The NIH Pragmatic Trials Collaboratory published a new chapter in its Living Textbook of Pragmatic Clinical Trials. The chapter, Patient Engagement, describes principles and strategies for effectively engaging patient partners.
Because patients can provide valuable insights and perspectives about clinical care for specific conditions, they are key partners for pragmatic clinical trials. The chapter highlights the value of patient engagement and provides practical tactics for patient engagement throughout the life cycle of a pragmatic trial. It also recognizes potential barriers to patient engagement and case studies in the pragmatic trials context.
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.
A new article from the NIH Pragmatic Trials Collaboratory outlines stakeholder insights and ethical considerations related to sharing deidentified, participant-level data in pragmatic clinical trials. While there are numerous arguments for and against data sharing in the context of pragmatic trials, the report aims to address the gap in documented stakeholder perspectives.
The authors, including members of the NIH Collaboratory’s Ethics and Regulatory Core, recruited stakeholders who represented a range of experiences. They then conducted 40 semistructured interviews focused on ethical considerations. The team identified 5 overarching themes after analyzing the interviews: (1) challenges in sharing data collected under a waiver or alteration of consent; (2) conflicting views regarding patient-subject preferences for data sharing; (3) identification of respect-promoting practices beyond consent; (4) concerns about elevated risks or burdens from sharing data; and (5) diverse views about the likely benefits resulting from sharing data.
A key insight was that a “one size fits all” model for promoting broader sharing of data from pragmatic trials is not feasible, and policies must be sensitive to the unique challenges that pragmatic trials present, such as variation among trials.
“Our data indicate unresolved tensions in how to fulfill this expectation for [pragmatic clinical trials],” the authors wrote. “Future work could inform efforts to tailor data-sharing policy and practice to reflect these and other challenges, including sharing experiences from trials that have successfully navigated these tensions.”
Lead author Stephanie Morain and co-authors Juli Bollinger, Kevin Weinfurt, and Jeremy Sugarman are members of the NIH Collaboratory’s Ethics and Regulatory Core. This work was supported within the NIH Pragmatic Trials Collaboratory by a supplemental grant award from the National Center for Complementary and Integrative Health.
An upcoming webinar in the NIH HEAL Initiative’s HEAL Headliners series will highlight the experiences of 2 projects in meaningfully engaging patients and community partners throughout the research process to ensure that the findings have the greatest public health impact. The webinar will be held on Friday, May 5, 2023, at 2:00 pm eastern.
Rosemarie Martin of Brown University and Linda Hurley of CODAC Behavioral Heathcare will report on a study of a systems-change approach for increasing use of medications for opioid use disorder across 7 probation and parole sites to improve linkage of justice-involved individuals to evidence-based treatment. In addition, Kirsten Johansen of Hennepin Healthcare will describe the role of patient and community engagement in the HOPE clinical trial of tailored patient-centered interventions to manage pain and reduce opioid use among patients receiving hemodialysis.
HEAL Headliners: Engaging Patients and Communities in HEAL Research Venue: Live online via Zoom Date and Time: May 5, 2023; 2:00 pm ET Register free online
In this webinar we will discuss equity, what it is and isn’t, the role of equity in medical decision making and gaps in the literature. There will be a keynote presentation defining equity in the context of medical decision making, followed by parallel break out rooms with moderated discussions on how equity relates to:
Shared decision making (SDM), including discussion of the role of health literacy and the importance of engaging patients in decisions in the way they can understand.
Patient Decision Aid (PtDA) development, including discussion of the importance of adapting PtDAs to ensure they meet the needs of end users.
Modelling including discussion of the importance of addressing equity in modelling to prevent bias in model-based medical decision making.
Equity in Medical Decision Making: Perspectives from Patient Partners and SMDM Members Venue: Live online via Zoom
March 2, 2023; 11:00 am-12:30 pm ET Register free online
Program
Welcome and Introductions – Jesse Jansen & Mary Politi
Keynote Presentation: “Definition of equity and how it impacts MDM” – Norah L Crossnohere
Break Out Room Discussion 1: Equity in shared decision-making conversations – Elisa Douglas & Frank Gavin
Break Out Room Discussion 2: Equity and accessibility in decision aids – Jesse Jansen & Karen Prantl
Break Out Room Discussion 3: Modelling: attending to equality and inclusion of biased on modelling – Anton Avanceña & Robin Wright-Jones
Keynote Speaker
Norah L Crossnohere
Assistant Professor at The Ohio State University
Norah L Crossnohere, PhD, is a patient-centered outcomes researcher. Her research areas include advancing methods for measuring patient and caregiver preferences; engaging patients to inform medical decision-making, and evaluating health information technologies.
Facilitators
Mary C. Politi, Professor @Department of Surgery, School of Medicine, Washington University in St. Louis
Ashley Housten, Assistant Professor @Department of Surgery, School of Medicine, Washington University in St. Louis
Elisa E. Douglas, PhD, MSPH, Research Scientist @Decision Support Lab, Department of Health Services Research, The University of Texas MD Anderson Cancer Center
Frank Gavin, MA, Chair @Public Advisory Council Health Data Research Network Canada
Jesse Jansen, PhD, Associate Professor @Family Medicine, Family Medicine, School for Public Health and Prim Care, Fac. Health, Medicine and Life Sciences
Senator (Ret.) Robin Wright-Jones, Executive Director @The Empowerment Network, Inc
Karen Prantl, Policy officer @Needs assessment and patient participation Dutch Kidney Patients Association (NVN)
Anton Avanceña, Assistant Professor @The University of Texas at Austin
Edward Mills PhD, FRCP
Professor, McMaster University
Craig R Rayner FRCP Edin PharmD MBA
Certara Distinguished Scientist
Adjunct Associate Professor, Monash University
Keywords
COVID-19; Ivermectin; TOGETHER trial; Patient engagement; Adaptive platform trial
Key Points
The TOGETHER trial is a randomized adaptive platform trial investigating 11 different treatments for COVID-19 including Ivermectin.
