PCORI

Grand Rounds February 13, 2026: The Making of the COMPARE-Pediatric IBD Study (Michael D. Kappelman, MD, MPH)

Posted on by Sophia Ramirez

Speaker

Michael D. Kappelman, MD, MPH
Professor, Pediatric Gastroenterology
University of North Carolina at Chapel Hill

Keywords

PCORnet; PCORI; Inflammatory Bowel Disease; Pediatrics; Common Data Model; Study Design

Key Points

  • Inflammatory Bowel Disease (IBD) is a chronic gastrointestinal condition affecting roughly 100,000 youth in the United States. It has a profound impact on nutrition, growth, physical, and psychosocial development. Anti-TNF biologics are the only FDA-approved advanced therapies for children, and approximately 30% of patients experience treatment failure within 2 years. There’s an urgent need for comparative effectiveness research that can guide treatment decisions when anti-TNF fails.
  • COMPARE-Pediatrics IBD, a PCORnet® study, includes 2 parallel multi-center, prospective cohort studies and retrospective cohort studies. The former, developed with multi-stakeholder input, will compare the effectiveness of emerging therapies in children with IBD; the latter will characterize the safety of these treatments and explore the heterogeneity of treatment effects across subgroups.
  • The study is utilizing PCORnet’s® infrastructure, including Prep-to-Research Queries and the PCORnet® Common Data Model (CDM), to inform the study design; identify administrative efficiencies; support recruitment; ease site burden; assess representativeness of the study population; and otherwise bolster their research.

Discussion Themes

Planning a PCORnet® study is a lot of work (and takes time). Start the process early and know that benefits may be on the back-end.

The study team opted not to conduct a randomized pragmatic trial because they anticipated that desperate families would be reluctant “roll the dice” with randomization and because insurance coverage for expensive off-label medications often dictates which therapy a patient can receive.

While the CDM is effective for structured data (like labs and diagnoses), Dr. Kappelman noted it cannot yet capture nuanced interpretations, such as specific MRI findings, which require more advanced AI or manual review.

November 4, 2024: Update to Living Textbook Offers Tips for Developing a Compelling PCORI Grant Application

Posted on by Karen Staman

Living Textbook iconThe NIH and the Patient-Centered Outcomes Research Institute (PCORI) are major funders of pragmatic clinical trials embedded in healthcare systems. An existing chapter of the Living Textbook of Pragmatic Clinical Trials provided guidance on how to Develop a Compelling Grant Application for the NIH. The updated chapter now includes information about how to develop a grant application for PCORI.

While both organizations share common goals to advance public health, PCORI-funded research requires investigator and patient/community partnership throughout the entirety of the research process from design through results dissemination and implementation.

The updated chapter provides practical advice about how to develop and submit an application in the following sections:

  1. Find the Right Program Official and Study Section
  2. Find the Notice of Funding Opportunity
  3. Write a Strong Proposal That Addresses Review Criteria
  4. Review Criteria
  5. Diversity, Disparities, and Inclusion Across the Lifespan
  6. Award Status
  7. Additional Resources

Regardless of whether the submission is for NIH or PCORI, first and foremost, investigators should develop and clearly define a clinical research question with a testable hypothesis and select an experimental design best suited to answering the research question.

March 25, 2024: PCORI Announces Foundational Expectations for Partnerships in Research

Posted on by Damon Seils

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.

June 27, 2023: Reflections on Health Equity in the Pragmatic Trials Landscape From the Annual Steering Committee Meeting

Posted on by Damon Seils
Headshots of Anne Trontell and Ana Quiñones
Anne Trontell and Ana Quiñones

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.”

Learn more about the NIH Pragmatic Trials Collaboratory’s Health Equity Core and see the complete materials from the 2023 Steering Committee Meeting.

April 10, 2023: Li Receives New PCORI Award to Develop Causal Inference Methods for Stepped-Wedge Cluster Randomized Trials

Posted on by Damon Seils
Headshot of Dr. Fan Li
Dr. Fan Li

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.

The new study, entitled “Toward Improved Design and Analysis of Stepped Wedge Trials: An Estimand-Aligned and Efficiency-Focused Framework,” will contribute new methods and software for planning and analyzing stepped-wedge cluster randomized trials that enable investigators to (a) target transparent causal estimands under the counterfactual outcomes framework and (b) to leverage baseline information for achieving higher statistical efficiency.

This is Li’s second PCORI award. Read a summary of his previous PCORI award.

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.

March 22, 2023: A Decade of PCORnet Research in This Week’s PCT Grand Rounds

Posted on by Damon Seils

Headshots of Erin Holve, Russell Rothman, Schuyler Jones, and Neha PagidipatiIn this Friday’s PCT Grand Rounds, Erin Holve, Russell Rothman, Schuyler Jones, and Neha Pagidipati will present “From Observational Studies to Pragmatic Clinical Trials: (Almost) A Decade of Research in PCORnet®.” The Grand Rounds session will be held on Friday, March 24, 2023, at 1:00 pm eastern.

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.

Join the online meeting.

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

November 5, 2021: Benefits of Stroke Treatment Delivered Using a Mobile Stroke Unit Compared to Standard Management by Emergency Medical Services: The BEST-MSU Study (James C Grotta, MD)

Posted on by terren.green@duke.edu

Speaker

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.

 

Read more about The BEST-MSU study here and in the paper publish in September 2021 “Prospective, Multicenter, Controlled Trial of Mobile Stroke Units.”

 

Tags

#pctGR, @Collaboratory1

October 8, 2021: Lessons Learned and Patient Partnership in ADAPTABLE (Schuyler Jones, MD; Madelaine Faulkner Modrow, MPH)

Posted on by terren.green@duke.edu

Speakers

Schuyler Jones, MD
Associate Professor of Medicine
Duke University School of Medicine

Madelaine Faulkner Modrow, MPH
Program Director
Department of Epidemiology and Biostatistics
University of California, San Francisco

Topic

Lessons Learned and Patient Partnership in ADAPTABLE

Keywords

Patient engagement; Pragmatic clinical trial; Aspirin; ADAPTABLE; Study design; Stakeholder engagement; Recruitment

Key Points

  • 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.

 

Read more about the ADAPTABLE trial.

Tags

#pctGR, @Collaboratory1

August 26, 2021: Li Receives PCORI Award to Study Methods for Cluster Randomized Trials

Posted on by Damon Seils
Headshot of Dr. Fan Li
Dr. Fan Li

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.

The study, entitled “New Methods for Planning Cluster Randomized Trials to Detect Treatment Effect Heterogeneity,” will contribute new methods, guidance, and user-friendly software for planning parallel and stepped-wedge cluster randomized trials to enable confirmatory “heterogeneity of treatment effect” (HTE) analyses with sufficient statistical power.

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.