News

Rare Disease Day Shines a Light on Lennox-Gastaut Syndrome Research Powered by PCORnet®

Posted on by Jodi Snare

Each year on the last day of February, Rare Disease Day raises awareness for the more than 30 million people in the U.S. living with rare diseases. In honor of Rare Disease Day, we are shining a spotlight on a PCORnet® Study addressing one such condition: Lennox-Gastaut Syndrome (LGS). LGS is a severe form of epilepsy that begins in early childhood, causing multiple types of drug-resistant seizures, cognitive impairments, and significant challenges for families seeking effective treatment options. LGS is a challenging condition to study due to its rarity and variability, but with access to insights from the everyday health encounters of 47 million people across the U.S., the PCORnet infrastructure is uniquely positioned to support this research.

Addressing the Challenges of Lennox-Gastaut Syndrome

Children and young adults with LGS may experience hundreds of seizures per week and are often unresponsive to standard treatments. Families navigating this complex disorder frequently face uncertainty about which therapies—whether additional medications or surgical interventions—will offer the best possible quality of life. The study, led by Principal Investigator Sandi Lam, MD, MBA, at Ann & Robert H. Lurie Children’s Hospital of Chicago, aims to address this question using the PCORnet infrastructure while also establishing foundational knowledge to support future research into LGS.

“One of the major challenges in studying LGS is that there is no established computable phenotype,” said Dr. Lam. “By leveraging the vast data resources available through the PCORnet infrastructure, we have an opportunity to develop a standardized way to identify LGS patients within electronic health records. This is critical because having a reliable method to identify and study LGS at scale can help researchers uncover new insights, improve diagnoses, and ultimately lead to better treatment options for these patients and their families.”

Once the computable phenotype is established after multicenter manual chart review of almost a thousand patients, the study team will use it to conduct a comparative clinical effectiveness study exploring the records of thousands of patients to determine whether medication or surgery is most likely to improve the clinical outcomes important to children with LGS and their caregivers.

PCORnet: Unique Advantages for Patient-Centered Rare Disease Research

The study involves five PCORnet® Clinical Research Networks (CRNs) working together to provide comprehensive, high-quality data:

  • Greater Plains Collaborative (GPC)
  • OneFlorida+
  • PaTH
  • PEDSnet
  • STAR

As with all PCORnet® Studies, a key component of this research is its strong patient engagement approach. In partnership with the LGS Foundation, researchers are incorporating insights from families living with LGS to ensure the study reflects what matters most to patients. One aspect of this work focuses on gathering input from patients and their caregivers on the best ways to collect patient-reported data.

“Our hope is that this study can start delivering answers that reflect the needs of the LGS community,” said Dr. Lam.

Dr. Lam and her team expect to publish study results in early 2026.

 

Are you interested in leveraging the PCORnet infrastructure to power your next rare disease research effort? With a new PCORI funding opportunity coming soon, now is the time to reach out. PCORnet is a national resource open to all, regardless of research affiliation or funding source. Visit the PCORnet® Front Door to get started.

Have You Ever Wondered What Kind of Research PCORnet® Can Power?

Posted on by Jodi Snare

Join our upcoming Best Practice Sharing Sessions to get inspired! On March 19 at 12:00 p.m. ET, we’ll explore the operational strategies and lessons learned from ACTIV-6, a PCORnet® Study.

ACTIV-6 was a decentralized platform trial that leveraged the PCORnet infrastructure to study repurposed medications for treating acute COVID-19. The study team recently shared results on medRxiv, a pre-publication server. Guest speakers Rhonda Wilder, project leader, and Martin Oyelakin, lead clinical research associate, will discuss how the study implemented innovative research approaches to accelerate discovery.

What Are Best Practice Sharing Sessions?

These monthly forums bring together researchers, clinicians and community members to exchange ideas, improve methods, and explore new ways of using the PCORnet infrastructure to advance health research. The sessions are open to the public, and we welcome anyone interested in using the PCORnet infrastructure for research.

