June 14, 2018: NIH Collaboratory Members to Host Session on Embedded PCTs at AcademyHealth Annual Research Meeting

The upcoming AcademyHealth Annual Research Meeting, June 24-26, 2018, will feature a special session on Embedding Pragmatic Clinical Trials in Health Care Systems: Trials and Tribulations, Monday, June 25, at 5:15 pm, hosted by members of the Health Care Systems Interactions Core Working Group:

Chair: Cathy Meyers, MD, NIH/NICCH

Discussant: Eric B. Larson, MD, MPH, Kaiser Permanente Washington Health Research Institute

Speakers: Vince Mor, PhD, Brown University School of Public Health; Greg Simon, MD, MPH, Kaiser Permanente Washington Health Research Institute; and Lynn DeBar, PhD, MPH, Kaiser Permanente Washington Health Research Institute

Held at the Washington State Convention Center in Seattle, this large gathering of health services researchers and policy analysts will include workshops, poster and podium sessions, emerging issues panels, policy roundtables, and special topic sessions. Other NIH Collaboratory members planning to present at the meeting include Leah Tuzzio, MPH, Jerry Jarvik, MD, MPH, Miguel Vazquez, MD, Kathryn James, MPH, Gloria Coronado, PhD, and Beverly Green, MD, MPH.

 “Attendees will learn about the challenges of pilot testing, studying patient-reported outcomes, using existing data, and the multiple levels of implementation in dynamic systems.” – Leah Tuzzio, Kaiser Permanente Washington Health Research Institute

For details, visit the meeting event site at http://www.academyhealth.org/events/site/2018-annual-research-meeting.

June 4, 2018: New Article Explores Misleading Use of the Label “Pragmatic” for Some Randomized Clinical Trials

A recent study published in BMC Medicine found that many randomized controlled trials (RCTs) self-labeled as “pragmatic” were actually explanatory in nature, in that they assessed investigational medicines compared with placebo to test efficacy before licensing. Of the RCTs studied, one-third were pre-licensing, single-center, or placebo-controlled trials and thus not appropriately described as pragmatic.

Appropriately describing the design and characteristics of a pragmatic trial helps readers understand the trial’s relevance for real-world practice. The authors explain that RCTs suitably termed pragmatic compare the effectiveness of 2 available medicines or interventions prescribed in routine clinical care. The purpose of such pragmatic RCTs is to provide real-world evidence for which interventions should be recommended or prioritized.

The authors recommend that investigators use a standard tool, such as the CONSORT Pragmatic Trials extension or the PRECIS-2 tool, to prospectively evaluate the pragmatic characteristics of their RCTs. Use of these tools can also assist funders, ethics committees, and journal editors in determining whether an RCT has been accurately labeled as pragmatic.

The BMC Medicine article cites NIH Collaboratory publications by Ali et al. and Johnson et al., as well as the Living Textbook, in its discussion of pragmatic RCTs and the tools available to assess their relevance for real-world practice.

“Submissions of RCTs to funders, research ethics committees, and peer-reviewed journals should include a PRECIS-2 tool assessment done by the trial investigators. Clarity and accuracy on the extent to which an RCT is pragmatic will help [to] understand how much it is relevant to real-world practice.” (Dal-Ré et al. 2018)

April 24, 2018: NIH Collaboratory ePCT Training Workshop Resources Now Available

Interested in learning more about the complexities of designing and conducting embedded pragmatic clinical trials (ePCTs)? Now you can view and download the slide sets and handouts from the first NIH Collaboratory ePCT Training Workshop. Held on February 20-21, 2018, at Duke University, this pilot workshop offered training in the conduct of ePCTs to 27 mid- and senior-level investigators. Also present at the workshop were representatives from the NIH, NIH Collaboratory Demonstration Projects and Core Working Groups, and Coordinating Center staff. NIH Collaboratory subject matter experts used experiences from the program and chapters from the Living Textbook to develop workshop content.

The workshop comprised 10 topics across the 2 days and included hands-on exercises and case studies from the Demonstration Projects illustrating the particulars of designing and conducting ePCTs and providing resources for planning and overcoming challenges. Among the topics covered were Engaging All Stakeholders and Aligning with Healthcare System Partners, Design and Analytic Considerations, Pilot and Feasibility Testing, and Developing a Compelling Application. The NIH Collaboratory will be making additional training resources available to the public in the near future.

The training was funded as an NIH Roadmap Initiative 3U54AT007748-05S2.

February 15, 2018: Timely Tips from Demonstration Project Principal Investigators

The NIH Collaboratory will soon welcome a new group of Demonstration Projects and guide them through the piloting and implementation phases of their embedded pragmatic clinical trials (ePCTs). We asked three seasoned principal investigators—Drs. Laura Dember, MD (TiME), Lynn DeBar, PhD (PPACT), and Jerry Jarvik, MD, MPH (LIRE)­—to share tips and advice for investigators who are new to ePCTs.

Download the PDF.

