PCORnet

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.

February 11, 2026: The Making of the PCORnet® COMPARE-Pediatric IBD Study, in This Week’s Rethinking Clinical Trials Grand Rounds

Posted on by Sophia Ramirez

In this Friday’s Rethinking Clinical Trials Grand Rounds, Michael D. Kappelman of the University of North Carolina at Chapel Hill will present “The Making of the PCORnet® COMPARE-Pediatric IBD Study.”

The Grand Rounds session will be held on Friday, February 13, 2026, at 1:00 pm eastern.

Kappelman is a professor of pediatric gastroenterology at the University of North Carolina at Chapel Hill.

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Grand Rounds October 17, 2025: Making Effective Use of Data Infrastructure in PCORnet® (Charles Bailey, MD, PhD; Keith Marsolo, PhD)

Posted on by Sophia Ramirez

Speakers

Charles Bailey, MD, PhD
Department of Pediatrics
Perelman School of Medicine
University of Pennsylvania
Biomedical and Health Informatics
Children’s Hospital of Philadelphia

Keith Marsolo, PhD
Associate Professor
Department of Population Health Sciences
Duke Clinical Research Institute
Duke University School of Medicine

Keywords

PCORnet®; Data; Clinical Research Network; Patient-Centered Research; Common Data Model

Key Points

  • PCORnet® is a clinical research network that connects communities (namely providers; researchers; patients, caregivers, and advocates) and data (EHR, claims, and patient-reported). It functions as a learning health system to help researchers generate answers that advance health outcomes.
  • The network is made up of healthcare institutions, from large academic health centers to local community clinics. As of August 2025, PCORnet® had collated data from healthcare encounters in all 50 states, representing over 47 million people. There had been 57 PCORnet® studies and 991 publications supported by PCORnet® resources.
  • To be useful, data have to be standardized across systems. Frequent data curation and a single language enabled by the PCORnet® Common Data Model (CDM) facilitates this. Data that are in the CDM and currently available for use in research include demographics, diagnoses, and vital signs. Data that may or may not be in the CDM and require additional work for research include immunizations, social determinants of health, and patient-reported outcomes.
  • Quarterly, the PCORnet® team executes a data curation process. This includes a range of checks looking at data completeness; plausibility; persistence; and conformance to the PCORnet® CDM. Over the last decade, PCORnet® network performance has improved in terms of data mapping and latency.
  • Researchers can approach the PCORnet® Front Door with both simple univariate and bivariate statistical questions – i.e. how often a particular medication is used within the PCORnet® population – and with prep-to-research queries, which may identify an eligible population and generate some information about how that population behaves.
  • Once a team is running a PCORnet® study, they can submit queries for study-specific data extracts. This involves identifying a cohort and extracting patient-level data.
  • In the near future, PCORnet® will include additional data visualization options to increase the ease of navigating complex results. The team is also working on a Query Tools repository that will show what other people have already asked about a given set of data.
  • Because each study operates on specific variables and general characteristics do not predict specific characteristics, study-focused assessment of data fitness is critical.
  • The presenters walked attendees through 5 different PCORnet® studies and how they utilized this data infrastructure in their projects.

Discussion Themes

There is no charge for Front Door queries; they are part of the research engagement process. However, prep-to-research queries are limited to those that can be turned around in a reasonable period of time; they don’t extend to statistical modeling or requests that involve asking sites to get new kinds of data. At the pilot level, researchers can execute custom queries that provide a deeper look at the data.

Linkage partners will depend on the needs of a study. For example, PCORnet® Studies have linked to claims data from Centers for Medicare & Medicaid Services, registries that collect lived experience information, and commercial vendors that perform specialty lab or image testing.

An advantage of using PCORnet® for pragmatic and prospective trials is the connection with the health system, local investigators, and data experts. These can serve as valuable resources during the design, recruitment, and analysis stages.