Want to attend? Email [email protected] for an invite!

Two Recent Data Reports Show that PCORnet® May Potentially Power Impactful Research into Mental and Behavioral Health and Pediatric Populations

Posted on by Jodi Snare

Two recent data reports are providing researchers and the public with insights into the PCORnet® infrastructure. These reports offer topic-specific snapshots of the extensive, real-world patient data available through PCORnet® Clinical Research Networks (CRNs), helping research teams to refine research questions, identify relevant data elements, and design more informed, impactful studies.

Mental and Behavioral Health

The Mental and Behavioral Health Data Report demonstrates that PCORnet has the potential to support national-scale patient-centered comparative clinical effectiveness research (CER) efforts and generate real-world evidence to improve our understanding of the effectiveness of interventions for mental and behavioral health conditions.

This report presents aggregate data on patients with recorded diagnosis codes for mental and behavioral health conditions, as well as the demographic characteristics, healthcare use, and prescribing of common psychiatric medications for these patients. Data from 59 sites participating in PCORnet are included in this report, reflecting the experience of nearly 29.5 million patients.

Read the Mental and Behavioral Health Data Report.

Pediatric Population

A Pediatric Population Data Report is also available. This report demonstrates the magnitude of pediatric patients that are currently connected to sites participating in PCORnet that may be available for future research activities.

The Pediatric Population Data Report describes the sociodemographic and clinical characteristics of more than 12 million pediatric patients from birth to 20 years old who received care in 2023 at a site participating in PCORnet.

Read the Pediatric Population Data Report.

Data reports are also available for other areas of research, including maternal morbidity and mortality, telehealth, and intellectual and developmental disabilities. Most offer the largest national query of real-world data on conditions, populations, or service utilization. Read them all.

Interested in conducting national-scale research in one of the areas highlighted in the data reports? PCORnet may be used by all interested investigators, regardless of affiliation or source of funding. Contact the PCORnet® Front Door to start the conversation.

A New Year, a New Era of Patient-Centered Health Research Powered by PCORnet®

Posted on by Jodi Snare

In December 2024, the Patient-Centered Outcomes Research Institute (PCORI) approved $134 million in funding for Phase 4 of PCORnet, reaffirming its commitment to advancing patient-centered comparative clinical effectiveness research (CER). This new funding will drive innovative developments, expanding the PCORnet infrastructure to support large-scale, transformative research.

What’s Coming in 2025

The PCORnet® Playbook: A Guide to Research Success

One of the most anticipated initiatives for Phase 4 is the release of the PCORnet® Playbook, a comprehensive resource designed to help researchers make the most of the PCORnet infrastructure. Whether you are funded by PCORI or another source, this guide will provide practical tools, best practices, strategies to navigate the PCORnet infrastructure and integrate patient perspectives. The Playbook will streamline the research process and accelerate discovery, helping researchers hit the ground running. Look for it in early 2025.

Interactive Data Reports: Dynamic Insights at Your Fingertips

In 2025, PCORnet will launch interactive data reports, making data insights from participating sites more accessible and user-friendly. These reports will offer dynamic, interactive tools to explore data by health condition, demographics, and more. Researchers, policymakers, and other partners will find these new tools invaluable for refining research questions, designing impactful studies, and assessing the capabilities of the PCORnet infrastructure to support upcoming research. Expect the initial reports to be available in the first quarter of 2025.

Expanding the PCORnet Infrastructure

The PCORnet infrastructure has grown into a robust, national resource supporting hundreds of studies across designs and funders. With Phase 4 funding, PCORnet will expand further, adding new participating sites and increasing capacity to support large-scale, patient-centered research. This phase will continue to prioritize patient involvement all stages of PCORnet® Studies, ensuring real-life experiences shape every step of the process.

A Transformative Year Ahead

2025 promises to be a transformative year for patient-centered health research. With the launch of the PCORnet® Playbook, new interactive data reports, and an expanded network, PCORnet is poised to empower researchers and deliver results that improve health and change lives.

