Grand Rounds Archive

Grand Rounds March 14, 2025: Spillover Due to Constraints on Care Delivery: A Potential Source of Bias in Pragmatic Clinical Trials (Sean Mann)

Posted on by Sophia Ramirez

Speaker

Sean Mann
Senior Policy Analyst
RAND Corporation

Keywords

Development Economics; Spillover; Bias; Pragmatic Clinical Trials

Key Points

  • Individually randomized, parallel-group embedded pragmatic trials randomly assign patients to a new healthcare intervention or to a control group that receives some form of usual care. They take place in real-world health settings, where clinical resources can be scarce.
  • If an intervention increases patient use of scarce resources – e.g., appointments, hospital beds, provider attention – this can reduce the availability of those resources for the control group and bias results. This is known as negative spillover, or crowding out, in the field of development economics. However, spillover due to resource constraints is absent from the major frameworks that inform clinical trial methods.
  • 4 conditions must be met for spillover to affect trial results: 1) Resources are shared across trial arms 2) Care delivery resources are constrained or limited in some way 3) The intervention being studied affects utilization of the scarce resource 4) Having fewer resources available to care for patients affects their health outcomes. These conditions are fairly common in pragmatic trials.
  • Spillover is not just a source of bias; it has implications for patient safety. If negative spillover occurs, patients who are assigned to a control arm are no longer receiving usual care as it’s commonly understood. Mann provided strategies that research teams can use to detect spillover or avoid it entirely, including utilizing a cluster-randomized design or a demand-balanced trial design.
  • Spillover remains hypothetical, and positive spillover is also possible. Study results affected by spillover can still be a valid indication that an intervention affects care utilization.
  • In many pragmatic trials, spillover is likely negligible. Mann and his colleagues at Rand are trying to help determine when spillover concerns should prompt changes to study design or justify additional data collection and analysis. They recently published a paper in Trials that explores the issue in further detail: Mann, S. Negative spillover due to constraints on care delivery: a potential source of bias in pragmatic clinical trials. Trials 25, 833 (2024). https://doi.org/10.1186/s13063-024-08675-9

Discussion Themes

While the Cochrane Risk of Bias Tool v. 2.0 (RoB 2) can be useful for looking at disparities in the level of care provided to the control group versus the intervention group, it exclusively looks at bias due to lack of blinding.

IRB applications could be expanded to include further questions about how resources are being used at a study site, how a study may potentially increase demands on those resources, and how it might affect others who are receiving those health services.

Clinical settings aren’t necessarily a zero sum game; negative spillover will apply in certain contexts, while in others the presence of an intervention will lead to positive spillover or no effect.

Grand Rounds March 7, 2025: A Trial of a “Kidney Action Team” for Hospitalized Patients with Acute Kidney Injury (F. Perry Wilson, MD MSCE)

Posted on by Sophia Ramirez

Speaker

F. Perry Wilson, MD MSCE
Associate Professor of Medicine and Public Health
Director, Clinical and Translational Research Accelerator
Yale University
New Haven, CT

Keywords

Electronic Alerts; Acute Kidney Injury; Action Team

Key Points

  • Acute Kidney Injury (AKI), or abrupt decline in kidney function, is common, affecting about 15% of hospitalized patients. A hospitalized patient with AKI has an inpatient mortality rate 8.5% higher than average. However, early recognition and nephrologist involvement can improve clinical outcomes.
  • First, the research team conducted a multicenter, parallel-group, randomized controlled trial (RCT) to test the effect of an electronic alert system on best practice utilization and three clinical outcomes (progression of AKI, dialysis, and death). The pragmatic trial, Electronic Alerts for Acute Kidney Injury Amelioration AKA ELAIA-1, instituted “best practice alerts” in the electronic medical record.
  • The Grand Unified Theory of Electronic Alerts states that alerts can’t work if 1) the provider already knows what’s wrong with the patient; 2) they don’t care about what’s wrong with the patient; 3) they have no specific action to take in response; or 4) the action doesn’t matter – i.e., it doesn’t change outcomes.
  • For ELAIA-2, the second iteration of the trial, the researchers focused on one tenet of this theory: alerts should be tied to actions. This open-label, parallel group RCT used alerts to encourage cessation of kidney-relevant medications – NSAIDs, RAASi, and PPI. They looked at rates of cessation of one of those medications and clinical outcomes (progression of AKI, dialysis, and death).
  • Though they found that automated alerts for AKI can increase medication cessation, there was limited evidence that these alerts would change clinical outcomes. The rate of discontinuation was highest for PPIs, which are an under-recognized contributor to AKI. Alerts may be beneficial in physicians whose patients are receiving PPIs – a population that tends to be sicker.
  • These findings led to a new hypothesis: AKI is heterogenous, caused by many factors. The research team sought to customize recommendations given to providers. To this end, they created a Kidney Action Team (KAT) with the goal of improving in-hospital mortality and AKI progression.
  • The KAT-AKI trial, a multicenter RCT administered across 2 hospital systems, looked at the proportion of recommendations implemented in 24 hours and clinical outcomes (progression of AKI, dialysis, and death). 34% of KAT recommendations were implemented within 24 hours in the intervention arm compared to 24% in usual care. However, this also did not have an effect on clinical outcomes.
  • The research team concluded that more personalized AKI alerts could potentially lead to better outcomes.