Initial in vitro experiments of Ivermectin in April of 2020 showed promising results, but many of those original papers were later retracted.
The TOGETHER trial used PBPK modelling to determine trial dose regimen. The trial started with 1 dose and later moved to 3 doses.
The primary outcome of the TOGETHER trial was emergency room visits due to COVID-19 or hospitalization due to the progression of COVID-19.
No evidence was found of treatment effect for ivermectin compared to placebo, but a small treatment effect cannot be ruled out. It may have an effect for a subgroup of patients.
Discussion Themes
A low confidence interval could signal that the trial was stopped too early.
Building trust within the community is key to the success of a trial. Recruitment is difficult if the patients don’t trust the trial or researchers.
The MIPACT study combined patient reported outcome data with electronic health record data and data collected from wearable devices.
The MIPACT study followed over 7,000 participants for 3 years. Both in-person and virtual recruitment had similar success rates. Participant diversity was a priority during recruitment.
The VALENTINE Study was a prospective randomized controlled study using mobile wearable devices to enhance cardiac rehabilitation.
Older participants in the VALENTINE study were receptive and capable of using wearable technology to assist data collection for the study.
THRIVE is pragmatic clinical trial studying Propofol anesthesia in 22 states and 2 countries.
Discussion Themes
Economic barriers to participation may be present when using wearable device technology as an inclusion criteria for a study. There are various methods to reduce these barriers, such as providing participants with the wearable device and a minimal data plan.
There may be limits to the types of participant reported outcomes that can be collected remotely. Ideally, participants will decide what data researchers will have access to.
Angelo E. Volandes, MD, MPH
Associate Professor, Harvard Medical School and Massachusetts General Hospital
James A. Tulsky, MD
Chair, Department of Psychosocial Oncology and Palliative Care
Dana-Farber Cancer Institute
Professor of Medicine, Harvard Medical School
ACP COVID combines the VitalTalk program and ACP DECISIONS videos to help patients and families navigate difficult decisions around advanced care planning.
VitalTalk is a non-profit organization providing evidence-based communication training. VitalTalk trained health care providers to communicate advanced care planning options with patients.
The ACP COVID study compared advanced care planning documentation rates at three specific timepoints: 6 months prior to COVID-19(control period, Sep 2019-Mar 2020), during the first wave of COVID-19(control period, March 2020-Sep 2020), and during the time period when vaccines were first rolled out in New York state(intervention period, Dec 2020-June 2021).
Advanced care planning conversations best happen in the outpatient setting, not in the hospital right before intubation.
Almost 24% of patients in the intervention period had Advance Care Planning conversations which was significantly higher than the control periods.
Discussion Themes
The clinician training program gave clinicians the skills to cope with the implications of the pandemic and led to increased clinician engagement.
Dr. Taylor-Zapata, a board-certified pediatrician, leads the NICHD’s Best Pharmaceuticals for Children Act clinical program, a drug development program that sponsors clinical trials to inform and improve pediatric drug labeling.
Jerry H. Gurwitz, MD
Chief, Division of Geriatric Medicine
University of Massachusetts Medical School and UMass Memorial Medical Center
Executive Director, Meyers Health Care Institute
A joint endeavor of University of Massachusetts Medical School, Fallon Health, and Reliant Medical Group
Richard Platt, M.D., M.Sc.
President, Harvard Pilgrim Health Care Institute
Topic
D-PRESCRIBE-AD: A Pragmatic Trial to Educate and Sensitize Caregivers and Healthcare Providers to Reduce Inappropriate Prescription Burden in Persons Living with Dementia
Keywords
Distributed Research Network; Dementia; Alzheimer’s Disease; Deprescribing
Key Points
The Distributed Research Network, with 45 million members currently accruing new data, facilitates the conduct of multi-center research requiring access to full text medical records and the collection of patient-generated data.
Inappropriate prescribing can increase the likelihood of adverse drug events and may have a heightened impact for Alzheimer’s patients.
D-PRESCRIBE-AD study is a randomized educational intervention to improve medication safety among Alzheimer patients by discontinuing potentially inappropriate prescriptions.
The D-PRESCRIBE-AD study found incidents of prescribing cascades to be less common than expected in the AD population, but prescription of high-risk medications were relatively high.
The challenges of the D-PRESCRIBE-AD study are deciding who should receive the provider letter and who is the appropriate caregiver of the patient.
Discussion Themes
One challenge of a study of this nature is getting buy-in from primary care providers.
Randomization was attempted at the level of Metropolitan Statistical Areas, but challenges with this idea proved too great. Randomization was instead done at an individual level.
Multiple arms of the study were necessary to answer questions about primary care doctor buy-in for this type of study.
Learn more about partnering with the DRN. Read more about. Read more about the D-PRESCRIBE-AD study.
The Society for Medical Decision Making (SMDM) will host a free, live webinar on Thursday, March 2, to discuss equity and patient engagement. The webinar, 