Interested in how the PCORnet infrastructure can support your patient-centered health research study? Reach out to the PCORnet® Front Door today to start the conversation and explore opportunities to collaborate.

PCORI Approves $134 Million to Bolster the Infrastructure of PCORnet®

Posted on by Jodi Snare

The Patient-Centered Outcomes Research Institute (PCORI) recently approved $134 million for a fourth phase of funding for PCORnet®. This funding will provide continued support for eight PCORnet® Clinical Research Networks (CRNs) and the Coordinating Center for PCORnet®, demonstrating PCORI’s continued commitment to strengthening patient-centered comparative clinical effectiveness research (CER) through this powerful national resource.

Funding from PCORI will be used for several initiatives to sustain, expand, and innovate the PCORnet infrastructure to enhance data quality and research capacity to support multi-site CER studies that meaningfully improve the lives of patients. Read the full media release.

‘Haven’t We Always Done This?’ Greg Merritt’s Journey to Normalize Patient Partnership in National-Scale Research

Posted on by Jodi Snare

When Greg Merritt, a Patient Partner on the PCORnet® Steering Committee, first learned about the ADAPTABLE study in 2016, he was stunned. As a heart attack survivor who had been taking aspirin since 2012, he couldn’t believe that researchers were just now launching a study of 15,000 participants to determine optimal aspirin dosage for secondary prevention of heart attacks and strokes.

“Don’t we already know this?” Greg wondered. Like many patients, he had assumed these basic questions about common treatments had long been answered through large-scale research.

The reality surprised him. Without networks like PCORnet®, conducting research at a national scale is enormously challenging. “I had no idea that enrolling 15,000 patients was a big deal,” Greg reflected. “I just assumed this was how research was always done when it came to questions that affect millions of people.”

The truth is far different. Most clinical studies involve only a few hundred participants and rarely capture America’s population. This revelation sparked something in Greg—a dedication to transforming healthcare research. He envisioned a system where large, representative studies become the norm for addressing critical health questions, with patients involved every step of the way.

From Participant to Partner
Greg joined the ADAPTABLE study as one of its Patient Partners, known as “Adaptors.” As he became more involved, he noticed a gap: patients rarely had a voice in shaping research questions or study designs. At the time, incorporating the patient voice in research was still in its early stages, but PCORnet was paving the way for a future where patients were included from the very beginning. When Greg was invited to join the PCORnet® Steering Committee in 2019, he saw a chance to address this gap head-on by using his voice to shape and improve research powered by the national-scale, patient-centered infrastructure.

The journey from participant to partner would take some time. Greg found himself thrust into complex discussions about the PCORnet® Common Data Model and study designs, most of which were still a mystery to him.

“I spent the first year just trying to get up to speed,” Greg said. “During that time, I could see that the principal investigators and Steering Committee leadership were eager for my input, but I was worried that I would derail important conversations if I asked too many questions.”

Fortunately, Greg had support from other Patient Partners like Neely Williams and Nadine Zemon, who helped him navigate this new world. “With Neely and Nadine’s help, I found my voice. Now, the three of us cause more good trouble than most on these Steering Committee meetings,” he said with a smile.

At times, that “good trouble” is pointing out an overly burdensome requirement in a protocol that investigators may not flag as a recruitment risk, but that patients understand would be a barrier to participation. Other times, it may be questioning the endpoints of a study to ensure it delivers results that are truly helpful to those the research aims to benefit.

“We are not experts in clinical research, but we are experts in what it is like to be a patient considering participation in a clinical trial—and for a research team designing a study, that expertise can be quite valuable,” he said.

That value is reflected in a deeper integration of the patient perspective in the Steering Committee since Greg joined. For example, the Steering Committee recently began setting aside dedicated time at the end of each meeting for Patient Partners to understand what was discussed at the meetings and pose questions. The number of Patient Partners on the committee has also grown from three to eight, achieving an important balance by elevating patient voices to equal standing with research investigators.