Discussion Themes

An optimized user interface – e.g., delivering information at the right place in a provider’s work flow, catching them at the right time – may further increase the adoption of best practices.

There’s some doubt about the nephrotoxicity of RAAs. In the ELAIA-2 study, though there were higher rates of RAA discontinuation in the intervention group, the clinical outcomes were nearly identical.

The heterogeneity of AKI patients interfered with the intervention’s effectiveness. To improve clinical outcomes, researchers may want to devote resources to prevention rather than response.

Grand Rounds February 28, 2025: Behavioral Economic and Staffing Strategies To Increase Adoption of the ABCDEF Bundle in the Intensive Care Unit (BEST-ICU): Protocol, Challenges, and Major Updates (Eduard Vasilevskis, MD, MPH; Michele C. Balas PhD, RN, CCRN-K, FCCM, FAAN)

Posted on by Sophia Ramirez

Speakers

Eduard Vasilevskis, MD, MPH
Professor of Medicine
Department of Medicine, Division of Hospital Medicine
University of Wisconsin-Madison

Michele C. Balas PhD, RN, CCRN-K, FCCM, FAAN
Associate Dean of Research
Dorothy Hodges Olson Distinguished Professor of Nursing
University of Nebraska Medical Center College of Nursing

Keywords

Intensive Care; ICU; Implementation; Evidence-Based Practices

Key Points

  • With recent increases in survivorship for Intensive Care Unit (ICU) patients has come the rise of Post-ICU Syndrome, or PICS. PICS is characterized by cognitive and physical impairment; financial toxicity; and family impacts. Some of the factors associated with PICS are modifiable: Sedation use, for example, immobility, and mechanical ventilation.
  • A set of evidence-based best practices based on these modifiable factors are encapsulated in the ABCDEF Bundle: Assess, prevent, and manage pain; Both spontaneous awakening trials (SATs) and spontaneous breathing trials (SBTs); Choice of analgesia and sedation; Delirium: assess, prevent, and manage; Early exercise and mobility; and Family engagement.
  • In a study of 68 ICUs, patients treated with all elements of the ABCDEF bundle in a given day had better outcomes. The next day, they were 70% less likely to be on a mechanical ventilator; 65% less likely to be in a coma; and 40% less likely to be delirious. Though there wasn’t a significant impact on pain, the likelihood of being discharged from the hospital increased by 20%.
  • We have a safe and efficacious set of evidence-based practices that people can deliver in the ICU. However, many of them are not being delivered to critically ill patients. And they’re not being delivered due to numerous implementation challenges clinicians experience in everyday care.
  • The Behavioral Economic and Staffing Strategies to Increase Adoption of the ABCDEF Bundle in the Intensive Care Unit (BEST-ICU) study aims to evaluate 2 strategies grounded in behavioral economic and implementation science theory to increase adoption of the ABCDEF bundle. The strategies target a variety of ICU team members and known behavioral determinants of ABCDEF bundle performance.
  • BEST-ICU is ongoing. The hybrid type III effectiveness-implementation pragmatic trial will take place in 3 hospitals and 12 ICUs across 33 months. The study team will monitor fidelity through real-time monthly tracking of audit and feedback information and through direct observation by Registered Nurse (RN) Implementation Facilitators.
  • Over 3,000 work intensity surveys have already been completed, split between RNs and non-nurses. Given the intensive nature of these surveys and the dearth of studies investigating work intensity in the ICU, this alone will be a notable contribution to the literature.
  • The research team outlined some of their sticking points around dashboard development and data acquisition/sharing, as well as how they’ve addressed these challenges. Solutions included the standardization of definitions for bundle process elements and engagement of clinical, operational, and legal leadership from the University & Health system.

