research methods

Grand Rounds September 18, 2026: The 4th Trimester Initiative: A Randomized Controlled Trial of Support for Childbearing Medical Trainees (Erika Rangel, MD)

Posted on by terren.green@duke.edu

Speaker

Erika Rangel, MD
Associate Professor of Surgery
Department of Surgery
Harvard Medical School
Medical Director for Clinician Wellbeing
Mass General Brigham

Keywords

Childbearing medical trainees, Burnout, Parental support, Pregnancy support

Key Points

  • Previous studies have shown that surgical residents experience difficulties during medical training and pregnancy and early parenthood. Modified schedules were rare, less than 6 weeks parental leave was common, pumping was difficult, and mentorship regarding balancing family and career was lacking. Many questioned the sustainability of a surgical career affecting an already existing workforce shortage.
  • The aim of this study was to improve the work environment and culture for the vulnerable group of childbearing medical trainees while preserving the rigor and quality of the training program.
  • Study outcomes were chosen in 3 domains that reflect positive return on investment for the workplace. Domains included well-being (burnout), workforce outcomes, and functioning at work and home. Burnout was used as the primary outcome; burnout costs healthcare organizations $4.6 billion a year and has a human cost to the trainee as well.
  • The pragmatic design of the study lessened the burden on participating institutions by including interventions that could be delivered directly to the trainee. The parental support package included a wearable breast pump, a smart bassinet, faculty mentorship, and 24/7 virtual prenatal care via an app.
  • Across 7 institutions, 156 pregnant residents and fellows were enrolled and randomized to either usual support or the parental support package group. All participants were given a fitbit to track sleep and all were asked to track medical errors. The research question was deliberately phrased as ‘what happens when this parental support package was offered at an institution.’
  • The parental support package was used variably by participants. Institutional environments also varied. These variable factors were analyzed separately. The support package appeared to buffer the rise in burnout for the intervention group. Interpersonal disengagement was significantly improved and improved sleep decreased medical error.

Discussion Themes

Institutions understand that burnout is an issue for childbearing medical trainees, and response to the study was enthusiastic. Originally only 3 institutions were targeted, but 7 were ultimately included in the study.

Medical trainees were also enthusiastic to participate with a 92% retention rate. Word of mouth and social media were instrumental in recruitment.

 

Read more about the The 4th Trimester Initiative.

 

October 15, 2024: Case Study Describes a Reassessment of Sample Size in an Ongoing Cluster Randomized Trial

Posted on by Damon Seils

FM-TIPS logoA new case study from the NIH Pragmatic Trials Collaboratory highlights an interim reassessment of sample size during an ongoing cluster randomized trial. The case study was published this week in the Living Textbook of Pragmatic Clinical Trials.

Researchers in cluster randomized trials must account for potential correlation between clusters in the design and analysis of their trial by estimating the intraclass correlation when calculating the target sample size. Often they use preliminary data from the planned enrollment sites to estimate the correlation. However, when preliminary data are unavailable at the time of study design, they may use interim data collected during the trial itself to reassess the trial’s sample size.

The contributors of the case study focus on FM-TIPS, an NIH Collaboratory Trial, to describe an approach to conducting an interim reassessment of sample size in an ongoing trial. Read the full case study.

FM-TIPS is examining whether the addition of transcutaneous electrical nerve stimulation to routine physical therapy improves movement-evoked pain compared with physical therapy alone among patients with fibromyalgia. The trial is supported by the National Institute of Arthritis and Musculoskeletal and Skin Diseases through the NIH HEAL Initiative. Learn more about FM-TIPS.

The contributors of the case study include members of the FM-TIPS study team and leaders of the NIH Collaboratory’s Biostatistics and Study Design Core. David-Erick Lafontant is a statistician, Bridget Zimmerman is a clinical professor of biostatistics, and Emine Bayman is an associate professor of biostatistics—all at the University of Iowa. Megan McCabe is an assistant professor of biostatistics at the University of Alabama at Birmingham. Patrick Heagerty is a professor of biostatistics at the University of Washington. Liz Turner is an associate professor of biostatistics and bioinformatics at Duke University.

November 28, 2023: Workshop Summary Now Available From ‘Getting the Right Evidence to Decision-Makers Faster’

Posted on by Karen Staman

The workshop summary is now available from the NIH Pragmatic Trials Collaboratory’s recent workshop, “Getting the Right Evidence to Decision-Makers Faster.” The 2-day workshop explored the critical cycle of evidence generation by researchers to decision-making by healthcare system leaders to implement the findings of pragmatic clinical trials conducted within healthcare systems.