“In its first decade, the PCORnet infrastructure has made great strides in helping researchers embed the patient voice at every level,” Greg said. “The increase in Patient Partners and the dedicated time for our input are significant steps toward true collaboration, where patients and researchers work side by side to shape the future of health research.”

A Vision for the Future
As Greg and others like him look ahead to the next 10 years, they envision a future in which large-scale research is not an exception but the rule, especially for answering fundamental health questions. With established connections and insights from everyday encounters with 47 million people across the U.S. each year, the PCORnet infrastructure is poised to play a key role in making large-scale, patient-centered research a standard practice.

But for this vision to be realized, patient engagement must also continue to evolve. Although PCORnet and others have raised the bar on integrating the patient voice, there is more to be done, particularly in ensuring that engagement efforts include voices from across social and economic backgrounds. There may also be opportunities to rethink how research findings are shared. For example, Greg and other Patient Partners are advocating for methods that go beyond traditional academic journals to make results more accessible and impactful. Podcasts, toolkits for clinical practice, and community forums could all help bridge the gap between research and real-world practice. Patient Partners can be instrumental in making these innovations happen, creating pathways that keep participants engaged even after a study concludes.

“I envision a time when involving patients from the earliest planning stages through to the dissemination of research findings is so ingrained in the research process that no one questions its necessity,” he said. “In 10 years, I want people to look at a study and say, ‘Haven’t we always done this? Was there ever a time we didn’t have Patient Partners guiding us along the way?'”

Are you interested in using PCORnet to power your next research effort? Visit the PCORnet® Front Door to start the conversation.

PCORnet® Study Shows Digital Interventions May Help Reduce Early Childhood Obesity

Posted on by Jodi Snare

Results from the Greenlight Plus Study were recently published in JAMA, demonstrating that adding a digital component to pediatric counseling could effectively reduce obesity in children. Conducted across two PCORnet® Clinical Research Networks, INSIGHT and STAR, the study involved families from a variety of backgrounds.

Study Highlights and Key Findings

The JAMA publication Effectiveness of a Digital Childhood Obesity Prevention Intervention Combined with Pediatric Health Behavior Counseling describes how the study enrolled 900 parent-infant pairs across six U.S. medical centers, following them for two years. Researchers compared two approaches:

  • Standard health behavior counseling at routine pediatric visits (the Greenlight Intervention)
  • Combined counseling and tailored digital intervention (the Greenlight Plus Intervention), featuring personalized text messages and a web-based dashboard.

At the end of the 24-month period, results showed the digital intervention group showed a significant reduction in early obesity markers compared to the counseling-only group. Specifically, the children who received the digital intervention achieved:

  • Lower average weight-for-length trajectory
  • 41% lower obesity rate (7.4% vs. 12.7% in counseling only group)

The findings suggest a combined digital and clinical approach can play a role in promoting healthy growth patterns in early childhood, especially for those at higher risk of obesity due to socioeconomic factors.

PCORnet® Infrastructure Enables Success

This PCORnet® Study tapped into the extensive resources and collaborative framework provided by the PCORnet infrastructure, which played a crucial role in providing:

  • The PCORnet® Common Data Model for streamlined data harmonization
  • Single IRB process for expedited multi-site
  • Standardized, yet efficient data collection methods
  • Patient-centered implementation framework

These helped the study team successfully implement a digital health intervention at scale while addressing public health challenges.

Learn more about conducting research through PCORnet at the PCORnet® Front Door.

Leverage PCORnet® to Advance Pain Research with New PCORI Funding Opportunity

Posted on by Jodi Snare

A PCORI funding opportunity is opening new pathways to solutions for the millions of people in the U.S. whose quality of life is limited by pain. With up to $12 million in direct costs available for patient-centered studies addressing acute and chronic pain, this funding initiative presents an opportunity for researchers to make a real difference—and PCORnet® is perfectly positioned to support these efforts.