Discussion Themes

Patient-reported pain was the only outcome that didn’t improve following full implementation of ABCDEF bundle. Dr. Balas noted that patients who aren’t in a coma anymore can then report pain. Dr. Vasilevskis pointed out that the same goes for delirium, which is far preferable to coma from a mortality perspective.

Dr. Balas suggested that a patient who is more cognitively engaged and able to report pain is actually in a better spot, as it enables medical staff to treat them.

Grand Rounds February 21, 2025: Texting for Behavior Change: Lessons Learned Across 2 Interventions to Improve Chronic Care Management (Michael Ho, MD, PhD; Sheana Bull, PhD)

Posted on by Sophia Ramirez

Speakers

Michael Ho, MD, PhD
Kaiser Permanente Colorado

Sheana Bull, PhD
University of Colorado School of Public Health

Keywords

Text Messaging; Artificial Intelligence; Chatbots; Health Behaviors

Key Points

  • Ample evidence now exists demonstrating the benefit of using text messaging in support of health behavior and access to care. It’s ubiquitous, increasing reach; theory in message design is impactful; and it can improve adherence to medical appointments and health behaviors.
  • Two NIH Collaboratory Trials, Nudge and Chat 4 Heart Health (C4HH), test the effectiveness of text messaging interventions to support behavior change. Nudge randomized patients to receive usual care, generic texts, behavioral texts, or behavioral texts plus chatbot messages. Their primary outcome was medication adherence.
  • C4HH, the subsequent trial, is randomizing patients to receive a generic text message curriculum; an AI chatbot messaging curriculum; or AI chatbot messages plus proactive pharmacist support. Their primary outcome is cardiovascular risk factors, as measured by the American Heart Association’s “Life’s Essential 8” adherence.
  • Nudge used an opt-out consent approach where CC4H used an opt-in consent approach. In the former, the research team noted, patients who identified as Black, Hispanic, and primary Spanish speakers were more likely to remain in the study. An opt-out approach in the appropriate context may be a way to diversify clinical trial populations and improve external validity of results.
  • The use of AI chatbots allows users to generate questions in their own words and the system to retrieve a response from a closed, curated library.
  • Message engagement is key to text messaging interventions. Participants in the Nudge study who were randomized to optimized texts had more questions. Questions were related to medications, refill logistics, and costs. The study team hypothesizes that the optimized texts may have led to greater patient engagement, and therefore more questions about their medications.
  • Over 12 months, the Nudge study found no significant difference in the rates of prescription refills, between the 3 intervention arms and usual care. CC4H is ongoing, and will send a higher volume of messages in an effort to engage patients and change patient behavior.
  • So far, the top 5 topics in messages initiated by C4HH participants have been healthy eating, physical activity, managing cholesterol, quitting smoking, and medication management.

Discussion Themes

The study team had to be very careful to ensure that patient health data, including cell phone numbers and the messages sent, were encrypted. Vendors and phone carriers were not able to access this data and it was not stored on their servers.

One of the challenges they encountered was that their systems weren’t integrated into the health care organizations’ pharmacies or electronic health records. The integration piece will be key to any future sustainability.

As technology evolves significantly over the course of, say, a 5-year study, developing the skillset to utilize interactive interventions or a SMART design could be helpful for investigators interested in conducting research in this area.

Grand Rounds February 14, 2025: A Clustered-Randomized Stepped-Wedge Pragmatic Trial to Enhance Goals-of-Care Communication for Older Adults with Cancer (ACP PEACE) (Angelo Volandes, MD, MPH; James A. Tulsky, MD)

Posted on by Sophia Ramirez

Speakers

Angelo Volandes, MD, MPH
Vice Chair of Research, Department of Medicine
Dartmouth Health
Professor of Medicine, Geisel School of Medicine

James A. Tulsky, MD
Poorvu Jaffe Chair, Department of Supportive Oncology
Dana-Farber Cancer Institute
Professor of Medicine, Harvard Medical School