The workshop included 4 panels:

  • Panel 1: How Have Health Systems Made Decisions Based on Evidence Collected in PCTs?

    • Panelists: Devon Check, Vincent Mor, Lynn DeBar, Kathryn Glassberg, Douglas Zatzick, Eileen Bulger, Susan Huang, Kenneth Sands, Edward Septimus; Moderator: Gregory Simon

  • Panel 2: How Do We Generate the Right Evidence to Support Decision-Makers?

    • Panelists: Kenneth Sands, Eileen Bulger, Edward Septimus, Amy Kilbourne, Rosa Gonzalez-Guarda, Patrick Heagerty; Moderator: Hayden Bosworth

  • Panel 3: Learning Faster

    • Panelists: Gloria Coronado, Natalia Morone, Corita Grudzen, Kevin Chan, Pearl O’Rourke, Cheryl Boyce, Andrea Cook; Moderator: Kevin Weinfurt

  • Panel 4: Potential Structures and Incentives for Faster Learning

    • Panelists: David Chambers, Wynne Norton, Tisha Wiley, Kenneth Sands, Edward Septimus, Gloria Coronado, Natalia Morone, Corita Grudzen; Moderator: Richard Platt

Access the complete workshop materials, including slides and videocast recordings, as well as the keynote presentation by Andrew Bindman, executive vice president and chief medical officer for Kaiser Permanente.

September 5, 2023: NIH Pragmatic Trials Collaboratory Announces Grand Rounds Series on Design and Analysis of Pragmatic Clinical Trials

Posted on by Damon Seils

Promotional graphic showing details of the upcoming sessions of special Grand Rounds series, "Advances in the Design and Analysis of Pragmatic Clinical Trials"The NIH Pragmatic Trials Collaboratory is launching a special Grand Rounds series to share advances in the design and analysis of pragmatic clinical trials.

Join us on the first Friday of each month, October through January, to hear the latest best practices and explore emerging questions with experts from the program’s Biostatistics and Study Design Core.

Over the past decade, the Core has worked with investigators to fine-tune study designs, develop rigorous analysis plans, and offer guidance to the broader community of researchers who are planning pragmatic trials. With this new Grand Rounds series, the Core is bringing together biostatisticians, clinical trials methodologists, and investigators to discuss challenges and share lessons learned in the design, implementation, and analysis of pragmatic trials.

The webinar series, Advances in the Design and Analysis of Pragmatic Clinical Trials, will kick off on Friday, October 6, at 1:00 pm ET with a presentation on design and analysis considerations for implementation trials by David Murray, NIH associate director for disease prevention and director of the NIH Office of Disease Prevention.

The series will include 3 additional moderated webinar discussions. These sessions will focus on a range of topics, including complex clustering, best practices in the design and analysis of stepped-wedge trials, and handling missing data in cluster randomized trials.

Download the series flyer and see the full schedule below:

All sessions are free and open to the public; no registration is required. Recordings will be archived on the Rethinking Clinical Trials website.

Grand Rounds June 16, 2023: BeatPain Utah: Partnering With Community Health Centers Within a Socio-Technical Framework (Julie Fritz, PT, PhD, FAPTA; Guilherme Del Fiol, MD, PhD)

Posted on by Damon Seils

Speakers

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.

Tags

#pctGR, @Collaboratory1

Grand Rounds October 28, 2022: The HERO (Healthcare Worker Exposure Response & Outcomes) Program: An Online Community to Support Observational Studies, Randomized Trials, and Long-Term Safety Surveillance (Emily O’Brien, PhD, FAHA; Russell Rothman, MD, MPP)

Posted on by terren.green@duke.edu

Speakers

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

 

 

Keywords

HERO Registry; HERO TOGETHER; Hydroxychloroquine; COVID-19; PCORI; PCORnet

 

Key Points

  • 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.

Learn more

On the HERO Program website.

Discussion Themes

– 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. .

Tags

#pctGR, @Collaboratory1

December 16, 2021: NIH Collaboratory Publishes COVID-19 Checklist for Statistical Analysis Plans in Pragmatic Trials

Posted on by Damon Seils

Thumbnail image of the COVID-19 checklistA new tool from the NIH Collaboratory assists investigators in identifying impacts of the COVID-19 public health emergency on ongoing pragmatic clinical trials. The Statistical Analysis Plan Checklist for Addressing COVID-19 Impacts summarizes impacts on trial conduct that study teams should document, measure, analyze, and report.

