cluster randomized trials

June 12, 2020: A Cluster Randomized Pragmatic Trial of an Advance Care Planning Video Intervention in Long-Stay Nursing Home Residents: Main Findings from the PROVEN Trial (Susan Mitchell, MD, MPH)

Posted on by Gina Uhlenbrauck

Speaker

Susan L. Mitchell, MD, MPH
Senior Scientist
Hebrew SeniorLife
Hinda and Arthur Marcus Institute for Aging Research
Professor of Medicine
Harvard Medical School

Topic

A Cluster Randomized Pragmatic Trial of an Advance Care Planning Video Intervention in Long-Stay Nursing Home Residents: Main Findings from the PROVEN Trial

Keywords

Embedded pragmatic trial; PROVEN; Advance care planning; Nursing homes; Video intervention; Medicare; Care preferences; Decision support tool; Minimum data set; Intention to treat

Key Points

  • The PROVEN trial was the first large-scale embedded pragmatic trial conducted in nursing homes.
  • The advance care planning (ACP) video intervention in PROVEN was meant as an adjunct to first-person discussions with the clinical care provider.
  • The levels of care preferences described in the ACP videos were life prolongation, limited care, and comfort care.
  • PROVEN’s primary outcome was the number of transfers to the hospital from the nursing home.

Discussion Themes

Widely adoptable, effective interventions to improve ACP in nursing homes remain elusive.

Of the challenges of conducting PCTs embedded in nursing homes, it is important not to overlook the real-world priorities of stakeholders. A high level of endorsement, from C-suite to frontline care providers, is needed before attempting such a trial.

While a priority for nursing home administrators is the number of residents who transfer to the hospital, an essential question for patients and palliative care experts is whether patients receive care that matches their goals and preferences. This is hard to ascertain in a pragmatic way.

Read more about the PROVEN trial, and learn about a new research initiative built on the success of the NIH Collaboratory: the National Institute on Aging’s IMPACT Collaboratory, which is directly funding pilots of embedded PCTs across diverse healthcare settings to improve the care of patients with dementia and their caregivers.

Tags

#pctGR, @Collaboratory1

June 10, 2020: PROVEN Trial Will Report Main Findings During NIH Collaboratory Grand Rounds

Posted on by Damon Seils

A co–principal investigator of the Pragmatic Trial of Video Education in Nursing Homes (PROVEN), a recently completed NIH Collaboratory Trial, will report the main outcomes of the study during NIH Collaboratory Grand Rounds this week.

The PROVEN team developed video-assisted decision support tools for advance care planning that showed efficacy in small randomized controlled trials. The goal of PROVEN was to evaluate the effectiveness of those video tools in real-world nursing home settings by embedding the intervention in 2 large healthcare systems that operate 492 nursing homes nationwide.

Dr. Susan Mitchell of Harvard Medical School will present “A Cluster Randomized Pragmatic Trial of an Advance Care Planning Video Intervention in Long-Stay Nursing Home Residents: Main Findings from the PROVEN Trial.” The Grand Rounds session will be held on Friday, June 12, at 1:00 pm eastern. Join the online meeting.

PROVEN is supported within the NIH Collaboratory by a cooperative agreement from the National Institute on Aging.

May 28, 2020: New Updates to Design and Analysis Plan Chapters in the Living Textbook

Posted on by Damon Seils

The annual update of the Living Textbook has brought new content and organization to the Experimental Designs and Analysis Plan chapters. We invite you to explore these chapters and the external resources linked from the resources sidebar in each section.

The NIH Collaboratory Coordinating Center regularly refreshes content in the Living Textbook to improve the robust collection of resources it offers to the wider research community about how to plan and implement pragmatic clinical trials.

Sections of the Experimental Designs and Randomization Schemes chapter include:

  • Statistical Design Considerations
  • Cluster Randomized Trials
  • Randomization Methods
  • Choosing Between Cluster and Individual Randomization
  • Alternative Cluster Randomized Designs
  • Concealment and Blinding
  • Designing to Avoid Identification Bias
  • Additional Resources

Sections of the Analysis Plan chapter include:

  • Intraclass Correlation
  • Unequal Cluster Sizes
  • Accounting for Residual Confounding in the Analysis
  • Missing Data and Intention-to-Treat Analyses
  • EHR Data Extraction
  • Unanticipated Changes
  • Case Study: STOP CRC Trial

April 23, 2020: New Workshop Summary on the Design and Analysis of Pragmatic Clinical Trials

Posted on by Karen Staman

In 2019, NIH Health Care Systems Research Collaboratory held a comprehensive workshop to explore and discuss statistical issues encountered with embedded pragmatic clinical trials (ePCTs). The new Workshop Summary describes panel discussions with the principal investigators and statisticians of NIH Collaboratory Trials and the challenges and solutions encountered during the design and analysis of their trials.