Keywords

Palliative Care; Goals of Care; Advanced Care Planning; Cancer; Oncology

Key Points

  • Cancer is one of the leading causes of death for people age 65 and older, and patients have diverse preferences for medical care. However, most patients are unfamiliar with advance care planning (ACP) and clinicians are often unprepared to discuss it. Ultimately, many patients do not have goals-of-care (GOC) conversations with their providers, leading to billions of dollars in unwanted (and painful) interventions.
  • The Advance Care Planning: Promoting Effective and Aligned Communication in the Elderly (ACP PEACE) trial tested a pair of interventions – ACP Decisions and Vitaltalk – to help prepare clinicians and elderly patients to have GOC conversations.
  • ACP Decisions consists of short, accessible video decision aids that aim to help patients share in decision making. They offer a scalable, speedy strategy to improve GOC, and were associated with an increase in GOC conversations in smaller trials.
  • Vitaltalk is a communication training for clinicians discussing goals of care in serious illness. It is evidence-based, designed around the idea that learning new skills requires practice, observation, and feedback. Vitaltalk is associated with documented improvement in conversation quality.
  • ACP PEACE was a large, pragmatic, multicenter stepped-wedge trial that compared an intervention period and a control period, AKA usual care. The trial occurred across three healthcare systems (A, B, and C) and began with 36 clinics, 6 months before the onset of the COVID-19 pandemic. In April 2020, the trial was re-randomized across 29 clinics.
  • Participants were 65 and older with advanced cancer. The primary endpoint was documentation of a GOC conversation.
  • The study team found a 6.8% increase in ACP documentation during the intervention period, driven by a 6.9% increase in documented GOC conversations. There was no statistically significant increase in documentation of referrals to palliative care or to hospice. There was no statistically significant decrease in the limitation of life-sustaining treatments.
  • The results varied significantly by healthcare system. In Healthcare System A, documentation decreased by nearly 8% over the course of the study – whereas in Healthcare Systems B and C, it increased by 11% and 8.3%, respectively.
  • The researchers considered whether GOC conversation documentation was the right target. There has been a conversation in palliative care about how helpful these documents are; goal-aligned medical care (goal concordance) might be a better target.
  • Volandes and Tulsky noted that the approach to pragmatic clinical trials should better reflect the shifting nature of healthcare in the U.S. Researchers in this space must be better stewards of limited resources, so that interventions are sustainable and outcomes are useful to decision-makers.

Discussion Themes

Efforts to enhance rigor in intervention development may prevent effective interventions from getting to scale. Leaders in this space need to make it easier to get implement and test interventions, even if that means they aren’t perfectly optimized.

Right now, researchers are disincentivized to move quickly during the UG3 start-up year. Funders could incentivize research teams to move more quickly, e.g., by encouraging them to use leftover funds to conduct another study.

People in healthcare don’t see 10 years ahead – they see 1 to 2 years ahead. If pragmatic trials are meant to inform decision makers, it’s incumbent on researchers to provide the information healthcare systems need in a manner that is not outdated.

Ongoing partnerships with appropriate health systems can provide a platform for more efficient intervention implementation across different areas of healthcare. For instance, the research team conducted three other trials in Healthcare System C within the time it took them to complete ACP PEACE.

Grand Rounds February 7, 2025: Improving Symptom Control in Pediatric Cancer Patients With SSPedi and SPARK (Lillian Sung, MD, PhD)

Posted on by Sophia Ramirez

Speaker

Lillian Sung, MD, PhD
Canada Research Chair in Pediatric Oncology Supportive Care
Division of Haematology/Oncology
Chief Clinical Data Scientist
The Hospital for Sick Children
Ontario, Canada

