News

PCORnet®-Supported PREVENTABLE Study Achieves 2K Participant Milestone Leveraging Pragmatic Lessons from the Network

Posted on by Jodi Snare

A pragmatic study of the effectiveness of statins in adults aged 75 or older without known cardiovascular disease has successfully randomized the first 2,000 participants in partnership with PCORnet®, the National Patient-Centered Clinical Research Network. The study, known as Pragmatic Evaluation of Events and Benefits of Lipid-Lowering in Older Adults (PREVENTABLE), builds on past Network success in pragmatic approaches for clinical study design, including the highly lauded ADAPTABLE study of aspirin dosing in 15,000 patients with heart disease.

“Few studies are focused exclusively on participants aged 75 or older, in part because recruitment in this population is historically challenging,” said Schuyler Jones, who is helping lead the recruitment core for the study. “But insights into this population are also critically important—these are our friends, neighbors, parents and grandparents, and answers to research questions are central to their quality of life. Tailoring a trial to meet their needs is key, and as a large, nationally representative network with proven expertise in pragmatic trials, PCORnet is a huge asset in helping us achieve that goal.”

Bringing the ADAPTABLE approach to a harder-to-recruit patient population

The PREVENTABLE study is important because nearly half of Americans aged 75 or older take statins to reduce risk of cardiovascular events, but we don’t yet know if statins are helpful for older adults without heart disease. To find out, PREVENTABLE will enroll 20,000 participants, whom the team is engaging through a similar pragmatic design as the PCORnet-enabled ADAPTABLE study. Specific approaches carried forward from ADAPTABLE to make participation easier include:

  • Embedding research in the health care system by enrolling patients in their usual care settings and in partnership with their primary care clinicians;
  • Engaging potential participants during screening and recruitment by using informational videos, panel discussions with research participants, and an e-consent platform; and
  • Pairing electronic health record data with other forms of follow-up, including calls and in-person visits for cognitive and functional assessments, to ensure complete collection of outcomes.

However, several aspects of PREVENTABLE make recruitment more challenging than ADAPTABLE. For instance, while ADAPTABLE looked at patients with known heart disease, PREVENTABLE participants do not have known heart disease and thus have fewer medical visits to facilitate recruitment. Because they are not ill, they may also be less motivated to join a study. Finally, PREVENTABLE’s recruitment effort is in the midst of the COVID-19 pandemic, which has disproportionately affected older adults and perhaps made them hesitant regarding trial participation.

“I have been proud of the study team’s efforts to pivot in response to these challenges and find novel ways to engage patients, whether that is in the clinic, in community centers, or on the pickleball court,” said Jones. “Slowly, but surely, we are bringing more participants into the fold, and we are tasked with enrolling our first 5,000 patients by August 2022. At the end of this study, we will have amassed a huge amount of knowledge around what it takes to recruit older participants for research, which will support future efforts to seek insights into this important, but vastly understudied population.”

Data Privacy statement for PCORnet® released, detailing how the Network protects patient information

Posted on by Jodi Snare

Network Partners with PCORnet®, the National Patient-Centered Clinical Research Network, have released a statement explaining how the Network handles sensitive data and protects patient privacy. The statement is yet another way that the PCORnet community continues to demonstrate its commitment to deliver on the founding values of patient centricity and trust.

“The PCORnet infrastructure was designed to facilitate research that is uncompromising in its patient centricity, and there is no question that patients have a right to know how we are safeguarding their privacy,” said Keith Marsolo, PhD, a co-investigator in the Distributed Research Network Operations Center of the Coordinating Center for PCORnet.  “We have always had stringent protections in place, and now our data privacy statement takes the additional important step of sharing with patients exactly how their personal data is handled with care.”

Shedding light on common questions

The PCORnet data privacy statement is comprehensive, detailing the Network’s protective infrastructure, how patient information is collected and securely shared, and how the data is accessed and used for research. It also clears up some common misconceptions about PCORnet-enabled data. For example, a key security feature of the PCORnet infrastructure is that the data actually stay under the care of the participating healthcare institution and are not amassed into a single data pool or data warehouse.  Data are only accessed within the context of specific research projects, and the participating institutions decide whether or not to share the minimally necessary data needed for research on a case by case basis.