The 4 panel discussions covered the following topics:

  • Measurement and Data: Outcomes, Exposures, and Subgroups Based on EHR Data
  • To Cluster or Not to Cluster?
  • Choosing a Parallel Group or Stepped-Wedge Design
  • Unique Complications

This Workshop Summary also provides lessons learned and recommends tools to help others design and analyze future ePCTs. For more on the design and analysis of pragmatic clinical trials, see the tools provided by the Biostatistics and Study Design Core and Living Textbook chapters on Experimental Designs and Randomization Schemes and Analysis Plans.

March 30, 2020: New Living Textbook Chapter Describes Ways to Monitor Intervention Fidelity and Adaptations During the Conduct of ePCTs

Posted on by Gina Uhlenbrauck

The new Monitoring Intervention Fidelity and Adaptations chapter of the Living Textbook was developed to introduce how to evaluate changes that may be encountered while conducting an embedded pragmatic clinical trial (ePCT). For example, a health system might experience competing clinical initiatives; turnover in leadership, clinicians, or staff; changes in technologies; new clinical practice guidelines; or regulatory changes.

The chapter offers strategies for study teams to anticipate, monitor, and document adaptations to the intervention in order to support study analysis and set the stage for dissemination and implementation of successful interventions in other healthcare settings. The chapter was developed by experts from the Collaboratory’s Health Care Systems Interactions Core, along with principal investigators conducting ePCT Demonstration Projects.

Topics include:

  • Anticipating changes that might impact intervention fidelity
  • Frameworks to assist in monitoring fidelity and adaptations
  • Strategies and case examples from the NIH Collaboratory Demonstration Projects
  • Pointers to additional resources

We encourage you to explore this new content.

 

March 17, 2020: Cheat Sheet on the Intraclass Correlation Coefficient

Posted on by Karen Staman

The NIH Collaboratory Biostatistics and Study Design Core has created an Intraclass Correlation Coefficient (ICC) Cheat Sheet to provide an introductory description of the ICC, which is important for the design and analysis of cluster-randomized trials.

“The intraclass correlation coefficient (ICC) is a descriptive statistic that describes the extent to which outcomes 1) within each cluster are likely to be similar or 2) between different clusters are likely to be different from each other, relative to outcomes from other clusters. The ICC is an important tool for cluster-randomized pragmatic trials because this value helps determine the sample size needed to detect a treatment effect.” —from the ICC Cheat Sheet

The tool is a 2-page handout that can be used in trainings or classes regarding pragmatic clinical trials involving cluster randomization.

For more on the ICC, see the Intraclass Correlation section in the Living Textbook or this in-depth working document on the ICC from the Biostatistics and Study Design Core. If you have questions, feedback or suggestions regarding this tool, please contact us at nih-collaboratory@dm.duke.edu.

March 4, 2020: FM TIPS Explores Novel Fibromyalgia Treatment in Physical Therapy Practices: An Interview With Dr. Kathleen Sluka and Dr. Leslie Crofford

Posted on by Damon Seils

The recently completed Fibromyalgia Activity Study With TENS (FAST) established the efficacy of transcutaneous electrical nerve stimulation (TENS) for the treatment of musculoskeletal pain in patients with fibromyalgia. Physical therapists generally are trained in the use of TENS, but the technique is underused in clinical practice.

The Fibromyalgia TENS in Physical Therapy Study (FM TIPS), a new NIH Collaboratory Demonstration Project, will assess the feasibility of adding TENS to routine physical therapy for patients with fibromyalgia. In addition to evaluating symptom improvement, the study will measure the effect of TENS on adherence to physical therapy, achieving therapeutic goals, and medication use.

At the NIH Collaboratory PRISM kickoff meeting in November, we asked co–principal investigators Dr. Kathleen Sluka and Dr. Leslie Crofford to discuss the rationale for their study and the value of engaging with a network of experts in pragmatic clinical trials.

“Delivering [the intervention] and seeing how difficult it is to use in a clinical practice on a day-to-day basis—and delivering it to any patient who happens to have [the condition]—seems to me to be the best way to find out if it’s really going to work in clinical practice,” explained Dr. Sluka.

Dr. Crofford added, “What we’re aiming to do is to make it easy for the clinician to choose these nonpharmacologic strategies for treatment of pain that improve both symptom and function in patients with fibromyalgia.”

FM TIPS is a project of the PRISM program (Pragmatic and Implementation Studies for the Management of Pain to Reduce Opioid Prescribing), part of the NIH’s Helping to End Addiction Long-term (HEAL) Initiative. The NIH Collaboratory serves as the PRISM Resource Coordinating Center.

“The Collaboratory has a wealth of expertise across multiple domains that can help us implement this and give us the knowledge we need in order to make this a successful trial,” said Dr. Sluka.

“We don’t think that anybody’s ever tried to do a pragmatic trial in physical therapy practice. So this is something that we hope that we can share back with the Collaboratory about how do you do this in nontraditional settings for research. We’re hoping to learn, and we hope they learn from us,” said Dr. Crofford.

