and competing initiatives.…”

Section: Discussionmentioning

confidence: 99%

The TiME Trial: A Fully Embedded, Cluster-Randomized, Pragmatic Trial of Hemodialysis Session Duration

Dember

Lacson

Brunelli

et al. 2019

JASN

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BackgroundData from clinical trials to inform practice in maintenance hemodialysis are limited. Incorporating randomized trials into dialysis clinical care delivery should help generate practice-guiding evidence, but the feasibility of this approach has not been established.MethodsTo develop approaches for embedding trials into routine delivery of maintenance hemodialysis, we performed a cluster-randomized, pragmatic trial demonstration project, the Time to Reduce Mortality in ESRD (TiME) trial, evaluating effects of session duration on mortality (primary outcome) and hospitalization rate. Dialysis facilities randomized to the intervention adopted a default session duration ≥4.25 hours (255 minutes) for incident patients; those randomized to usual care had no trial-driven approach to session duration. Implementation was highly centralized, with no on-site research personnel and complete reliance on clinically acquired data. We used multiple strategies to engage facility personnel and participating patients.ResultsThe trial enrolled 7035 incident patients from 266 dialysis units. We discontinued the trial at a median follow-up of 1.1 years because of an inadequate between-group difference in session duration. For the primary analysis population (participants with estimated body water ≤42.5 L), mean session duration was 216 minutes for the intervention group and 207 minutes for the usual care group. We found no reduction in mortality or hospitalization rate for the intervention versus usual care.ConclusionsAlthough a highly pragmatic design allowed efficient enrollment, data acquisition, and monitoring, intervention uptake was insufficient to determine whether longer hemodialysis sessions improve outcomes. More effective strategies for engaging clinical personnel and patients are likely required to evaluate clinical trial interventions that are fully embedded in care delivery.

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“…In particular, questions of consent in pragmatic trials have been consistently raised as topics of interest and have been studied through vignette-based research with healthcare professionals and patients [ 36 , 37 ], interviews with physicians [ 39 ], deliberative engagement activities [ 42 ] and surveys [ 41 ]. For example, in a study using case vignettes, respondents preferred specific disclosure and options to either opt-in or opt-out of research compared to a general policy in which patients were informed broadly that a healthcare system engages in Comparative Effectiveness Research (CER) [ 37 , 42 ] .…”

Section: Introductionmentioning

confidence: 99%

“…Other studies have explored attitudes toward consent under different trial scenarios. This work found that attitudes toward consent differed not only by the type of intervention, but also due to different design elements such as randomization and the degree to which the trial design departed from routine clinical care [ 39 ].…”

Section: Introductionmentioning

confidence: 99%

Stakeholder views regarding ethical issues in the design and conduct of pragmatic trials: study protocol

et al. 2018

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BackgroundRandomized controlled trial (RCT) trial designs exist on an explanatory-pragmatic spectrum, depending on the degree to which a study aims to address a question of efficacy or effectiveness. As conceptualized by Schwartz and Lellouch in 1967, an explanatory approach to trial design emphasizes hypothesis testing about the mechanisms of action of treatments under ideal conditions (efficacy), whereas a pragmatic approach emphasizes testing effectiveness of two or more available treatments in real-world conditions. Interest in, and the number of, pragmatic trials has grown substantially in recent years, with increased recognition by funders and stakeholders worldwide of the need for credible evidence to inform clinical decision-making. This increase has been accompanied by the onset of learning healthcare systems, as well as an increasing focus on patient-oriented research. However, pragmatic trials have ethical challenges that have not yet been identified or adequately characterized. The present study aims to explore the views of key stakeholders with respect to ethical issues raised by the design and conduct of pragmatic trials. It is embedded within a large, four-year project that seeks to develop guidance for the ethical design and conduct of pragmatic trials. As a first step, this study will address important gaps in the current empirical literature with respect to identifying a comprehensive range of ethical issues arising from the design and conduct of pragmatic trials. By opening up a broad range of topics for consideration within our parallel ethical analysis, we will extend the current debate, which has largely emphasized issues of consent, to the range of ethical considerations that may flow from specific design choices.MethodsSemi-structured interviews with key stakeholders (e.g. trialists, methodologists, lay members of study teams, bioethicists, and research ethics committee members), across multiple jurisdictions, identified based on their known experience and/or expertise with pragmatic trials.DiscussionWe expect that the study outputs will be of interest to a wide range of knowledge users including trialists, ethicists, research ethics committees, journal editors, regulators, healthcare policymakers, research funders and patient groups. All publications will adhere to the Tri-Agency Open Access Policy on Publications.Electronic supplementary materialThe online version of this article (10.1186/s12910-018-0332-z) contains supplementary material, which is available to authorized users.

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Physicians’ perspectives regarding pragmatic clinical trials

Cited by 12 publications

References 18 publications

Pragmatic clinical trials embedded in healthcare systems: generalizable lessons from the NIH Collaboratory

Pragmatic clinical trials embedded in healthcare systems: generalizable lessons from the NIH Collaboratory

The TiME Trial: A Fully Embedded, Cluster-Randomized, Pragmatic Trial of Hemodialysis Session Duration

Stakeholder views regarding ethical issues in the design and conduct of pragmatic trials: study protocol

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