August 14, 2018: Interview With NIH Collaboratory Leadership Highlights New Demonstration Projects

In a recent video interview, NIH Collaboratory leaders Dr. Lesley Curtis, Dr. Adrian Hernandez, and Dr. Catherine Meyers share their enthusiasm for the Collaboratory’s 6 new Demonstration Projects. The projects include new areas of expertise, such as pediatrics, new digital technologies, and the Collaboratory’s first A vs B trial.

“The new demonstration projects coming into the Collaboratory provide the opportunity to generate more knowledge and new knowledge about what works best in these settings. That’s really exciting.”—Lesley Curtis, PhD

July 30, 2018: Learn More About the New NIH Collaboratory Demonstration Projects

Check out the new program materials from the Collaboratory:

Study information for the 6 new UG3 Demonstration Projects:

  • ACP PEACE: Improving Advance Care Planning in Oncology: A Pragmatic, Cluster-Randomized Trial Integrating Patient Videos and Clinician Communication Training
  • EMBED: Pragmatic Trial of User-Centered Clinical Decision Support to Implement Emergency Department-Initiated Buprenorphine for Opioid Use Disorder
  • GGC4H: Pragmatic Trial of Parent-Focused Prevention in Pediatric Primary Care: Implementation and Adolescent Health Outcomes in Three Health Systems (Guiding Good Choices for Health)
  • Nudge: Personalized Patient Data and Behavioral Nudges to Improve Adherence to Chronic Cardiovascular Medications
  • PRIM-ER: Primary Palliative Care for Emergency Medicine
  • HiLo: Pragmatic Trial of Higher vs. Lower Serum Phosphate Targets in Patients Undergoing Hemodialysis

In addition, the May 2018 Steering Committee Meeting materials are now available, including presentations from the UG3 and UH3 Demonstration Projects and the full-day intensive workshop “Embedded Pragmatic Clinical Trials of Therapeutic A vs. B Interventions.”

July 16, 2018: JAMA Editorial Highlights mSToPS Trial, Discusses Strengths and Challenges of Pragmatic Clinical Trials

recent editorial in JAMA highlights the mHealth Screening to Prevent Strokes (mSToPS) randomized clinical trial in a discussion of the strengths and challenges of pragmatic clinical trials. The mSToPS trial, which studied patients at increased risk of atrial fibrillation who used a self-applied electrocardiogram sensor patch, found that immediate monitoring, compared with delayed monitoring, led to a higher rate of diagnosis after 4 months.

The editorial’s authors, Dr. Eric Peterson of Duke University and Dr. Robert Harrington of Stanford University, describe the mSToPS trial as an “innovative example of the potential (and challenges) inherent in a pragmatic IT trial.” The trial “scores quite high for its pragmatic approach” while offering lessons in the trade-offs that such an approach often requires.

The editorial notes that efforts like the NIH Collaboratory are bringing about innovations in health systems collaborations and clinical data platforms that “could provide the ideal setting to conduct highly pragmatic and efficient [randomized clinical trials] of the benefits, risks, and costs associated with novel health IT solutions.”

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)

March 7, 2018: FDA Offers Workshop on Submitting Draft Guidance on Patient Experience Data

The FDA is conducting a public workshop on Monday, March 19, to obtain input from stakeholders—including patients, patient advocates, academic and medical researchers, expert practitioners, drug developers, and other interested persons—to inform the drafting of a patient-focused drug development guidance as required by the 21st Century Cures Act. Workshop attendees will discuss considerations for development and submission of a proposed draft guidance regarding patient experience data submitted by an external stakeholder. The guidance is intended to help stakeholders continue progress in developing new medicines to respond to patient’s needs.

Registration for the event, either in person or via a live webcast, ends March 12. More meeting details, including background materials, will be posted by FDA as available.

January 17, 2018: The “All of Us” Research Program Asks for Research Ideas

The groundbreaking “All of Us” research program, which aims to enroll and track more than a million people, is asking prospective researchers, community organizations, and citizen scientists for suggestions regarding potential research questions. Ideas can be submitted through a special research page and are due by February 23, 2018. At a Research Priorities Workshop in March 2018, meeting attendees will use the input to set research priorities that will drive the development of the All of Us research platform and associated tools.

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).

October 2, 2017: New multi-agency initiative modeled on NIH Collaboratory to focus on non-drug approaches to managing chronic pain in U.S. service members and veterans.

A new research project modeled on the NIH Health Care Systems Research Collaboratory has been created to investigate non-drug approaches to helping U.S. service members and veterans manage chronic pain. The U.S. Department of Health and Human Services, the U.S. Department of Defense (DoD), and the U.S. Department of Veterans Affairs (VA) jointly announced the NIH-DoD-VA Pain Management Collaboratory project this week. The project, which includes funding for 12 demonstration projects over the next 6 years, will focus on large-scale, cost-effective, pragmatic trials conducted in military and VA healthcare systems.

The Pain Management Collaboratory Coordinating Center (PMC3) at Yale University will establish work groups to assist demonstration projects and provide support similar to that offered by NIH Collaboratory Core groups. A team from Duke Clinical Research Institute is leading one of the demonstration projects, a trial designed to improve access to recommended non-drug therapies for low back pain in the VA Health Care System.

Read more:

NIH & FDA seek feedback on new clinical trial protocol template


As part of their ongoing effort to improve the speed and efficiency of conducting clinical trials, the NIH-FDA Joint Leadership Council has created a draft clinical trial protocol template. The template contains instructional and sample text intended to assist NIH-funded investigators in writing protocols for phase 2 or 3 clinical trials that require Investigational New Drug (IND) or Investigational Device Exemption (IDE) applications. Feedback is sought from investigators, investigator-sponsors, institutional review board members, and other stakeholders involved in protocol development and review.

Our goal is to provide an organized way for creative investigators to describe their plans so that others can understand them. – Dr. Pamela McInnes, NIH

Details on the rationale and development of the protocol template are on these blog posts:

Notice Number: NOT-OD-16-043. Responses accepted through April 17, 2016.

You can access the template document as well as a template shell, comment form, and other resources at NIH’s Clinical Research Policy website.