Keywords

Cancer; Pediatrics; Cancer Symptoms; Symptom Control

Key Points

  • Symptom control in pediatric cancer patients is very poor; almost all children undergoing treatment for cancer will experience difficult symptoms, including fatigue, changes in hunger, pain, and nausea or vomiting.
  • The Multinational Association for Supportive Care in Cancer issued ambitious goals for care of cancer patients in 2030, including routine symptom screening to facilitate timely individualized care and pairing symptom identification with evidence-based treatment.
  • With this goal in mind, the research team developed Symptoms Screening and Pediatrics tool, or SSPedi. SSPedi is a validated instrument for measuring self-reported symptoms that is simple, quick, and designed for use in clinical care.
  • They integrated SSPedi with care pathways through SPARK (Supportive care Prioritization Assessment and Recommendations for Kids), a web-based application that the team used to enroll patients and remind them to report their symptoms via SSPedi.
  • The researchers conducted two randomized controlled trials (RCTs): A cluster RCT in the United States, from July 2021 to August 2023, and an inpatient RCT in Canada, from July 2018 to September 2023. In both cases, they randomized cancer patients, aged 8 to 18, to either symptom screening or usual care. In the U.S. study, they worked with the sites to adapt 14 care pathways.
  • Their primary outcome was the patient’s symptom severity, measured by their SSPedi score.
  • In both trials, the research team found a reduction in symptom severity for the intervention group. The benefits were more pronounced in the U.S. trial, with statistically significant improvements for 12 out of 15 symptoms (compared to 8 out of 15 in the Canadian trial).
  • In the U.S. study, symptom-specific interventions (i.e., treatment was clearly provided to address a given symptom) were most common for hurt or pain; changes in hunger; constipation; peripheral neuropathy; feelings of disappointment or sadness; and nausea and vomiting. The first four symptoms were also identified as being documented or treated more often in the Canadian study, in addition to feelings of crankiness or anger.
  • The research team concluded, following both the Canadian and U.S. studies, that symptom screening improves symptom control and that there was strong support for its integration into routine care.
  • There were a couple of additional takeaways from the U.S. study: 1) Care pathway use has independent effects on improving symptoms and 2) symptom screening was associated with increased emergency department visits – likely due to identification of symptoms requiring medical attention.

Discussion Themes

Despite launching right before the COVID-19 pandemic and the labor-intensive process of adapting the care pathways for each individual site, the team completed accrual for the U.S.-based-study about a year ahead of schedule. Dr. Sung attributed this to enthusiastic support from the sites around operationalizing supportive care and a study design which allowed patients to be enrolled remotely and report their symptoms remotely.

Pragmatic trials are a team sport. Having a range of expertise and experience can provide a research team with an advantage that makes all the difference. For example, Dr. Sung’s team had a connection to Epic Systems leadership, which facilitated a partnership between the two groups and allowed them to pilot SSPedi in remote care management.

Grand Rounds January 31, 2025: Advancing Rural Back Pain Outcomes Using Rehabilitation Telehealth (ARBOR-Telehealth): Early Progress and Lessons Learned (Richard L. Skolasky, ScD; Kevin H. McLaughlin, DPT)

Posted on by Sophia Ramirez

Speakers

Richard L. Skolasky, ScD
Professor, Johns Hopkins School of Medicine
Department of Orthopaedic Surgery

Kevin H. McLaughlin, DPT
Assistant Professor, Johns Hopkins School of Medicine
Department of Physical Medicine and Rehabilitation

Keywords

Back Pain; Rural Health; Telehealth

Key Points

  • Low back pain (LBP) is the most common cause of disability globally and in the U.S. It’s the largest driver of U.S. healthcare spending growth and a top non-cancer reason for opioid prescriptions.
  • Physical therapy (PT) is a first-line treatment for LBP. It may include exercise, hands-on interventions, and modalities. These methods are cost-effective and decrease the risk of opioid use and surgery. However, due to barriers around the requisite travel, missed work, etc., only 7 – 13% of patients with LBP attend PT.
  • LBP patients in rural areas, where there are higher rates of opioid use and high-impact chronic pain, attend PT less frequently. PT is less accessible than it is in metropolitan areas; there are up to 40% fewer therapists per person; and the travel distance required to get to a clinic is farther.
  • PT was provided virtually (AKA telerehabilitation) during the pandemic, but the effectiveness of telerehabilitation for chronic LBP is unknown. Notably, not all components of PT can be delivered by video visit.
  • The research team’s aims include examining the effectiveness of risk-informed telerehabilitation in reducing LBP-related disability and comparing the prevalence of opioid use among those receiving telerehabilitation and education at 12 weeks.
  • The team is currently in the enrollment phase. They will begin disseminating their results in September 2028.
  • The research team randomized half the group to risk-informed telerehabilitation and the other half to an educational control group. Both groups received best practice advice through the study website, and the control group received eight video visits with a physical therapist over two months.
  • Community engagement strategies include a patient advisory panel and stakeholder advisory committee made up of physical therapists, primary care providers, and rural and public health advocates.

Discussion Themes

One challenge to flag for future studies is that many patients requested virtual appointments outside of office hours. However, the therapists weren’t able to accommodate these requests.