FM TIPS is supported by the National Institute of Arthritis and Musculoskeletal and Skin Diseases. The NIH Collaboratory PRISM Resource Coordinating Center is supported by the National Center for Complementary and Integrative Medicine. Support is also provided by the NIH Common Fund through a cooperative agreement from the Office of Strategic Coordination within the Office of the NIH Director.

February 28, 2020: Preparing for the Unknown: Conducting Pragmatic Research in Real-World Contexts

Posted on by Gina Uhlenbrauck

Speakers

Vincent Mor, PhD
Florence Pirce Grant University Professor
Department of Health Services Policy & Practice
Brown University School of Public Health

Leah Tuzzio, MPH
Research Associate
Kaiser Permanente Washington Health Research Institute

Jeffrey (Jerry) G. Jarvik MD MPH
Professor, Radiology, Neurological Surgery and Health Services
Adjunct Professor, Pharmacy and Orthopedics & Sports Medicine
University of Washington

Topic

Preparing for the Unknown: Conducting Pragmatic Research in Real-World Contexts

Keywords

Embedded research; Implementation; Healthcare systems; Nursing homes; Radiology reports; Electronic health records; Data collection; Pragmatic clinical trials

Key Points

  • Early engagement between study teams and health system leaders and staff can help ease the embedded intervention into the clinical workflow. Consider, for example, who will deliver the intervention and how difficult it will be to implement.
  • In the pilot phase, show that you can implement the intervention effectively. For example, demonstrate that you can collect the outcome data you will need within the pilot’s time frame. Ensure that your study questions are important to the partner healthcare system.

Discussion Themes

It is essential to anticipate, identify, and make a plan to address changes in the healthcare system as your trial is being conducted.

Among the NIH Collaboratory Trials, many have good measures of how much site-to-site variation there is, which helps to understand what the preconditions are for high levels of implementation.

Use the pilot study to maximize acceptability, maintain affordability, and consider the scalability of the intervention.

These topics and more are on the Living Textbook of Pragmatic Clinical Trials. Plan to hear the next topics in the Living Textbook Grand Rounds series.

Tags
#pctGR, @Collaboratory1

February 24, 2020: Study Snapshots for Six UH3 NIH Collaboratory Trials Are Available

Posted on by Gina Uhlenbrauck

Want to know more about the goals and challenges of the NIH Collaboratory Trials? New downloadable handouts summarize the study aims, implementation lessons, and recent publications and presentations from these innovative pragmatic clinical trials.

Study Snapshots are accessible from each NIH Collaboratory Trial page and the links below:

  • ACP PEACE: Improving Advance Care Planning: Promoting Effective and Aligned Communication in the Elderly
  • EMBED: Pragmatic Trial of User-Centered Clinical Decision Support to Implement Emergency Department-Initiated Buprenorphine for Opioid Use Disorder
  • GGC4H: Guiding Good Choices for Health: Testing Feasibility and Effectiveness of Universal Parent-Focused Prevention in Three Healthcare Systems
  • HiLo: Pragmatic Trial of Higher vs. Lower Serum Phosphate Targets in Patients Undergoing Hemodialysis
  • Nudge: Personalized Patient Data and Behavioral Nudges to Improve Adherence to Chronic Cardiovascular Medications
  • PRIM-ER: Primary Palliative Care for Emergency Medicine

January 31, 2020: Living Textbook Grand Rounds Series Part 1: Pragmatic Clinical Trials: How Do I Start? (Lesley H. Curtis, PhD, Greg Simon, MD, MPH)

Posted on by Gina Uhlenbrauck

Speakers

Greg Simon, MD, MPH
Senior Investigator
Kaiser Permanente Washington Health Research Institute

Lesley H. Curtis, PhD
Chair and Professor
Department of Population Health Sciences
Duke University School of Medicine
Interim Executive Director, Duke Clinical Research Institute

Topic

Pragmatic Clinical Trials: How Do I Start?

Keywords

Pragmatic clinical trials; PRECIS-2; Real-world evidence; Health systems research; Stakeholders; Clinical workflow; Study team

Key Points

  • Embedded pragmatic clinical trials (ePCTs) are large, efficient studies conducted in the real world that provide evidence for adoption of an intervention into clinical practice.
  • ePCTs are conducted in partnership with healthcare systems, use streamlined procedures and existing infrastructure, and answer important medical questions. However, high relevance to real-world decision-making can sometimes come at the expense of trial efficiency.
  • The PRECIS-2 scores are not absolute virtues; rather, the tool helps researchers determine if their trial is fit for purpose based on their study question.
  • For greater generalizability, ePCTs should be conducted in a diverse range of patients, and study results should be reported transparently.

Discussion Themes

How might we support health systems that serve more diverse populations to participate in a pragmatic clinical trial?

What concerns might be voiced by health system leaders regarding potential reputational risk of a PCT, and perhaps downstream issues about the results publication?

The question “Can everyone do this study?” is different from “Can everyone believe the research results?”

To see upcoming topics in the Living Textbook Grand Rounds series, download the flyer and share with your colleagues and institution. To learn more about the fundamentals of designing and launching a successful ePCT visit the Living Textbook.