The Patient-Centered Outcomes Core has developed a new tool kit to provide resources to support the capture of patient-reported outcome (PRO) measures in diverse study populations. The tool kit is intended for research teams conducting pragmatic clinical trials, including those participating in the NIH Pragmatic Trials NIH Collaboratory Trials.
Brief background about how PROs can provide critical information about the patient experience, but they must be culturally and linguistically appropriate to be valid.
A checklist focused on health equity considerations and PROs, which was adapted from the Center for Nursing Research at Duke University School of Nursing (DUSON) INFusE (Implementing a New Focus on Equity in Research) Checklist in order to focus on PRO measures and health equity.
An additional resources document that provides links to virtual libraries containing a range of existing translated and/or culturally adapted PRO measures, links to relevant peer-reviewed literature focused on PRO collection in pragmatic trials, and other tool kits to support equity in the collection of patient-centered outcomes and the conduct of pragmatic research.
The Grand Rounds session will be held on Friday, September 22, 2023, at 1:00 pm eastern.
Coker is the chief of general pediatrics and a professor of pediatrics at the University of Washington School of Medicine and Seattle Children’s Hospital. She is the former and founding research director for the Health Equity Research Program at Seattle Children Hospital’s Center for Diversity and Health Equity, and she currently serves as the codirector of the University of Washington’s NIH-funded Child Health Equity Research Fellowship.
From left: Dr. Stephanie Ibemere and Dr. Kaitlyn McLeod
The NIH Pragmatic Trials Collaboratory is welcoming its first class of fellows in a new program for early-career investigators with a scholarly interest in pragmatic clinical trials.
"We are very excited to welcome the 2 inaugural fellows to the NIH Pragmatic Trials Collaboratory Fellowship Program and give them the education and tools that they need to launch their careers as pragmatic clinical trials investigators," said Dr. Rosa Gonzalez-Guarda, cochair of the NIH Collaboratory’s Fellowship Program and Health Equity Core.
Dr. Stephanie Ibemere is an assistant professor in the Duke University School of Nursing. Dr. Ibemere will work closely with the Implementation Science Core and the GRACE NIH Collaboratory Trial team during her fellowship year.
Dr. Kaitlyn McLeod is a resident physician in the Internal Medicine Residency Program at the University of Colorado. Dr. McLeod will work closely with the Community Health Improvement Core and the Nudge NIH Collaboratory Trial team during her fellowship year.
"We are delighted that Stephanie and Kaitlyn are joining the NIH Collaboratory as fellows, and we look forward to the insights they will bring to the Cores and NIH Collaboratory Trials," said Dr. Kevin Weinfurt, co–principal investigator of the Coordinating Center and cochair of the new Fellowship Program.
During the 1-year fellowship, which began July 1, 2023, fellows will become members of the NIH Collaboratory program and have access to a wide range of activities and opportunities. Fellows will be active participants in a Core Working Group that aligns with their interests and will also be given education on the fundamentals of designing and conducting successful pragmatic clinical trials.
Working with a NIH Collaboratory Trial investigator mentor and a Core leadership mentor from the NIH Collaboratory, each fellow will complete a project that contributes to the field of pragmatic trials, resulting in a new publishable product or resource. In addition, fellows will participate in the following activities:
Regular presentations and discussions with experienced pragmatic trial investigators and leaders in the field
Individualized coursework and mentorship
Guided research program
Professional development training
Presentation and publication opportunities
Training on health equity as it relates to pragmatic trials
Ibemere and McLeod joined leaders from the NIH Collaboratory and their mentors for a welcome and orientation meeting on July 6 to kick off their fellowship year.
Julie Fritz, PT, PhD, FAPTA Distinguished Professor, Department of Physical Therapy & Athletic Training
Associate Dean for Research, College of Health
University of Utah
Guilherme Del Fiol, MD, PhD Professor, Biomedical Informatics
University of Utah
Keywords
Community Health Centers; Low Back Pain; Physical Therapy Modalities; Primary Care; Telemedicine
Key Points
Clinical practice guidelines support nonpharmacologic care as first-line management of low back pain. However, persons in low-income and rural communities have significantly higher odds of receiving a prescription opioid for a new back pain diagnosis.
Use of nonpharmacologic pain treatments is lower in rural settings and for persons of Hispanic/Latino ethnicity. Many of these communities are served by federally qualified health centers that often lack options to provide accessible nonpharmacologic alternatives.
Clinical research can exacerbate disparities, because clinical trials typically are based in urban, academic medical centers, underrepresent diverse populations, and overlook community engagement strategies in trial planning and design.
