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
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)
In a new article, Dr. Vincent Mor, an NIH Collaboratory investigator, and Dr. Paul Wallace describe the history, current status, and opportunities for funding training in health services research (HSR). While the number of organizations seeking to solve problems with health services research has been expanding, direct government support for HSR is declining. The authors project 5 key challenges for the field and its professional development:
Formulating and prioritizing research topics
Whether to use team- or individual-based approaches
How new data sources, analytic methods, and the need for faster results affect supply and demand for HSR
Shifts from public to institutional funding and the associated effects on generalizability
Balancing proprietary concerns regarding data, predictive models, and study results with the need to improve public health and rapidly disseminate information
According to the authors, sustainable solution will involve active collaboration between those who use HSR as a part of decision-making (and will likely pay for it) and those who produce it.
“We believe that the key change needed to productively address the above challenges will be a closer collaboration between HSR users, especially health systems, and academic HSR training programs to work towards producing timely, internally relevant, and externally generalizable knowledge (Mor and Wallace 2018).”
NIH Collaboratory investigators Dr. Susan Mitchell and Dr. Vincent Mor coauthored an editorial for an upcoming issue of JAMDA highlighting the need for pragmatic trials of nonpharmacologic interventions in Alzheimer disease and related dementias. The editorial summarizes conclusions from a recent workshop at the National Institute on Aging (NIA), where experts discussed the state of the science for pragmatic trials to improve dementia care.
The goals of the NIA workshop were (1) to identify criteria for determining the readiness of dementia interventions for pragmatic trials, and (2) to describe the research infrastructure needed to support such trials.
Drs. Mitchell and Mor are co-principal investigators of the NIH Collaboratory’s Pragmatic Trial of Video Education in Nursing Homes (PROVEN), which is evaluating the effectiveness of advance care planning video tools in nursing home settings. The editorial mentions that a supportive infrastructure for pragmatic research in dementia could be modeled after the NIH Collaboratory’s Coordinating Center, including Core Working Groups “focused on building investigator capacity, supporting pragmatic trial design, and maintaining the resource and knowledge base.”
Full citation: Baier RR, Mitchell SL, Jutkowitz E, Mor V. Identifying and supporting nonpharmacological dementia interventions ready for pragmatic trials: results from an expert workshop. J Am Med Dir Assoc. doi: 10.1016/j.jamda.2018.02.011.
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.
Registration is now open for a one-day workshop sponsored by the NIH Common Fund and organized by NIH Collaboratory thought leaders. The workshop, to be held on May 16, will focus on expanding the understanding of a specific type of pragmatic trial, the A vs. B trial, which compares two or more distinct therapeutic medical interventions. Over the past 6 years, the NIH Collaboratory has launched Demonstration Projects that address questions of major public health importance in real-world settings with usual care as a control arm. Much has been learned about the design, conduct, and dissemination of pragmatic clinical trials through this work. As a next step, there is considerable excitement at the prospect of exploring pragmatic clinical trials for comparing medical interventions.
The workshop will include a series of moderated discussions of opportunities and approaches for partnering with healthcare systems, and other stakeholders, to conduct embedded A vs. B trials. Speakers will include representatives from NIH, FDA, OHRP, health systems, academia, and industry.
The workshop will take place from 8:00 AM – 4:15 PM ET on Wednesday, May 16. Participants can join in person at the Bethesda Marriott in Maryland or via webcast. Choose an option for attendance on the registration page.
The organizing committee for the workshop includes Adrian Hernandez & Richard Platt (Co-Chairs), Laura Dember, Susan Huang, Catherine Meyers, Wendy Weber, and Dave Wendler.
Seeking to advance the study and practice of engagement in health research, the Patient-Centered Outcomes Research Institute (PCORI) recently launched the Engagement in Health Research Literature Explorer. Locating relevant research articles about engagement can be challenging because of a lack of standard terminology. The new tool searches a curated database of peer-reviewed literature on engagement. Articles are included in the database if they describe engagement experiences, report research findings on engagement practices, or present theories, concepts, or views on engagement. The database is updated monthly and is one way PCORI is helping to promote meaningful involvement of patients, caregivers, clinicians, and other healthcare stakeholders throughout the research process.
Congratulations to Dr. Lynn DeBar and the investigators of the Collaborative Care for Chronic Pain in Primary Care pragmatic trial for recently publishing their study design paper. One of the NIH Collaboratory Demonstration Projects, the trial is designed to test whether a primary care–based behavioral intervention the Pain Program for Active Coping and Training (PPACT)—will provide a “more effective, safer, and more satisfactory alternative to opioid-based chronic pain treatment” than usual care for patients on chronic opioid treatment (Debar et al 2018). Learn more about this innovative trial in the article in Contemporary Clinical Trials. You can also download a trial snapshot.
