The Patient-Centered Outcomes Research Institute (PCORI) announced the release of its new Foundational Expectations for Partnerships in Research. The resource is an update of the 2014 PCORI Engagement Rubric. It builds on a growing body of evidence about engaging patients and other partners in patient-centered comparative effectiveness research.
A webinar on April 4 will explore the foundational expectations, which PCORI describes as “building blocks for meaningful, effective, and sustainable engagement with patients, communities, and other partners in research.”
The 6 foundational expectations are:
diversity and representation
early and ongoing engagement
dedicated funds for engagement and partner compensation
build capacity to work as a team
meaningful inclusion of partners in decision-making
ongoing review and assessment of engagement
The April 4 webinar will describe the foundational expectations and explore how PCORI will incorporate them into future funding opportunities. This event is intended for current and future applicants for PCORI funding and anyone interested in participating in the healthcare research process. Register for the webinar.
PCORI is an independent, nonprofit organization authorized by Congress and is the nation’s leading funder of patient-centered comparative clinical effectiveness research.
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.”
Dr. Fan Li, a member of the NIH Pragmatic Trials Collaboratory’s Biostatistics and Study Design Core since 2013, has received approval of a 3-year funding award from the Patient-Centered Outcomes Research Institute (PCORI) to develop causal inference methods for stepped-wedge cluster randomized trials—a design that has been increasingly adopted in pragmatic trials. Li is an assistant professor of biostatistics at the Yale School of Public Health.
An estimand is a precise description of the treatment effect reflecting the scientific question, and is ideally a model-free concept. The research team will contribute weighted average effect estimands to quantify treatment effect evidence by recognizing that unequal cluster sizes may contribute to variations of treatment effects in each cluster-period. In addition, pragmatic trials that adopt a stepped-wedge cluster randomized design frequently collect baseline data on the patient-centered outcomes and/or patient-level characteristics. The research team will study and operationalize estimand-aligned methods that effectively leverage such baseline variables through parametric regression and nonparametric machine learning methods.
Li has assembled a multidisciplinary team for this study, including Dr. Patrick Heagerty, professor of biostatistics at the University of Washington and a cochair of the NIH Collaboratory’s Biostatistics and Study Design Core. In addition, Drs. Jeffrey Jarvik, principal investigator of the NIH Collaboratory’s LIRE NIH Collaboratory Trial, and Douglas Zatzick, principal investigator of the TSOS NIH Collaboratory Trial, serve as stakeholders of the study. The stakeholder team also includes colleagues from the NIA IMPACT Collaboratory, Drs. Thomas Travison and Monica Taljaard.
Holve is the chief research infrastructure officer for PCORI. Rothman is the principal investigator of the PCORI-funded Mid-South Clinical Data Research Network and a member of the PCORnet Executive Committee. Jones is an associate professor of medicine and population health sciences at Duke University. Pagidipati is an associate professor of medicine at Duke University.
Emily O’Brien, PhD, FAHA
Associate Professor
Duke Clinical Research Institute
Duke University School of Medicine
Department of Population Health Sciences
Russell Rothman, MD, MPP
Senior Vice President, Population and Public Health
Director, Vanderbilt Institute for Medicine and Public Health
Vanderbilt University Medical Center
On March 21, 2020, in response to the COVID-19 pandemic, PCORI contacted leadership at Duke Clinical Research Institute and PCORnet and a decision was made to focus on the space of healthcare workers. The HERO Program was fully approved and began recruiting participants on April 22, 2020.
The HERO Registry aimed to create a diverse virtual community of healthcare workers and their families and communities, ready for future COVID-19 research.
The HERO Registry explored topics that mattered most to participants and found these topics changed with time. Important issues early on included COVID-19’s effects on the workplace, vaccine access and willingness, and impact on home life. Later, burnout and lack of appreciation and support became larger issues.
The first trial undertaken by the HERO Program, HERO-HCQ, evaluated the efficacy of hydroxychloroquine (HCQ) to prevent COVID-19 in healthcare workers. Over 1300 participants were recruited from the HERO Registry. No statistically significant benefit was found.