October 21, 2025: New Podcast Episode Points Investigators to Strategies for “Avoiding the Fumble” in Pragmatic Trials

Posted on by Sophia Ramirez

In a new episode of the Rethinking Clinical Trials Podcast, Emily O’Brien of Duke University expanded on key takeaways from her recent Grand Rounds presentation, “Avoiding the Fumble: Building on a Decade of Lessons from Pragmatic Clinical Trials.”

Listen to the podcast. For alerts about new episodes, subscribe for free on Spotify, Amazon Music, Apple Podcasts, or SoundCloud.

The Grand Rounds presentation came on the heels of a big decade for pragmatic clinical trials, defined by new opportunities and challenges.

“We’ve seen a huge explosion in interest,” said O’Brien. “And that often comes some reality checks as people begin to understand what it takes to do one of these studies well across multiple sites.”

“The concept of the fumble has been an interesting way to acknowledge that we don’t always get things perfectly right the first time.”

O’Brien and her colleagues on the PCORnet® team developed “The Playbook” as a tool for sharing and refining the best approach to national-scale research. It is a pragmatic application of principles often discussed within the NIH Collaboratory, providing investigators with practical strategies for implementation.

Like the Living Textbook, the Playbook is a living document that will be updated to reflect learnings from the successes and fumbles of future trials.

“If we can establish a culture of transparency and sharing, that can accelerate the implementation of best practices and refinement of those practices,” O’Brien said.

O’Brien is an associate professor in population health sciences at Duke University. She serves as cochair of the NIH Collaboratory’s Patient-Centered Outcomes Core Working Group.

Grand Rounds August 22, 2025: Avoiding the Fumble: Building on a Decade of Lessons from Pragmatic Clinical Trials (Emily O’Brien, PhD, FAHA)

Posted on by Sophia Ramirez

Speaker

Emily O’Brien, PhD, FAHA
Associate Professor
Duke Clinical Research Institute
Duke University School of Medicine
Department of Population Health Sciences

Keywords

Pragmatic Trials; Best Practices; PCORnet; Evidence-Based Practices

Key Points

  • Historically, the healthcare industry has been limited by an insufficient body of evidence driving everyday clinical decision-making. Roughly a decade ago, pragmatic clinical trials (PCTs) began to gain traction as a promising solution.
  • There are several advantages of PCTs. They can be embedded within healthcare systems without disrupting the clinical workflow; answer questions of major public health importance; streamline procedures and infrastructure by making use of existing data; and include diverse, representative study populations for highly generalizable results.
  • But a recent analysis of clinical research site challenges noted that protocol complexity, site workload, and patient burden have increased since 2015. Though the analysis was not specific to pragmatic trials, a fundamental shift in how researchers think about study design is required across the clinical trials space.
  • Additionally, evidence-based practices – even those that have been stress-tested in PCTs – are not always adopted by health systems. Trial success does not necessarily coincide with system priorities; different audiences, i.e. systems and funders, require different kinds of evidence; and 5- to 10-year studies are misaligned with systems’ 2- to 3-year decision horizons.
  • The NIH Pragmatic Trials Collaboratory philosophy holds that fumbles are part of the game; we can’t improve if we only share wins, and transparency and teamwork has helped this community iterate and improve. Accordingly, the PCORnet team developed “The Playbook,” inspired by the NIH Collaboratory’s Living Textbook, as a tool for sharing and refining the best approach to national-scale research.
  • The Playbook contains practical “drills” for avoiding common fumbles in recruitment, workflow, and outcome capture, and was created using a user-centered design process. They engaged PCORnet groups, partners, and members of the Playbook’s intended audience to inform and guide the content.
  • Modules 1 – 5 of the Playbook, launching this year, will provide an introduction to the network. They include sections on getting started with PCORnet, utilizing the network’s resources, dissemination and implementation expectations for PCORnet studies, and case studies.
  • In the long-term, the PCORnet team plans to actively review, maintain, and expand the Playbook. Additional modules are in process and targeted for release in 2026.

Discussion Themes

The success of the Playbook may depend on the willingness of investigators to share both their “best plays” and their mistakes. Dr. O’Brien was optimistic that research teams will buy into this philosophy and acknowledge it as an important piece of the evidence generation process.