“Integrate really well with the key stakeholders from your sites.”
– Lynn DeBar, PhD

“Engage with senior mentors and people who have gone down this road before.” – Jerry Jarvik, MD, MPH

“Recognize that the perspectives of key stakeholders may be different than anticipated by investigators.” – Laura Dember, MD


November 20, 2017: NIH Collaboratory Core Working Group Interviews: Reflections from the Regulatory/Ethics Core

We recently asked Drs. Jeremy Sugarman and Kevin Weinfurt, Co-chairs of the Regulatory/Ethics Core, to reflect on the first 5 years of the Core as well as on the challenges ahead. The regulatory and ethical landscape for pragmatic clinical trials was not well defined when the Core began 5 years ago, and the Core helped to map and navigate the emerging landscape to enable the implementation of Demonstration Projects in ways that satisfied ethical and regulatory criteria.

“The Core’s work has led to the creation of a substantial body of scholarship contributing to the ongoing policy and ethics debates about pragmatic clinical trials.” – Drs. Sugarman and Weinfurt

Download the interview (PDF).

November 17, 2017: ICD-Pieces: From Planning to Performance


Miguel A. Vazquez, MD
Professor of Medicine
Clinical Chief Nephrology Division
University of Texas Southwestern Medical Center

George Oliver, MD
Vice President Clinical Informatics
Parkland Center for Clinical Innovation


ICD-Pieces: From Planning to Performance


Pragmatic clinical trial; Multiple chronic conditions; Diabetes; Hypertension; Chronic kidney disease; Pieces™; Parkland Center for Clinical Innovation; PCCI; University of Texas Southwestern Medical Center; Electronic health records

Key Points

  • ICD-Pieces is a pragmatic clinical trial conducted in 4 large, diverse healthcare systems, including a Veterans Affairs (VA) system.
  • This study is evaluating a collaborative care model for improving the management of 3 chronic conditions: diabetes, hypertension, and chronic kidney disease. This model of care combines a novel IT platform, primary care practitioners, and site practice facilitators.
  • The study is conducted with the aid of clinical decision support tools, previsit planning, performance monitoring, and outcomes ascertainment through EHR data and claims data.

Discussion Themes

It is essential to engage key stakeholders early and throughout the pragmatic trial to sustain enthusiasm for the trial’s outcomes. In ICD-Pieces, it was especially important to engage with individual healthcare practitioners to facilitate and simplify their workflow. The study team also collaborated with medical directors and other leaders at the participating healthcare systems.

Practice facilitators at each study site served as a link between the study team and the healthcare system operations.

As with many real-world pragmatic trials, ICD-Pieces needed to address some turnover of key study participants, including principal investigators, practice facilitators, and IT staff.

For More Information

Read more about the ICD-Pieces trial in the Living Textbook: http://bit.ly/2j1qkJu

Dr. Vazquez discusses lessons learned in ICD-Pieces: http://bit.ly/2zx8tEY

Visit the ICD-Pieces Demonstration Project page: http://bit.ly/2zQE1my

For more on dissemination, implementation & sustainability for ICD-Pieces: http://bit.ly/2A6zL5A


#pctGR, #chronicdisease, @PCTGrandRounds, @Collaboratory1, @PCORnetwork, @UWMedicine, @PCCIpieces

November 3, 2017: Dr. Miguel Vazquez Shares Lessons From the Improving Chronic Disease Management with Pieces (ICD-Pieces) Trial

In this interview, Dr. Miguel Vazquez gives an update on the first years of the Improving Chronic Disease Management with Pieces (ICD-Pieces) trial. Dr. Vazquez discussed the status of his trial, challenges and surprises, and advice he has for new investigators.

“Try to really learn from others who have done this—even if you are the first one doing your specific trial with your specific questions. It was helpful for us to learn from the other Collaboratory projects; they had already faced some problems, and we were able to anticipate and develop solutions proactively.” – Dr. Miguel Vazquez

Read more from Dr. Vazquez in the full interview (PDF).

October 18, 2017: NIH Collaboratory Core Working Group Interviews: Reflections from the Biostatistics and Study Design Core

We recently asked Dr. Liz DeLong, Chair of the Biostatistics and Study Design Core, to reflect on the first 5 years of the Core’s work and the challenges ahead. She says the biggest impact of the Core has been working with the individual Demonstration Projects to provide a sounding board to discuss statistical challenges. Further, Core members have contributed to new knowledge through manuscripts that address key methodological issues related to pragmatic clinical trials. She’s hoping the Core will continue to push the boundaries of statistical methods in the coming years.

“The statisticians on the individual trials have not only developed excellent statistical methods for their own studies, but also contributed substantively to the Core.” Dr. Liz DeLong

Download the interview (PDF).

October 16, 2017: NIH Collaboratory Core Working Group Interviews: Reflections from the Stakeholder Engagement Core

At the NIH Collaboratory Steering Committee meeting in May 2017, we asked Ellen Tambor, a member of the Stakeholder Engagement Core, to reflect on the first 5 years of the Core’s work and the challenges ahead. She says it’s key for stakeholder engagement to take place throughout the entire healthcare system, from leadership to the frontline providers and staff. And, because of the nature of pragmatic trials conducted in clinical settings, engagement is essential from the early stages through trial completion. Tambor also suggests asking two questions to ensure the right people are involved throughout pragmatic research: Who is going to use the evidence that results from the study? Who will help ensure that the study is implemented as seamlessly as possible?

“Pragmatic trials take stakeholder engagement to a new level of importance in terms of both the scope of engagement and the array of potential stakeholders.” Ellen Tambor

Download the interview (PDF).