PCORnet offers a robust infrastructure for conducting national-scale, comparative clinical effectiveness research into the experiences of more than 47 million people in the U.S. When it comes to pain research, a national scale is key. By studying large, varied groups, researchers can identify patterns, understand unique pain trajectories, and develop tailored interventions that are effective for a wide range of patients, reflecting the complexity of pain itself. With its ability to connect researchers to patient populations and real-world data, PCORnet was built to support studies that not only advance pain management but also tailor solutions to patients' specific needs, driving more effective interventions.

CaRISMA: A Case Study in Pain Research with PCORnet

To understand how PCORnet can be used to advance pain research, consider CaRISMA, a PCORnet® Study with early findings around the impact of depressive symptoms on chronic pain outcomes in people living with sickle cell disease. Many sickle cell disease patients experience severe chronic pain crises, often coupled with higher rates of depression and anxiety. Using the extensive data resources and patient recruitment capabilities available via PCORnet, the CaRISMA study team enrolled 357 adults with sickle cell disease to understand the relationship between mental health and pain.

“Using the PCORnet infrastructure, we have been able to overcome many of the typical challenges that come with research into pain caused by sickle cell disease, including the geographic spread of patients and the complex and varied nature of their pain,” said Charles Jonassaint, CaRISMA principal investigator.

To recruit participants and bring the pragmatic CaRISMA trial to life, the study team worked with PaTH, a PCORnet® Clinical Research Network. Through this partnership, the study team learned the sickle cell disease community preferred digital communication versus other formats. With this information in mind, the team developed a smartphone app to deliver digital cognitive behavioral therapy (CBT) to half of the study participants. The other half of participants received educational content on sickle cell disease via a smartphone app. The study team is exploring whether either approach impacted daily pain. Early findings were published in the British Journal of Hematology in January 2024.

“Education or CBT may not eliminate pain altogether, but if we can use one or both of these approaches to manage the pain in ways that meaningfully improve patients’ quality of life, that is an important win for the pain community,” said Jonassaint.

Get Started with PCORnet® for Pain Research

With this new PCORI funding opportunity, researchers have the chance to similarly explore new avenues in pain research using PCORnet. Here are three steps you can take today to get started:

  1. Visit the PCORnet® Front Door to learn how you can use PCORnet to support your pain research.
  2. Register for one or both of our upcoming information sessions:
  3. Sign up for a one-on-one Office Hours session with PCORI staff to answer your questions specific to this funding announcement.

This and other PCORI funding opportunities will open on Dec. 3, 2024, so start making your plans now to apply. Check out the full PCORI Managing Pain Topical Funding Announcement for more information.

New Paper Explores the Power of PCORnet® to Enhance Health Surveillance

Posted on by Jodi Snare

A new paper published in the journal Preventing Chronic Disease highlights the pivotal role of PCORnet® in transforming national health surveillance.

Surveillance, which involves collecting and analyzing health data from various sources to track the spread of diseases, monitor health trends, and detect any new health threats, is traditionally achieved through surveys and public health reports. While these provide valuable information on the epidemiology of chronic and infectious diseases, the increasing availability of electronic health records, or EHRs, brings opportunities to fill information gaps that national surveillance systems do not capture (e.g., patient-level geographic information, longitudinal data, etc.). This manuscript explores the promise of PCORnet to help researchers efficiently glean these kinds of insights from EHRs on a national scale to improve surveillance.

Key Highlights from the Manuscript

  • Leveraging PCORnet fills gaps in existing surveillance data: PCORnet offers unparalleled access to secure, deidentified patient-level geographic information on a broad scale, as well as comprehensive longitudinal clinical data generally not available through existing national surveillance systems. PCORnet can also offer a granular view of geographic data units, like ZIP codes and census tracts, to support more reliable surveillance data.
  • Pilot projects demonstrate value: Pilot surveillance projects within PCORnet have highlighted the robust infrastructure, which was instrumental during the national COVID-19 public health response. These projects offered critical lessons in utilizing PCORnet for efficient surveillance.
  • PCORnet supports faster surveillance: During fast-moving health crises, answers can’t Researchers can use PCORnet to glean rapidly refreshed data with short latency, making the infrastructure useful for tracking infectious disease outbreaks and chronic disease trends.