Importantly, the statement was drafted and approved as a collaborative effort by PCORnet Network Partners and written to be easily understandable by audiences despite its technical nature. “The commitment PCORnet places on data privacy is one of the many ways PCORnet exemplifies its commitment to patients. I take a great deal of pride in knowing that PCORnet is steadfastly committed to keeping patients, their goals, and their privacy at the heart of the mission. By involving partners, such as myself, in processes like the development of the data privacy statement, blind spots are alleviated and true collaboration is formed,” said PCORnet Patient Partner, Susan Lowe. PCORnet patient partners who participate in the governance of the Network reviewed the statement for clarity, and they –along with the rest of the PCORnet Steering Committee – voted to adopt the statement.

“We believe that this statement will serve as a win-win for both patients and researchers,” said Marsolo. “When patients are given full transparency as to how their personal information is handled with care, trust is built. With that trust, we can continue to build on and use the PCORnet infrastructure to deliver fast, trustworthy answers that deliver better health outcomes for all.”

Solid Data, Expert Vetted, Patient-Centered: What PCORnet® Designation Tells You About a Study

Posted on by Jodi Snare

Since its launch, PCORnet®, the National Patient-Centered Clinical Research Network, has become synonymous with impactful, pragmatic research conducted at national scale. To support more quality, patient-centered research, Network leadership worked in close partnership with the Patient-Centered Outcomes Research Institute (PCORI) to launch the PCORnet Study Designation program.

A study designated as a PCORnet study is one that meets criteria demonstrating commitment to conducting important patient-centered research differently, and that, in turn, benefits from the full support of PCORnet and its continued growth as a learning health network. Achieving PCORnet Study Designation is an accomplishment that research teams can use when promoting their studies and results.

“Study designation is one way that we support research efforts that are designed specifically in service to the patient, from engagement in the design and conduct of studies to dissemination of results,” said Russell Rothman, MD, the Chair of the PCORnet Steering Committee.  “If we can help those studies succeed by offering the knowledge and lessons learned from the studies and experts in the Network, we can help ensure research that matters to patients continues to progress—and, at the end of the day, that’s what the Network is all about.”

Some of the most consequential studies conducted through PCORnet carry Study Designation, including ADAPTABLE, HERO, PREVENTABLE, MedWeight and more. To qualify for Study Designation, a research project must:

  • Include two or more PCORnet Clinical Research Networks, or “CRNs”
  • Ensure that participants and patients are engaged throughout the project lifecycle
  • Are committed to broad dissemination of findings and returning study results to participants
  • Agree to share study progress, performance metrics, and best practices with the Network regularly
  • Leverage the PCORnet Common Data Model as appropriate

The Designation Process

Requests for Study Designation are submitted to the PCORnet Front Door for administrative review and intake, and then are reviewed by impartial volunteers from across the Network. Reviewers can approve, deny, or request clarification, and typically a decision is reached within two weeks of submission. Once approved, the Network is deeply invested in the quality and success of these efforts. That’s why Designated Studies enjoy a robust level of support across the Network through regular communication with experts at PCORnet CRNs and the PCORnet Coordinating Center. Designated studies are also required to share monthly metrics for start-up, enrollment, safety and more, which embeds transparency and accountability into the process.

To learn more, check out our Study Designation Fact Sheet.

Can Existing Medications Help Treat People with COVID-19? PCORnet® Will Help Researchers Find Out.

Posted on by Jodi Snare

A new COVID-19 study supported by the National Institutes of Health (NIH) as part of its Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV) program will use PCORnet®, the National Patient-Centered Clinical Research Network, to quickly access enrolling sites, streamline study start-up timelines, and engage the community perspective. The ACTIV-6 study will work collaboratively with participants to develop a platform that will generate evidence about a pressing question in the pandemic: Can medications that are approved for other indications also be helpful in treating patients with mild or moderate COVID-19?

