Rethinking Randomized Clinical Trials for Comparative Effectiveness Research: The Need for Transformational Change

Luce, Bryan R.; Kramer, Judith M.; Goodman, Steven N.; Connor, Jason T.; Tunis, Sean; Whicher, Danielle; Schwartz, J. Sanford

doi:10.7326/0003-4819-151-3-200908040-00126

Cited by 302 publications

(225 citation statements)

References 15 publications

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“…The need for better evidence of comparative clinical effectiveness in ESRD is great, yet high costs limit our ability to investigate all research questions with an RCT (49). When an RCT is not immediately realizable, a simulated trial may provide reasonable effect estimates to test a research hypothesis, determine study feasibility, and help leverage funding for future randomized trials.…”

Section: Discussionmentioning

confidence: 99%

Modeling the 4D Study

Chan

Thadhani

Lazarus

et al. 2010

Clinical Journal of the American Society of Nephrology

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Background and objectives: Randomized, controlled trials (RCTs) are the gold standard for defining causal inferences but are sometimes not feasible because of cost, ethical, or time considerations. We explored the accuracy and potential use of a "simulated trial" through the modeling of a previously published RCT, Die Deutsche Diabetes Dialyse Studie (4D Study), a landmark study that investigated the cardiovascular benefit of atorvastatin use in 1255 patients with ESRD.Design, setting, participants, & measurements: Using a large historical database of interventions and outcomes in dialysis patients, we conducted an observational model of the 4D Study in dialysis patients who had type 2 diabetes and were prescribed a statin (5144 patients) and matched to a non-statin user (5144 control subjects) before multivariate modeling. Inclusion, exclusion, and outcome parameters of the study, as prespecified by the 4D Study, were strictly modeled in this analysis.Results: In covariate-and propensity-adjusted Cox regression, statin use (versus nonuse) was associated with a decrease in the composite primary outcome of cardiac death, nonfatal myocardial infarction, and stroke. Statin use was also associated with a decrease in cardiovascular mortality and all cardiac events combined. The hazard ratios in this observational model were numerically comparable to the hazard ratios reported in the 4D Study; however, because of the larger number of patients "enrolled," results in this simulated study achieved statistical significance.Conclusions: Statin use was associated with some cardiovascular benefit in a simulated trial of patients with ESRD; however, the size of benefit was considerably smaller than that seen in the general population. Such simulated trials may represent an exploratory, cost-effective option when RCTs are not immediately feasible.

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Section: Discussionmentioning

confidence: 99%

Modeling the 4D Study

Chan

Thadhani

Lazarus

et al. 2010

Clinical Journal of the American Society of Nephrology

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“…Evidence generation involves a variety of methods including clinical trials, 28 practical trials, 29,30 and observational studies. 31,32 Some but not all of the engagement activities in this stage depends on complex conceptual or methodological expertise.…”

Section: What Roles and Responsibilities Can Stakeholders Have In Pcomentioning

confidence: 99%

A New Taxonomy for Stakeholder Engagement in Patient-Centered Outcomes Research

Concannon

Meißner

Grunbaum³

et al. 2012

J GEN INTERN MED

392

343

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Despite widespread agreement that stakeholder engagement is needed in patient-centered outcomes research (PCOR), no taxonomy exists to guide researchers and policy makers on how to address this need. We followed an iterative process, including several stages of stakeholder review, to address three questions: (1) Who are the stakeholders in PCOR? (2) What roles and responsibilities can stakeholders have in PCOR? (3) How can researchers start engaging stakeholders? We introduce a flexible taxonomy called the 7Ps of Stakeholder Engagement and Six Stages of Research for identifying stakeholders and developing engagement strategies across the full spectrum of research activities. The path toward engagement will not be uniform across every research program, but this taxonomy offers a common starting point and a flexible approach.

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“…The determination of this central content is not a new activity for scientists. Prioritization and determination of best practices are central objectives of comparative effectiveness research (27,28), standard-of-care guidelines (29), and cost-benefit analyses (30), to name a few. For a message to stick in the minds of the intended audience, it needs to be simple, and simplicity requires prioritizing what information is in the message and what is left out (6).…”

Section: Obstacles To Creating Memorable and Actionable Guidelinesmentioning

confidence: 99%

Communicating science-based recommendations with memorable and actionable guidelines

Ratner

Riis

2014

Proc. Natl. Acad. Sci. U.S.A.

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For many domains of basic and applied science, a key set of scientific facts is well established and there is a need for public action in light of those facts. However, individual citizens do not consistently follow science-based recommendations, even when they accept the veracity of the advice. To address this challenge, science communicators need to develop a guideline that individuals can commit to memory easily and act on straightforwardly at moments of decision. We draw on research from psychology to discuss several characteristics that will enhance a guideline's memorability and actionability and illustrate using a case study from the US Department of Agriculture's communications based on nutrition science. We conclude by discussing the importance of careful research to test whether any given guideline is memorable and actionable by the intended target audience.cognitive psychology | consumer behavior | social psychology I n many domains of basic and applied science, a key set of scientific facts is well-established, and there is a need for public action in light of those facts. And yet, individual citizens do not consistently follow science-based recommendations, even when they accept the veracity of the advice. The complexity of daily life and the limited capacity of the human mind prevent important science-based recommendations from getting the intended behavior change. To address this challenge, we argue that a specific type of communication tool is needed to promote a scientific field's most important recommendations. In this review paper, we refer to this tool as the memorable and actionable guideline.Because of the cognitive limitations of memory and attention that we review in this article, science-based recommendations must be focused on influencing the moments when key consumer decisions are likely to be made. People do what they do in their daily lives, and communications should be structured so that they can influence particular key moments, whether it is food choices at mealtime, energy use at home in the evening, intervening when you see a friend about to do something dangerous, etc. Recommendations must be memorable at those key moments and must be usable/actionable at those key moments.The challenge of getting people to take action is one that has interested psychologists and communications experts for decades. Research on attitude change demonstrates that persuasive communications need to convince people that the scientific facts are credible and that the science-based recommendations are sound. However, even once the audience is convinced that the facts and recommendations are legitimate, behavior change will not necessarily follow. For example, even though most Americans think it is important to be ready for emergencies, only a minority have taken the necessary actions to be prepared for a disaster (1). Science communicators need to activate people's behaviors to incorporate the science-based recommendations into their lives. Given this major challenge, considerable research in fields ra...

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Rethinking Randomized Clinical Trials for Comparative Effectiveness Research: The Need for Transformational Change

Cited by 302 publications

References 15 publications

Modeling the 4D Study

Modeling the 4D Study

A New Taxonomy for Stakeholder Engagement in Patient-Centered Outcomes Research

Communicating science-based recommendations with memorable and actionable guidelines

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