Another challenge is the inconsistent quality of calls between participants. Most folks have access to a smart device; not everyone has access to a desktop, laptop, or tablet, which are preferred for video calls. To address this, the research team is sending participants a phone stand in advance. They also provide basic exercise equipment, including resistance bands and a yoga mat.

While some patients feel a lack of connection with their therapists via telerehabilitation, others have reported that they felt the connection was stronger given the one-on-one nature of the appointments.

Grand Rounds January 24, 2025: The HEALing Communities Study – 10 Million People, 67 Communities: A Community-Based Cluster Randomized Trial to Reduce Opioid Overdose Deaths (Jeffrey H. Samet, MD, MA, MPH)

Posted on by Sophia Ramirez

Speaker

Jeffrey H. Samet, MD, MA, MPH
John Noble Professor in General Internal Medicine & Professor of Public Health
Boston University Chobanian and Avedisian School of Medicine and School of Public Health
Boston Medical Center

Keywords

Opioid Epidemic; Evidence-Based Practices; NIH HEAL Initiative; MOUD; OEND; Harm Reduction

Key Points

  • Over roughly 20 years, opioid overdose (OD) mortality in the U.S. increased tenfold: from a little over 8,000 in 1999 to over 80,000 in 2022. Health authorities urged medical professionals to address the crisis through stigma reduction, uptake of opioid use disorder (OUD) treatment, and other evidence-based practices (EBPs).
  • The goal of the HEALing Communities Study (HCS) was to reduce opioid OD through implementation of EBPs, including overdose education and naloxone distribution (OEND); access to medications for OUD (MOUD); and safer opioid prescribing and dispensing practices.
  • The research team conducted the cluster randomized trial in 67 communities across Ohio, Kentucky, New York, and Massachusetts – a total study population of about 10 million. HCS is the largest implementation science study funded by NIDA.
  • The primary objective of HCS was to compare the number of opioid OD deaths in adults during the comparison period (July 2021 – June 2022) between the intervention and control communities. Secondary outcomes included the rates of naloxone distribution; access to or utilization of MOUD; opioid + stimulant OD deaths; and non-fatal overdose events.
  • The intervention involved 3 pathways for implementing OUD EBPs: community engagement; the Opioid Reduction Continuum of Care Approach (ORCCA), consisting of a menu of strategies to support implementation of EBPs; and community-based health communications campaigns. Multi-level partnerships, Dr. Samet noted, are critical to the success of community-engaged research.
  • Between the intervention group and the control group, there was no difference in the number of opioid OD deaths. OD deaths involving opioids and psychostimulants (excluding cocaine) decreased by 37% in the intervention group, and there was a 15% reduction in nonfatal overdoses.
  • Factors that may have impacted results included the complex array of strategies; a limited period of time in which to achieve full benefits from the implementation of EBPs; COVID-19-related demands on coalition members and healthcare personnel; increasing rates of fentanyl in the drug supply with stimulant contamination; and statistical power.
  • Limitations included the fact that control communities could still access non-HCS funds to address the opioid epidemic and a variation in the affected population size within each community.

Discussion Themes

The research team had 18 months to get the intervention up and running; when the comparison period began, they were at 35% implementation – lower than their goal. This was partially a product of the stakes; the death toll of the opioid epidemic was high and the team was motivated to make quick progress. The COVID-19 pandemic also slowed things down.

Dr. Samet emphasized that the HCS team wasn’t testing whether MOUD and OEND worked; there is significant evidence demonstrating that these strategies are effective. Rather, the team was testing whether communities adopted them and if they had an effect on OD reduction.

When the team shared the results back with community partners, they heard anecdotal reports about the positive effects their partners had witnessed in their communities.

Engagement coalitions formed across communities. Ohio and Kentucky utilized existing coalitions while New York and Massachusetts built new ones. Dr. Samet noted that each of the four states performed well in different realms.