The HERO TOGETHER study leveraged the HERO Registry to estimate real-world incidence of safety events among vaccinated individuals. The most common safety events reported included non-hospitalized arthritis/arthralgia and non-hospitalized non-anaphylactic allergic reaction.
– The HERO Registry survey collected a large broad range of information. Participant feedback revealed that shorter more targeted surveys focusing on the most high-value information may have been less burdensome for participants..
– The creative multi-faceted approach to recruitment that includes diverse stakeholder engagement could be successful in creating research registries for other important health issues. .
James C Grotta, MD
Director Stroke Research and Mobile Stroke Unit
Memorial Hermann Hospital-Texas Medical Center
Houston, Texas
Topic
Benefits of Stroke Treatment Delivered Using a Mobile Stroke Unit Compared to Standard Management by Emergency Medical Services: The BEST-MSU Study
Keywords
Stroke; BEST-MSU study; Mobile Stroke Unit; Patient-centered outcome; tPA
Key Points
We can improve stroke outcomes by giving tPA in the first hour after stroke onset, but to do this, we have to bring the treatment to the patient.
The Mobile Stroke Unit (MSU) is an ambulance equipped with a CT scanner and other medical care equipment necessary to treat stroke.
The BEST-MSU Study was a partially randomized trial that examined outcomes of patients who were treated by the MSU versus in the hospital emergency department.
The BEST-MSU study compared the diagnosis of telemedicine and onboard neurologists, and they agreed that the patient was having a stroke 88% of the time, which is the same as the agreement rate of two neurologists in the hospital setting.
For every 100 patients treated with the MSU rather than the standard medical practice in a hospital emergency setting, 27 will have less final disability and 11 more will be disability-free.
Discussion Themes
Consent to deliver treatment wasn’t necessary in this case because the treatment delivered was the standard of care. Consent to use the patient’s data was obtained in the emergency department after the patient had stabilized. Very few patients refused to consent.
Implementation of the MSU with the local fire departments was straight forward, but the politics of the area served was a challenge for implementation.
One barrier to treating stroke quickly with the MSU is the patient’s hesitancy to call 911. Calling emergency services needs to be made less threatening and less expensive to promote better patient outcomes.
ADAPTABLE is a pragmatic clinical study of 15,000 patients to examine a simple, everyday decision, whether to take 81mg or 325mg of aspirin daily, to identify if that decision could prevent heart attacks.
ADAPTABLE used both pragmatic and personalized approaches to participant recruitment.
Pragmatic recruitment involves broad-based email and social media outreach to a large pool of potential subjects. This method of recruitment was lower cost, less time intensive, and faster paced than personalized recruitment.
Personalized recruitment involves traditional in-clinic or phone call personal outreach to potential participants. This method of recruitment was higher cost, time intensive, and slower paced than personalized recruitment.
ADAPTABLE learned a critical lesson in patient engagement: in-clinic enrollment was much higher at 81% of those approached versus e-communication enrollment at 35% of those contacted.
Key principles of patient engagement include trust and partnership, respect and listening, empowering patients to find solutions, and value and design the trial for the patient experience.
Discussion Themes
We are moving from a traditional model of research to a relational model with participants coming together with researchers and forming a kind of social contract where a participant’s voice is valued in the research process.
We have lessons left to learn in order to fully utilize technology for patient engagement including how to use broad outreach methods to increase enrollment of diverse populations.
Patients continue to look to their clinician or doctor for advice on whether to participate in a clinical trial. Trusted contacts will continue to play an important role in the recruitment process.
Dr. Fan Li, a longtime member of the NIH Collaboratory’s Biostatistics and Study Design Core, has received approval for a $1 million grant award from the Patient-Centered Outcomes Research Institute (PCORI) to develop methods and software for designing cluster randomized trials. Li is an assistant professor of biostatistics in the Yale School of Public Health.