The case studies that the team selected serve to illustrate A) that PCORnet trials are unique, innovative, and approaching challenges in a thoughtful, inspiring way and B) the many ways to engage with the network.

August 20, 2025: PCORnet Playbook Featured in This Week’s Rethinking Clinical Trials Grand Rounds

Posted on by Damon Seils
Emily O'Brien
Dr. Emily O’Brien

In this Friday’s Rethinking Clinical Trials Grand Rounds, Emily O’Brien of Duke University will present “The PCORnet Playbook: Avoiding the Fumble in Pragmatic Trials.”

The Grand Rounds session will be held on Friday, August 22, 2025, at 1:00 pm eastern.

O’Brien is an associate professor in population health sciences at Duke University. She serves as cochair of the NIH Collaboratory’s Patient-Centered Outcomes Core Working Group.

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October 11, 2023: In This Friday’s PCT Grand Rounds, Incorporating Social Determinants of Health Into PCORnet

Posted on by Damon Seils
Headshot of Dr. Keith Marsolo
Keith Marsolo, PhD

In this Friday’s PCT Grand Rounds, Keith Marsolo of Duke University will present “Incorporating Social Determinants of Health Into PCORnet.”

The Grand Rounds session will be held on Friday, October 13, 2023, at 1:00 pm eastern.

Marsolo is an associate professor of population health sciences at Duke University and a cochair of the NIH Pragmatic Trials Collaboratory’s Electronic Health Records Core Working Group.

Join the online meeting.

Grand Rounds October 13, 2023: Incorporating Social Determinants of Health Into PCORnet (Keith Marsolo, PhD)

Speaker: Keith Marsolo, PhD
Associate Professor, Department of Population Health Sciences, Duke University School of Medicine

Topic: Incorporating Social Determinants of Health Into PCORnet

Date: Friday, October 13, 2023, 1:00-2:00 p.m. ET

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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.

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November 22, 2022: ADAPTABLE Points to Need for Better Integration of Patient-Reported Health Data Into Pragmatic Trials

Posted on by Damon Seils

Headshot of Dr. Emily O'BrienA concordance analysis from ADAPTABLE, a large pragmatic, comparative effectiveness trial, found low to moderate agreement between patient-reported health data and data derived from the electronic health record (EHR). The findings highlight the need for better integration of patient-reported health data into pragmatic research studies.

Results of the study were published in JAMA Cardiology.

Patient-reported health data can assist investigators in capturing clinical events in pragmatic clinical trials. However, little information is available about the fitness for use of patient-reported data in large-scale health research.

In this study, analysts evaluated the concordance of a set of variables that were both reported by patients through an online portal and available in the EHR as part of the trial. The validity of participant self-report varied by the method of data collection, the participant population, and the type of information collected. For example, patient report of clinical events had low sensitivity compared with EHR data for myocardial infarction, stroke, and major bleeding events. Coronary revascularization was the most concordant event by data source, but agreement was only moderate.

“As the use of direct-to-participant research designs grows, it’s important to understand how participant-reported data lines up with other data types,” said Emily O’Brien, an associate professor in population health sciences at Duke University and the lead author of the study.

“Our findings suggest that some caution is warranted in this space—especially if relying on participant report alone for capture of cardiovascular events—and we need more research on how to best design questions and set up participant portals to maximize validity of this type of data,” O’Brien said.

ADAPTABLE, the first major randomized comparative effectiveness trial conducted by the National Patient-Centered Clinical Research Network (PCORnet), seeks to determine the optimal dose of aspirin therapy for secondary prevention of atherosclerotic cardiovascular disease. The trial relies on both existing EHR data sources and patient-reported health data captured through an online portal.

This is the first study to assess validity of participant-reported health data collected through an online portal. The study’s results are consistent with prior evidence suggesting that the validity of participant-reported events varies by data collection modality, participant subgroup, and the type of information collected.

This work was supported by a supplemental grant award to the NIH Pragmatic Trials Collaboratory Coordinating Center from the National Center for Complementary and Integrative Health.

Read the full report in JAMA Cardiology.