There is currently no approved medication that can be self-administered to ease symptoms of people suffering from mild disease at home and reduce the chance of their needing hospitalization. At the end of the study, the ACTIV-6 team hopes its ongoing evidence-generating clinical trial platform will shed light on the utility of up to seven medications that are already approved for other indications in alleviating the symptoms and clinical outcomes of people with COVID-19.

Putting the needs of everyday people front and center
“There is a lot of research devoted to treatment of severe COVID-19, but it is important to remember most people with COVID-19 are recovering from home, and they are looking for answers on how to feel better,” said Kris Anderberg, the project lead for the ACTIV-6 stakeholder committee. “We formed our ACTIV-6 stakeholder committee to ensure we are appropriately listening to the voices of everyday patients and focusing our research in a way that will make results meaningful to the broader community.”

Impressive progress has been made by vaccines to help curb the pandemic, but new variants and surges of infections in different regions are reminders that COVID-19 remains an evolving threat. To speed insights, ACTIV-6 will be conducted as a platform trial, meaning that it will study multiple medications concurrently, and will be conducted remotely, both key characteristics that will enable more pragmatic and efficient research. In addition, medications will be administered orally or by inhaler and will be easy for participants to take at home. Participants will be assigned randomly to receive either a placebo or one of the treatments, which will be sent to them by mail.

“The medications under consideration already have been tested in humans, so they provide an opportunity to deliver meaningful insights sooner on COVID-19 home treatment options,” said Anderberg. “We are excited about developing this platform as a meaningful tool to inform treatment of those affected by the pandemic.”

Inaugural PCORnet-Powered Study Delivers Insights and Inspiration for Novel Trial Design

Posted on by Jodi Snare

In a milestone moment for PCORnet®, the National Patient-Centered Clinical Research Network, the ADAPTABLE study team today shared results in a late-breaking clinical trials session during the 70th annual American College of Cardiology Scientific Session. The results, which were simultaneously published in the New England Journal of Medicine, establish PCORnet as a powerful resource for successfully conducting novel pragmatic clinical trials. They also provide important insights into optimal aspirin dosing and set a blueprint for the future of pragmatic research.

“As the first randomized controlled study using PCORnet, ADAPTABLE has been closely watched by researchers who are interested in the potential of large-scale, pragmatic, patient-centered trials to deliver fast, high-quality answers,” said ADAPTABLE Principal Investigator Schuyler Jones. “Today’s results leave no doubt that the novel research framework supported by PCORnet is not only possible, but necessary for the future of clinical research.”

ADAPTABLE compared two doses of aspirin (81 mg vs. 325 mg) to determine which dose was the safest and most effective for preventing a heart attack, stroke, or death in people diagnosed with existing cardiovascular disease. Ultimately, investigators found no significant difference in either protective effects or risk for bleeding between the two doses. However, the group taking the higher dose switched doses more frequently (41.6 percent of participants taking 325 mg switched doses, as compared to 7 percent who switched from the 81 mg group). Participants in the higher dose group were also more likely to discontinue aspirin completely (11 percent discontinued in the 325 mg group, as compared to 7 percent in the 81 mg group).

Inspiring a new research paradigm

While these answers are helpful for people with heart disease and providers, perhaps even more meaningful is the pragmatic and patient-inspired framework their work established. PCORnet is at the center of that framework, enabling access to data from participants’ everyday health care encounters via a distributed research network model. In collaboration with 40 health centers, one health plan, and numerous patient partners from PCORnet, the ADAPTABLE study team enrolled over 15,000 people to participate in the study.

Via PCORnet, the team leveraged electronic health records (EHRs) to identify those potential participants, and then followed up with traditional outreach such as phone calls, letters, emails, and conversations in the clinic. Data from PCORnet-accessible EHRs, patient-reported outcomes, insurance, and Medicare claims data were used to capture primary endpoints for the study where possible in lieu of in-person visits, minimizing participant burden.