The use of patient-centered distributed research networks like PCORnet has the potential to meaningfully enhance health surveillance research. With extensive data resources, streamlined processes, and successful pilot projects aimed at meaningfully improving the lives of patients, PCORnet has proven itself an invaluable resource for advancing public health.

Read the full manuscript.

PCORnet Leaders Highlight Transformative Research at ARM 2024: Five Key Takeaways

Posted on by Heather Wilson

The AcademyHealth Annual Research Meeting (ARM) 2024 offered the opportunity to share some of the unique features of PCORnet®, with leaders highlighting how the multi-stakeholder ecosystem has enhanced researchers’ capacity to conduct national-scale comparative clinical effectiveness research (CER). The panel session, “Multi-Stakeholder Engagement and Collaboration: What We’ve Learned from Patient-Centered Research Using PCORnet,” brought panelists together to discuss how the PCORnet infrastructure works. It also shed light, through real-world examples, on some of the ways use of PCORnet is speeding patient-centered insights that improve clinical care.

Held at the Baltimore Convention Center on June 30, the session featured a panel of experts: Dr. Erin Holve, Chief of Research Infrastructure at the Patient-Centered Outcomes Research Institute; Al Richmond, executive director of Community-Campus Partnerships for Health; Dr. Kathleen McTigue, principal investigator for PaTH, a PCORnet® Clinical Research Network; and Dr. Tom Carton, chair of the PCORnet® Steering Committee.

Five Key Takeaways

  1. Visit the PCORnet® Front Door: PCORnet can support a range of study designs, including data-only projects, observational research, and clinical trials, and the panel emphasized that all collaboration with PCORnet resources starts at the PCORnet® Front Door.
  2. PCORnet resources can address significant research questions: PCORnet® Network Partners are primed and equipped to tackle the “big audacious questions” that often require incredible resources and infrastructure, such as comparing clinic-based and community-based peer support for individuals with intellectual and developmental disabilities (IDD) and evaluating melatonin versus behavioral therapy for sleep. This point is underscored by public query reports highlighting how PCORnet data resources could be used to support three priority research areas: IDD, telehealth, and maternal morbidity and mortality.
  3. Community organizations can benefit from PCORnet resources: Community organizations can leverage PCORnet to address their specific research needs. Notable examples given by the panel include collaborations with rare disease communities. Case studies explored by the panel highlight the practical ways in which investigators can connect with and leverage PCORnet resources to enhance their research efforts.
  4. The collaborative nature of the PCORnet infrastructure is a differentiator: The PCORnet governance structure is designed to facilitate extensive support through its resources. When utilizing PCORnet, researchers gain access to a wealth of expertise across various therapeutic areas. They also benefit from the insights provided by PCORnet® Network Partners, who possess in-depth knowledge of how their site’s data can best support research endeavors. Panelists emphasized the critical role of patient engagement in research powered by PCORnet®, using studies like the PCORnet® Bariatric Study and ACTIV-6 as examples.
  5. Real-world success stories continue to prove the value of PCORnet: The value of PCORnet continues to be demonstrated through a wide array of real-world success stories. PCORnet® Network Partners have effectively addressed a wide range of health services research and public health questions, showcasing versatility and impact. Projects like BP Control Lab, the U.S. Centers for Disease Control and Prevention (CDC) COVID-19 collaboration, the RECOVER initiative, and the CHI-RON project, highlight the capacity of PCORnet to support large-scale research on emerging and chronic health issues, as well as opportunities for rare disease research and engagement across all communities.

Overall, ARM 2024 was a success that demonstrated the potential of PCORnet to transform CER through robust stakeholder engagement and collaboration. By highlighting real-world examples and providing practical guidance, presenters empowered health services researchers to harness PCORnet resources for impactful patient-centered research. Would you like to engage PCORnet resources? Start by visiting the PCORnet® Front Door.