HTE occurs when there is systematic variation in treatment effect across predefined patient or provider subgroups that can arise due to diverse practices, varying responses to treatment, or differing vulnerability to certain diseases, among other reasons. While understanding of HTE has been a recognized goal in individually randomized trials, methods for planning cluster randomized trials with HTE analyses are limited. This PCORI-funded study will expand the current cluster randomized design toolbox to accommodate confirmatory HTE analysis and meet a growing interest in better understanding how patient- and provider-level characteristics moderate the impact of new care innovations in pragmatic trials.
The award has been approved pending completion of a business and programmatic review by PCORI staff and issuance of a formal award contract.
Joining Li on the research team are coinvestigators Dr. Patrick Heagerty of the University of Washington, Dr. Rui Wang of Harvard Medical School and the Harvard Pilgrim Health Care Institute, and Dr. Denise Esserman of the Yale School of Public Health. Heagerty and Wang are members of the NIH Collaboratory’s Biostatistics and Study Design Core. The team will work closely with other NIH Collaboratory colleagues and stakeholders, including Dr. Adrian Hernandez of Duke University, Dr. Jerry Jarvik of the University of Washington, and Dr. Richard Platt of Harvard Medical School and the Harvard Pilgrim Health Care Institute.
Panel: Amanda Midboe, PhD Center for Innovation to Implementation U.S. Department of Veterans Affairs Chair of the Implementation Science Work Group for the NIH-DOD-VA Pain Management Collaboratory
Anne Trontell, MD, MPH Associate Director Clinical Effectiveness and Decision Science Program Patient-Centered Outcomes Research Institute (PCORI)
Guest Moderator: David Chambers, DPhil Deputy Director for Implementation Science Office of the Director in the Division of Cancer Control and Population Sciences (DCCPS) National Cancer Institute (NCI) National Institutes of Health (NIH)
Topic
Inclusion of Diverse Participants in Pragmatic Clinical Trials: Maximizing Diversity in PCTs – What Can We Learn From Implementation Trials?
The study of methods to best implement and disseminate interventions in diverse groups is crucial to the ultimate goal of benefiting population health.
Patient-centered outcomes are important measures to determine the total effectiveness of any health intervention.
By engaging the community in which research takes place and treating patients as partners in the development of implementation strategies, we increase diverse participation and improve patient retention.
Targeted recruitment strategies that leverage administrative data increase diverse study participation.
Outreach materials and methods that are tailored and adapted to the local culture and colloquial language are effective in engaging diverse populations.
Discussion Themes
Are there any ethical issues raised when underserved populations receive monetary incentives to participate in research trials?
What are the challenges of identifying diverse potential participants from the medical record or administrative data?
Do efforts to increase diversity in the study team improve the engagement and retention of a diverse study population?
The NIH Collaboratory is using its popular ePCT Grand Rounds platform for a special webinar series on diversity in pragmatic clinical trials.
In this Friday’s session—“Maximizing Diversity in PCTs – What Can We Learn From Implementation Trials?”—Dr. David Chambers of the National Cancer Institute (NCI) will facilitate a discussion about lessons from implementation science for maximizing participant diversity in pragmatic clinical trials. Dr. Chambers is deputy director for implementation science in the Office of the Director in the NCI’s Division of Cancer Control and Population Sciences.
The panelists will include:
Dr. Amanda Midboe of the US Department of Veterans Affairs, chair of the Implementation Science Work Group for the NIH-DOD-VA Pain Management Collaboratory
Dr. Anne Trontell, associate director in the Clinical Effectiveness and Decision Science Program at the Patient-Centered Outcomes Research Institute (PCORI)
The Grand Rounds session will be held on Friday, July 16, at 1:00 pm eastern. Join the online meeting.
Other upcoming sessions in the diversity workshop series include:
The Patient-Centered Outcomes Research Institute (PCORI) announced the release of its new Foundational Expectations for Partnerships in Research. The resource is an update of the 2014 PCORI Engagement Rubric. It builds on a growing body of evidence about engaging patients and other partners in patient-centered comparative effectiveness research.
In this Friday’s PCT Grand Rounds, Erin Holve, Russell Rothman, Schuyler Jones, and Neha Pagidipati will present 