ADAPTABLE patient partners, known as Adaptors, worked alongside researchers in all aspects of the trial, from helping to design the protocol, consent form, study portal, and study materials through the current dissemination of results. “By representing and sharing the patient voice at every touchpoint of the study, the Adaptors asked researchers to think about how research could be done differently,” said Greg Merritt, an ADAPTABLE patient partner. “I think if patients have more opportunities to share their lived experiences, we can shape the future of research.”

“Traditional research methods are often slow and cumbersome, so when we launched ADAPTABLE our strategy was to use PCORnet to pave a new pathway by enthusiastically embracing novel strategies that make research participation easier,” said Jones. “Since that time, the pandemic has only intensified the urgency for better, faster methods to address common clinical decisions. With the successful completion of ADAPTABLE, we now have a blueprint to meet that need.”

For more information on ADAPTABLE’s results, visit the website to read a summary of results and watch a Town Hall where Adaptors lead a discussion about ADAPTABLE results and lessons learned.

ADAPTABLE, a PCORnet® Demonstration Project, Offers a Template for Future Pragmatic Studies

Posted on by Jodi Snare

Two manuscripts recently published in peer-reviewed journals offer researchers a glimpse into the opportunities and challenges for novel pragmatic trials like Aspirin Dosing: A Patient-Centric Trial Assessing Benefits and Long-term Effectiveness (ADAPTABLE), a demonstration project of PCORnet®, the National Patient-Centered Clinical Research Network. The publications highlight separate, but equally important, topics of interest in pragmatic research: maximizing clinician engagement and navigating institutional review board (IRB) challenges.

ADAPTABLE, a multicenter, open-label, randomized controlled trial, is the first interventional study conducted across PCORnet. The ADAPTABLE study team is using PCORnet’s broad and rich data resources to examine high-dose versus low-dose aspirin for prevention of heart attack and stroke among patients with cardiovascular disease.

One manuscript published in Clinical Trials shares how ADAPTABLE has used new approaches to break down traditional barriers to clinician engagement, such as time limitations, insufficient research infrastructure, or lack of research training. While clinician engagement is important in all clinical research, it is particularly essential for pragmatic trials, which are conducted in real-world clinical settings and require that researchers and clinicians operate toward shared goals. The paper assesses how tactics like empowering clinician champions, periodic newsletters, and coordinated team celebrations fared in regard to their merit and utility in a pragmatic trial setting.

Novel pragmatic methods like those deployed in ADAPTABLE are intended to improve the participant experience, but unfamiliar approaches can also spark new — and justified — considerations for IRBs that can potentially make the evaluation of these studies more complex. A recent manuscript published in Trials explores unforeseen challenges the ADAPTABLE team encountered regarding centralized IRB evaluation, electronic informed consent, patient engagement, and risk determination, as well as how the study team pivoted in response. It offers lessons in upfront planning to mitigate these challenges in future pragmatic research.

Together, manuscripts like these are providing the research community a valuable template for pragmatic studies, which are important to help us understand how interventions perform in the real world. Full results from ADAPTABLE are expected in May 2021. Be on the lookout for more PCORnet-enabled research in the coming months!

CDC Uses PCORnet® Resources to Shape National Understanding of COVID-19

Posted on by Jodi Snare

Network Partners of PCORnet®, the National Patient-Centered Clinical Research Network, are leveraging their rich data to help answer important questions during the COVID-19 pandemic. One of the early collaborators with this work is the U.S. Centers for Disease Control and Prevention (CDC) in partnership with the Public Health Informatics Institute (PHII), a program of the Task Force for Global Health. Through this partnership (formally established in October 2020), PHII contracted with Harvard Pilgrim Health Care, a component of the PCORnet Coordinating Center responsible for developing and executing queries. Harvard Pilgrim Health Care is working with the other component of the PCORnet Coordinating Center and REACHnet to implement descriptive and advanced analytic queries across 43 PCORnet sites.

The CDC collaboration has been made possible through the efforts of PCORnet partners to rapidly adapt the PCORnet Common Data Model that captures and standardizes biweekly updates of patients with a diagnosis of COVID-19, a SARS-CoV-2 test, or other respiratory conditions. To date, aggregate data stripped of any personal identifying information related to nearly half a million patients with a COVID-19 diagnosis or SARS-CoV-2 test result have been shared with CDC.