BeatPain Utah, an NIH Pragmatic Trials Collaboratory Trial, is an embedded pragmatic clinical trial comparing the effectiveness of nonpharmacologic intervention strategies for patients with back pain seeking care in federally qualified health centers in Utah. The interventions include a telehealth strategy that provides a brief pain teleconsult along with phone-based physical therapy, and an adaptive strategy that provides the brief pain teleconsult first, followed by phone-based physical therapy among patients who are nonresponsive to treatment.
BeatPain Utah is using the Community-Engaged Dissemination and Implementation (CEDI) framework, which considers both social and technical factors in the implementation of health IT strategies, decentralizes the research methods and procedures, and grounds the implementation in a systematic, iterative mapping of how both clinic staff and patients interact with health IT.
Although there is evidence of a significant divide in the implementation of advanced health IT functions, low-resource settings can adopt advanced health IT with some assistance. Moreover, there is considerable opportunity to reduce inequities through increased adoption of telehealth strategies, given that 96% of people in low-resource communities have at least a text and voice phone.
Discussion Themes
Motivating patients to engage in self-management of health conditions is a challenge, irrespective of whether the intervention is delivered in person or remotely.
Designing interventions that can meet everyone’s needs can be a challenge in a study that involves rural/urban and racial/ethnic diversity. Implementation mapping at the beginning of the design process is key. This includes direct assessment of patients’ needs, such as by interviewing patients who seek care in the partnering clinics to understand their expectations and how they would think about a mode of care delivery, like telehealth, that is unfamiliar to them.
Another crucial element of the health equity–focused model is ensuring that the question at the heart of the research is of value to the clinics and their leadership. For every trial, one of the first important tasks is to reach out to the community health center leadership and see if they are interested. The clinic’s priorities should drive the design of the trial. “We have to be very accommodating to the needs of each [community health center] and respect their needs.”
How quickly can these types of studies proceed from trial completion to release of results? Especially in studies involving chronic conditions, having long-term follow-up data to answer the core effectiveness question in a hybrid trial means there could be a long wait for results. Researchers must also be prepared to consider the question of sustaining a service that many clinics in the study have come to rely on, even before the results are available.
Pragmatic research is vulnerable to differences in data capture and access to care for different subsets of the population, which, if left unaddressed, can worsen health gaps.
In a new article published online ahead of print in JAMIA, the NIH Pragmatic Trials Collaboratory's Trial teams reflect on the challenges encountered by their trials and share the specific strategies they used to increase the generalizability of research results.
“Poor generalizability can occur because detailed information about specific populations is missing, and critically, is missing not at random,” the authors cautioned.
The NIH Collaboratory Trials are implementing approaches designed to ensure inclusion and retention of all populations, and to enable the complete collection of data.
“By improving data capture, access to care, and patient technology support, ePCTs hold the potential to yield insights and estimates pertinent to the entire population, not just a subset of the population,” they wrote.
In an interview during the annual Steering Committee meeting, Drs. Rosa Gonzalez-Guarda and Cherise Harrington, cochairs of the Health Equity Core Working Group, shared insights and next steps for health equity in the NIH Pragmatic Trials Collaboratory.
One question discussed at the meeting was how to embed health equity in pragmatic clinical trials. Gonzalez-Guarda said that a key component of addressing health equity is acknowledging the structural and social drivers of that inequity and developing strategies to target them.
“The next phase is to figure out ways that we can encourage embedded pragmatic clinical trials that address the structural causes of health inequities,” said Gonzalez-Guarda, who noted that the National Institutes of Health is supporting more structural interventions.
One way the NIH Pragmatic Trials Collaboratory can lead is by integrating a pragmatic clinical trial lens into structural intervention work, Gonzalez-Guarda said. This work is central to the Health Equity Core, which was launched last fall and began meeting regularly in early 2023.
Health Equity Core members are reviewing a checklist on how to infuse a health equity lens into the research life cycle. As a next step, Harrington and Gonzalez-Guarda would like to ask Core members to think critically about how the checklist can be adapted for pragmatic clinical trials and develop it as a new tool for the research community.
Harrington said another way the Health Equity Core can make an impact is by gathering examples from the NIH Collaboratory Trials of data on underrepresented populations that are not statistically significant but may be clinically significant so that other projects can learn from them.
Drawing from and adding to the NIH Pragmatic Trials Collaboratory’s lessons learned is also a key strategy for the Health Equity Core’s work. Harrington said it is important to share knowledge outside of the research community.
“We need to remember to ask ourselves, 'What am I leaving with this community after the funding is gone that they can still leverage?,’” said Harrington.