The new checklist was developed by the NIH Collaboratory’s Biostatistics and Study Design Core Working Group. Since the beginning of the COVID-19 pandemic, many of the NIH Collaboratory Trials have had to postpone recruitment, alter methods of participant engagement, and modify tools for research assessment and intervention delivery.

The leaders of the Biostatistics Core, Dr. Patrick Heagerty and Dr. Liz Turner, spoke in a recent interview about the impacts of the pandemic on the NIH Collaboratory Trials. Early next year, the Coordinating Center will report the results of a survey of the study teams about their experiences with these impacts.

Download the Statistical Analysis Plan Checklist for Addressing COVID-19 Impacts.

November 29, 2021: New Article From the NIH Collaboratory Examines Use of Incentives and Payments in Pragmatic Clinical Trials

Posted on by Damon Seils
Head shot of Dr. Andrew Garland
Dr. Andrew Garland

Members of the NIH Collaboratory’s Ethics and Regulatory Core examined the use of incentives and payments to patients included in pragmatic clinical trials. Their findings and preliminary recommendations are published in the December issue of Clinical Trials.

Incentives and payments to patients are used in both pragmatic trials and conventional explanatory trials. However, because pragmatic trials typically evaluate interventions in the context of “real-world” clinical settings, the use of incentives and payments can raise logistical, ethical, and regulatory challenges.

Dr. Andrew Garland, a postdoctoral fellow at the Johns Hopkins Berman Institute of Bioethics who works in the Ethics and Regulatory Core, and who is the lead author of the article, reviewed 9 NIH Collaboratory Trials that used incentives and other payments to patients. Garland and coauthors Dr. Kevin Weinfurt and Dr. Jeremy Sugarman used these examples to describe how the standard conceptual framework for ethical payments and incentives may not always be appropriate for pragmatic trials.

Read the full report.

This work was supported within the NIH Collaboratory by the NIH Common Fund through a cooperative agreement from the Office of Strategic Coordination within the Office of the NIH Director. This work was also supported by the NIH through the NIH HEAL Initiative.

August 24, 2021: Trans-NIH Workshop Will Explore Methods for Whole Person Research

Posted on by Damon Seils

The National Center for Complementary and Integrative Health (NCCIH) will lead a trans-NIH workshop on methodological approaches for whole person research. The workshop will focus on the methods needed for rigorous research on interconnected systems, multicomponent interventions, and multisystem or multiorgan outcomes.

Registration for this event is required.

Event: Methodological Approaches for Whole Person Research Workshop
Date and Time: September 29-30, 2021; 11:30 am-5:30 pm ET
Location: virtual

The new NCCIH strategic plan identifies 3 components of whole person research: (1) exploring the fundamental science of interconnected systems; (2) investigating multicomponent interventions or therapeutic systems; and (3) examining the impact of these interventions on multisystem or multiorgan outcomes.

The “Methodological Approaches for Whole Person Research” workshop will provide examples of research in these 3 areas from diverse fields. The workshop will also identify opportunities for this type of research in complementary and integrative health and discuss relevant study designs, statistical methods, and computational approaches.

Workshop collaborators include the National Institute on Aging, the National Institute on Minority Health and Health Disparities, the National Institute of Nursing Research, the National Institute of Dental and Craniofacial Research, the Fogarty International Center, the Office of Research on Women’s Health, the Office of Behavioral and Social Sciences Research, the Office of Disease Prevention, and the Office of Nutrition Research within the NIH Office of the Director.

Read more about this workshop on the NCCIH events page. Free registration is required, and the workshop will be videocast and archived for future viewing.

July 8, 2021: New Living Textbook Section on Intervention Functions and Forms

Posted on by Gina Uhlenbrauck

Readers are invited to visit a recent addition to the chapter Monitoring Intervention Fidelity and Adaptations in the Living Textbook. The new content offers considerations for study teams who are preparing to track the fidelity of an embedded—and potentially complex—clinical intervention. Considerations include identifying which features of the intervention (i.e., functions) are so essential to its effectiveness that modifying them could negatively affect the study’s outcomes and impact. Equally important is knowing which elements (i.e., forms) can be adapted to accommodate contextual factors or local needs without affecting fidelity. The section provides an introduction and link to methodology standards for complex interventions recently developed by the Patient-Centered Outcomes Research Institute (PCORI).

“Functions of a complex intervention represent a purpose or goal, while forms are the tools or processes used to achieve a function. Identifying forms and functions allows adapted complex interventions to retain a level of standardization and integrity in design.” (Hill et al. 2020)