Because many clinicians do not have the skill set to engage patients in conversations about advance care planning (ACP), many older Americans with advanced cancer receive aggressive interventions at the end of life that do not reflect their values, goals, and preferences. The ACP PEACE trial is investigating whether a comprehensive approach to ACP improves patient outcomes. The program will combine two evidence-based complementary interventions: clinician communication skills training (VitalTalk) and patient video decision aids (ACP Decisions). The goal is to provide both patients and clinicians with communication skills and tools so they can make informed decisions about end-of-life care.
“We’re doing an intervention where we include videos for patients about what the possible interventions are, and we’re also training the clinicians, the oncologists and their teams to have better communication about their goals of care and about treatment planning.” —James Tulsky, MD, Co-Principal Investigator of the ACP PEACE trial.
ACP-PEACE is one of the new NIH Collaboratory Trials and is led by Drs. James A. Tulsky and Angelo Volandes with support from the National Institute on Aging. Read more about ACP PEACE.
Before the end of high school, more than half of all adolescents will use an illicit drug, about a quarter will meet the criteria for depression, and many others will engage in behaviors such as delinquency and violence. Guiding Good Choices is a universal evidence-based anticipatory guidance curriculum for parents of early adolescents that has been shown to reduce adolescent alcohol, tobacco, and marijuana use; depression; and delinquent behavior. Evidence-based parenting interventions shown to prevent these behavioral health concerns could improve adolescent health trajectories if implemented widely in pediatric primary care.
Guiding Good Choices for Health (GCC4H): Testing Feasibility and Effectiveness of Universal Parent-Focused Prevention in Three Healthcare Systems is a cluster-randomized trial that will partner with pediatric primary care clinics to offer the training in three large, integrated healthcare systems serving socioeconomically diverse families.
“We already have an effective intervention, so we’re not trying to test whether it works or not; it’s really about getting the intervention into a population.”—Richard Catalano, PhD, co-Principal Investigator of the GGC4H trial.
GGC4H is led by Richard Catalano, PhD, Margaret Kuklinski, PhD, and Stacy Sterling, DrPH, with support from the National Center for Complementary and Integrative Health. Read more about GGC4H.
The ADAPTABLE pragmatic trial relies on patients to report key information at baseline and throughout follow-up. To capture these data, ADAPTABLE investigators developed a LOINC (Logical Observation Identifiers Names and Codes) patient-reported item set, which is now publicly available.
The development of the item set is part of the ADAPTABLE Supplement, an initiative funded by the Office of the Assistant Secretary for Planning and Evaluation to develop best practices for capturing patient-reported outcome data and optimal analytic approaches for using the data in a pragmatic clinical trial. Additional reference material can be found in the ADAPTABLE Supplement Roundtable Meeting summary, in a report describing the results of a literature review of data standards and metadata standards for variables of interest, and on GitHub. The project is expected to inform future efforts to integrate patient-reported data in the electronic health record and provide opportunities to streamline data for use in pragmatic trials. Information from the project is being added to the Living Textbook as it accumulates; learn more in the chapters on Using Electronic Health Record Data and Choosing and Specifying End Points and Outcomes.
ADAPTABLE (Aspirin Dosing: A Patient-Centric Trial Assessing Benefits and Long-Term Effectiveness) aims to identify the optimal dose of aspirin therapy for secondary prevention in atherosclerotic cardiovascular disease and is the first major randomized comparative effectiveness trial to be conducted by the National Patient-Centered Clinical Research Network (PCORnet).
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).”
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 NIH Collaboratory Trials 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.
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 Trials, 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, NIH Collaboratory Trials, 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.
Wendy Weber, ND, PhD, MPH, Acting Deputy Director of the National Center for Complementary and Integrative Health (NCCIH), presenting on pilot and feasibility testing in ePCTs.
“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.
The NIH Collaboratory will soon welcome a new group of NIH Collaboratory Trials 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.
We recently asked Dr. Kevin Weinfurt, Chair of the Patient-Reported Outcomes (PRO) Core, to reflect on the first 5 years of the Core’s work and the challenges ahead. He says the biggest impact of the Core has been working with national initiatives to improve inclusion of PROs in the electronic health record (EHR). Further, Core members have contributed to new knowledge through white papers and chapters in the Living Textbook. In the coming years, he’s hoping the Core will be able to identify the value proposition of PROs.
“Because there are costs associated with collecting PROs, we need to determine when PROS are essential, supporting, or not at all informative for the clinical questions. This gets at the value proposition: When are they of value and to whom?” —Dr. Kevin Weinfurt.
One of the 