Many of the NIH Collaboratory Trials are already implementing health equity plans and engagement strategies to reach diverse patient populations, providers, and community stakeholders.
Gonzalez-Guarda said that it is important to continue this work and to support the careers of junior researchers working on pragmatic clinical trials so they can use engagement practices and be successful. In addition to preparing investigators to engage with diverse communities, part of the engagement work may also be training for communities to better engage with projects in a more meaningful way, Harrington said.
Developing tools and resources is a top priority as the Health Equity Core moves forward, and Gonzalez-Guarda and Harrington look forward to providing guidance for pragmatic clinical trials and continuing the conversation from the Steering Committee meeting.
A focus of the 2023 NIH Pragmatic Trials Collaboratory Annual Steering Committee Meeting was health equity in embedded pragmatic clinical trials (ePCTs). In an interview during the meeting, Drs. Anne Trontell and Ana Quiñones reflected on health equity in the ePCT landscape.
Trontell, who is the associate director for comparative clinical effectiveness research at the Patient-Centered Outcomes Research Institute (PCORI), said that health equity is a key component of the patient-centered efforts underlying all of PCORI’s research.
“From its beginning, PCORI has dedicated research funding to address the unique needs of historically underserved populations. The achievement of health equity is one of PCORI’s 5 guiding National Priorities for Health,” said Trontell.
As a funder, PCORI directs funds to those who pursue health equity goals and targets specific requirements that are important to the development of equity-focused research, such as working with communities and emphasizing inclusivity and diversity in the institutions and participants in research.
“It is a multipronged effort, and one that we are very excited to participate in,” said Trontell.
Quiñones, who is the core lead for the NIA IMPACT Collaboratory Health Equity Team and an associate professor at Oregon Health & Science University, said she agrees that bringing health equity into ePCTs requires a team-based approach.
“We’ve learned that leaning on ‘health equity experts’ or relying on individuals to provide answers for big gaps in the research evidence base isn’t just unrealistic, it is probably not great practice either,” said Quiñones.
One thing the NIA IMPACT Collaboratory has done is ask everyone involved in the program to think about and contribute to the process. A resulting product of this requirement is a series of best-practice recommendations for integrating health equity into ePCTs for dementia that were created by asking experts from areas across the IMPACT Collaboratory to apply a health equity perspective to their area of expertise to collectively develop recommendations for some of the issues and hidden pitfalls.
“This doesn’t mean that you don’t need people who are deeply embedded and knowledgeable about the [health equity] work, and knowledgeable about the best scientific principles, asking questions, but it does mean that everybody is a participant in solving the problem, not just the health equity expert,” said Quiñones.
Trontell said that one way PCORI is supporting health equity is by funding the groundwork that can help researchers be successful, such as building relationships with diverse communities through its engagement awards. In addition, PCORI’s Phased Large Awards for Comparative Effectiveness Research (PLACER) opportunities fund a feasibility phase that supports study refinement, infrastructure establishment, stakeholder engagement, and feasibility testing of study operations, including the ability to recruit and enroll participants successfully.
“We have heard from our investigators that meaningful engagement deserves attention, effort, and time, so our engagement awards and PLACER funding opportunity offer upstream support for strong and diverse engagement to occur,” said Trontell.
Networks such as the NIA IMPACT Collaboratory and the NIH Pragmatic Trials Collaboratory have an important role to play in these efforts as well. Quiñones said these large research networks have opportunities to build synergies around common goals related to health equity and share knowledge and best practices.
Trontell agrees. “I’m a big fan of bringing people together to figure out something and to listen to each other, particularly when they have a shared goal and excitement around trading ideas and tips. That creates a real learning community.”
This year’s annual Steering Committee meeting for the NIH Pragmatic Trials Collaboratory featured health equity as a central topic of discussion. In an interview after his keynote presentation, Eliseo J. Pérez-Stable, MD, Director of the National Institute on Minority Health and Health Disparities (NIMHD), shared his thoughts on national priorities for reducing health disparities and opportunities for clinical researchers to help promote health equity.
Pérez-Stable outlined 5 key ways science can help reduce inequities:
Standardized measurement of social and demographic factors that affect health
Facilitate discovery science with big data
Be an engine for promoting diversity of the scientific and clinical workforce
Cultivate community engagement and build trust for sustainable relationships
Implement what we know can work to promote health equity
A major theme of his presentation was that a community-engaged approach has to be leveraged in order to get a representative research study. “People just don’t show up because you have a good study. You have to have some kind of community connection,” he said. He recommended sitting down with people as equals to co-develop research. “Don’t come to them with ‘here’s what I’ve got to offer you’—there has to be some evolution of goals during that process.”