Full Citation: DeBar L, Benes L, Bonifay A, et al. Interdisciplinary team-based care for patients with chronic pain on long-term opioid treatment in primary care (PPACT) – Protocol for a pragmatic cluster randomized trial. Contemporary Clinical Trials. 2018;67:91-99. doi:10.1016/j.cct.2018.02.015
A recently released summary from the ADAPTABLE Roundtable Meeting explores ways to better understand the sets of circumstances and considerations that could guide when and how to gather and integrate patient-reported health data with other data sources in pragmatic trials.
For outcomes that represent subjective experiences, such as pain, symptoms, and physical functioning, the patient is the unique and privileged source of information. Other patient-reported health data may not have a clear source of truth, such as co-morbidities and hospitalizations. In such cases, patient-reported health data may supplement, contradict, or agree with EHR and claims data. For example, medication data reported by patients might be a more accurate reflection of what patients are actually taking than medication data in the EHR, especially for over-the-counter medications.
Patient-reported health data come from various sources and can be feasibly collected in the conduct of a pragmatic clinical trial, but the optimal approaches for capturing and analyzing these data are unclear. Questions include how to integrate this information with other data collected as part of a study, including data from the EHR.
To better understand patient-reported health data and how to use them in pragmatic trials, 18 experts from 8 institutions convened at the roundtable meeting, coming from a wide variety of backgrounds including biostatistics, epidemiology, oncology, nursing, psychiatry, health policy, and regulation. Representatives from the NIH Collaboratory included Drs. Lesley Curtis and Rachel Richesson from the EHR Core and Dr. Kevin Weinfurt from the Patient-Reported Outcomes Core.
In addition to the meeting summary, two white papers are forthcoming. For more information about using patient-reported data in pragmatic trials, see the Living Textbook Chapter on Endpoints and Outcomes.
On February 20-21, 2018, a group of 27 clinical investigators met in Durham to learn from the NIH Collaboratory about the design and conduct of embedded pragmatic clinical trials (ePCTs). Through the experiences of the NIH Collaboratory, much has been learned about how to launch and implement successful ePCTs. The workshop’s goal was to help investigators advance their ePCT research ideas and build a larger community of researchers capable of conducting high-quality ePCTs. Participants from across the country were selected based on a rigorous application process.
“ePCTS hold the promise of an efficient and powerful way to generate evidence. We’ve learned so much through the Collaboratory, but the knowledge is not helpful if we’re not passing it along to other clinical investigators so they can contribute even more evidence to the knowledge base,” said Kevin Weinfurt, PhD, a co-principal investigator of the NIH Collaboratory’s Coordinating Center who helped develop and organize the workshop along with a planning committee.
Representatives from the Coordinating Center, Demonstration Projects, and Core Groups were on hand to provide information, guidance, and lessons learned from their experience with the NIH Collaboratory ePCTs. The topics included an introduction to ePCTs, engaging stakeholders and aligning with healthcare system partners, designing with implementation in mind, design and analytic considerations, regulatory and ethical challenges, measuring outcomes, dissemination, and ePCT team composition. Representatives from the NIH were also in attendance and presented sessions about the importance of pilot and feasibility testing and developing a compelling application for funding. During the workshop, participants completed hands-on exercises and worked through trial design challenges with the experts and their colleagues.
“We’re trying to change the world, and to change the world you need generalizable data and interventions that are scalable. The chasm for implementation of clinical evidence is huge, and PCTs have the capacity to take great interventions and get them to the patients so that they can have impact and improve care,” said Wendy Weber, ND, PhD, MPH, acting deputy director of the National Center for Complementary and Integrative Health (NCCIH), and one of the workshop presenters. “While local improvements can be accomplished through quality improvement activities, if you have outcomes that matter, randomizing interventions and studying them on a large scale can provide the evidence that will make a real difference in the care of people across the United States.”
Another goal of the workshop was to pilot the educational materials and collect feedback on their quality and appropriateness from both attendees and subject matter experts. The Collaboratory Coordinating Center will use the feedback to refine the content for future workshops and educational materials. The NIH Collaboratory’s Living Textbook provided a foundation for much of the workshop, and a host of new information was presented that can be used in future materials and updates to the textbook.
The training was funded as an NIH Roadmap Initiative 3U54AT007748-05S2.