Grand Rounds January 17, 2025: Design for Diversity: Designing Studies for Representativeness and Generalizability (Christopher J. Lindsell, PhD)

Posted on by Sophia Ramirez

Speaker

Christopher J. Lindsell, PhD
Professor and co-Chief, Biostatistics, Duke University
Director, Data Science and Biostatistics, DCRI
Director, Biostatistics and Bioinformatics, CTSI
Editor in Chief, Journal of Clinical and Translational Science

Keywords

Study Design; Diversity; Health Disparities; Evidence Generation

Key Points

  • Health disparities are factors that contribute to preventable differences in health status and outcomes. They can be environmental, sociocultural, behavioral, and biological. These are preventable differences with adverse effects for populations.
  • When research teams don’t consider factors that change outcomes for certain populations but not others, research can contribute to a difference in health status and outcomes. A flawed evidence generation system compounds the problem.
  • One popular solution is to measure and adjust for diversity variables; however, research teams often get the variable wrong or use it incorrectly.
  • By designing for diversity, research teams can begin to address the generalizability of evidence; develop an understanding of factors that contribute to success or failure of interventions among diverse populations; and remove the evidence generation system as a contributor to health disparities.
  • Designing for diversity is an optimization problem. Historically, study designs have been optimized for the researcher; Dr. Lindsell proposed that researchers optimize for the participant.
  • Research that is optimized for the participant is rigorous and flexible; safe and practical; and complete and simple. Participants should be embedded in every part of the research process. This can be difficult – there are tradeoffs involved – but it is effective.
  • The interface between data generation and data use is crucial. Making systems that work to bring in the right information and systems that use that information appropriately are two pieces of the same puzzle.
  • Dr. Lindsell included a call to ditch the ordinary subgroup analysis, noting that groups are not binary and not all groups have meaning. He suggested a focus on interaction terms.

Discussion Themes

Institutions should be supporting research teams in their ability to achieve diversity; this should be a matter of course, rather than something to be achieved without support and then celebrated as a remarkable accomplishment.

Looking forward, as data infrastructure becomes increasingly robust, research teams and communities may be able to collaborate to build a more complete understanding of individuals’ health states and who may be in need of an intervention.

Grand Rounds January 10, 2025: FM-TIPS Community Engagement Methods for Recruitment (Dana Dailey PT, PhD; Heather Schacht Reisinger, PhD)

Posted on by Sophia Ramirez

Speakers

Dana Dailey PT, PhD
Assistant Research Scientist
Physical Therapy and Rehabilitation Science
University of Iowa
Associate Professor, Physical Therapy Department
St. Ambrose University

Heather Schacht Reisinger, PhD
Director, Implementation Science Center
Associate Director for Engagement, Integration, and Implementation
Institute for Clinical and Translational Science
Professor, Division of General Internal Medicine at the University of Iowa

Keywords

Community-Engaged Research; Recruitment

Key Points

  • The Fibromyalgia TENS in Physical Therapy (FM-TIPS) study sought to test the feasibility and effectiveness of adding Transcutaneous Electrical Nerve Stimulation (TENS) to standard physical therapy (PT) care in a real-world PT setting. Their primary outcome was the change in movement pain, from baseline to 50 days.
  • By February 2022, the team had activated 25 active PT clinics across seven Midwestern states. In February 2023, the team received a diversity supplement for community engagement and brought on a Community Engagement Coordinator.
  • After developing a community engagement process and implementing six community engagement strategies, inquiries about the study increased significantly, screening rates held steady, and enrollment rates increased. These effects were more pronounced in targeted clinics.
  • Engagement with the clinics, clinicians, and community had a significant impact on the pragmatic trial. Dr. Dailey noted that clinicians felt more supported and seen as a part of both the clinic and the community. Conversations with participants were also fruitful, helping the researchers understand how participants were feeling when they weren’t in therapy.
  • Clinical research in community physical therapy clinics is not common, and there was a steep learning curve for many of the physical therapists involved. Community support helped them navigate screening and enrollment.
  • The study team developed individualized clinic plans through inquiry of clinicians and clinics. This feedback helped them identify organizations to collaborate with, local events, etc.
  • Process building, including implementation and complete documentation of outcome measures, was important to site evaluation. As a result of these activities, the research team was able to carry lessons over into low-enrolling clinics, adapt, and make productive changes.

Discussion Themes

When researchers think about pragmatic trials involving clinics, Dr. Reisinger noted, it becomes difficult to delineate the concepts of “community members” and “participants.” For example, it’s important to also think about clinicians also as community members who are living and working in those spaces, and who have a deep knowledge of and commitment to their community. All of that knowledge will be important to decision-making.

Intensive and responsive community engagement was made possible for this pragmatic trial by the diversity supplement, which enabled the team to hire two full-time community engagement coordinators. At the rate they were going before they implemented community engagement, the study team would not have hit their enrollment target; in the end, they exceeded it.

Researcher teams should always be adaptive and flexible when doing community engagement, even when it comes to documentation.