Pérez-Stable described the extra effort needed for researchers to build sustainable bonds in communities. “You have to go to the community, go to the stores, be there, and participate, even if unrelated to your research or your studies,” he said. “The saying ‘move at the speed of trust’ is so essential.”
Furthermore, diverse clinical investigators at the principal investigator and co-investigator level really matter in terms of achieving inclusive participation in clinical studies. While there have been some improvements in diversifying the clinical workforce, there has not been enough progress, according to Pérez-Stable.
Similarly, although the research community has made strides in capturing reliable data on participant race and ethnicity, there is still much room for improvement, especially when it comes to electronic health record data in the clinical setting, which is particularly relevant for pragmatic trials. Systematic measurement of race and ethnicity are essential to understanding disparities and providing equitable care. Going forward, there is also tremendous value in capturing more granular data on individuals’ heritage and background.
Finally, Pérez-Stable cautioned that researchers must keep an eye on factors such as the digital divide so health disparities do not get exacerbated. Digital methods are easy for researchers to rely on, but we must remember there is less technology uptake in populations such as older adults, minorities, and the working class. “We can’t just jump to digital methods and expect everyone to join us,” he warned. For example, a significant number of households do not have a computer, and data usage for mobile devices costs money.
Pérez-Stable stated that he is impressed with the work the NIH Collaboratory is doing to bring health equity to the forefront in clinical research, including through the new Health Equity Core, and he hopes the research community will continue to think in terms of what we should be doing to reduce health disparities.
Stay tuned for more interviews with program leaders from the 2023 Steering Committee meeting.
In a secondary analysis from the EMBED pragmatic clinical trial, Black patients with opioid use disorder were less likely than White patients to be initiated on buprenorphine in the emergency department.
The study’s findings were published recently in Academic Emergency Medicine.
EMBED, an NIH Collaboratory Trial, was a cluster randomized trial across 21 emergency departments in 5 healthcare systems in the United States. The trial evaluated a clinical decision support system for initiating buprenorphine in emergency department settings.
The racial disparity in buprenorphine initiation remained after adjustment for patient, clinician, and site characteristics. Even in emergency departments in academic hospitals, where rates of buprenorphine were higher overall, Black patients received proportionally less buprenorphine initiations than White patients. Hispanic patients were more likely to receive buprenorphine than non-Hispanic patients in both community and academic emergency departments. However, adjustment for discharge diagnosis attenuated the association between ethnicity and buprenorphine initiation.
“Attention should be focused on identifying continued disparities in [emergency department] treatment of opioid use disorder by race and ethnicity,” the authors concluded, “as well as the barriers and inequities that continue to limit patients’ ability to access the [emergency department] for treatment of opioid use disorder.”
EMBED was supported within the NIH Collaboratory by a cooperative agreement from the National Institute on Drug Abuse and received logistical and technical support from the NIH Collaboratory Coordinating Center. Read more about EMBED in the Living Textbook, and learn about the other NIH Collaboratory Trials.
The NIH National Center for Advancing Translational Sciences (NCATS) recently announced a new Request for Information (RFI) entitled, “Advancing Clinical and Translational Science Through Accelerating the Decentralization of Clinical Trials.” The goals of the RFI are to identify ways that decentralized clinical trials can help make trials more effective, efficient, and equitable. In particular, comments are sought on ways to decrease health disparities by enabling participation of those who are underrepresented in clinical trials, including by race, ethnicity, socioeconomic status, background, gender, and mental or physical ability.
The potential areas for commentary include
Application of research methods in decentralized clinical trials (DCTs)
Resources, Infrastructure, and Enabling Technologies
Community Engagement
Workforce Development
Partnerships and/or Collaborations
Study Participation and Adherence
Data integration, quality, accessibility, and reproducibility
The Patient-Centered Outcomes Core has developed a new tool kit to provide resources to support the capture of patient-reported outcome (PRO) measures in diverse study populations. The tool kit is intended for research teams conducting pragmatic clinical trials, including those participating in the NIH Pragmatic Trials NIH Collaboratory Trials.
In this Friday’s PCT Grand Rounds, Tumaini Rucker Coker of the University of Washington and Seattle Children’s Hospital will present 
Pragmatic research is vulnerable to differences in data capture and access to care for different subsets of the population, which, if left unaddressed, can worsen health gaps.
In a secondary analysis from the EMBED pragmatic clinical trial, Black patients with opioid use disorder were less likely than White patients to be initiated on buprenorphine in the emergency department.