“As a massive research network that can capture information from many healthcare systems, PCORnet has had an important role during a pandemic response,” said Tom Carton, current chair of the PCORnet Steering Committee. “The health records that underpin the PCORnet data infrastructure offer much more depth than the simple case reports with limited information that have been largely available to date. Via PCORnet, CDC and others are learning detailed surveillance information about the patterns of infection and course of the virus that is essential to help our leaders, institutions, and patients understand and combat this pandemic.”

“With our queries, we are able to capture information on patients with COVID-19 through diagnostic codes as well as lab test results,” said Jason Block, who is supporting efforts of the PCORnet Coordinating Center to better define COVID-19 using data accessible from the Network. “As a result, we are able to look at a large, representative sample of patients and get to the bottom of the characteristics of people who are getting infected and seeking care for this virus, predictors of who develops severe disease or complications, and whether there are long-term consequences of COVID-19 infection.”

“Our most recent queries are highlighting some interesting trends across COVID-19-postive patients,” said Block. “For example, we are seeing substantial changes in the use of medications for treatment of COVID-19. In hospitals, nearly all patients received hydroxychloroquine in the early phase of the pandemic; very few are receiving that treatment now, replaced by common use of remdesevir and dexamethasone. It will be important to understand these trends as we continue to pursue vaccine adoption across the U.S.”

Block and his colleagues have begun discussions with CDC about potential options to track uptake of vaccines across the Network.

Networks like PCORnet protect patient data by keeping it safe within healthcare organizations and only sending de-identified, aggregated information from patient records through secure means to inform public health action. CDC continues to work with PCORnet leaders to determine how to best leverage data from a large network of healthcare organizations to support CDC’s COVID-19 response.

“At a time when the entire world is seeking answers to combat this pandemic, PCORnet’s Network Partners have rallied to collaborate with CDC as part of the solution,” said Pastor Bruce Hanson, a patient advocate serving on the PCORnet CDC COVID-19 Workgroup. “We are optimistic that we will glean many meaningful insights in the coming weeks and months.”

The team is working on disseminating those insights more broadly across healthcare and public health communities. Information on the type of data the team is gathering is currently shared publicly on GitHub. Stay tuned for more on the PCORnet-enabled COVID-19 response.

PCORnet is Delivering Important COVID-19 Answers

Posted on by Jodi Snare

With access to coordinated heath data from more than 70 million people across the United States, PCORnet®, the National Patient-Centered Clinical Research Network, is a key resource in the fight against COVID-19. To maximize its utility, the Patient-Centered Outcomes Research Institute (PCORI) offered enhancement funding for nine existing PCORnet-enabled research studies. The idea behind these enhancement awards is to leverage existing infrastructure from PCORI-funded research to more efficiently expand understanding of COVID-19 and address this public health crisis.

One of many lessons learned from the COVID-19 pandemic is that good public health decisions in times of crisis require fast analysis of patient data. While many healthcare organizations across the U.S. have the capacity to use their electronic health records (EHRs) and claims data as tools for horizon-scanning and disease surveillance, few offer the infrastructure to support the large-scale integration needed in the pandemic. The coordinated, interoperable infrastructure of PCORnet supports that needed speed and efficiency.

Following are a few snapshots of how PCORI-funded enhancement awards are supporting the use of PCORnet to combat the pandemic:

For more information about PCORI’s enhancement awards for COVID-19 research, check out the PCORI funding website.

PCORnet®-Enabled HERO Registry Tops 20K Participants as Pfizer Partners for Vaccine Safety Study

Posted on by Jodi Snare

The Healthcare Worker Exposure Response & Outcomes (HERO) Registry, which unites healthcare workers to better understand and stop COVID-19, has enrolled over 22,000 participants. HERO is funded by the Patient-Centered Outcomes Research Institute (PCORI), coordinated by the Duke Clinical Research Institute (DCRI), and enabled by PCORnet®, the National Patient-Centered Clinical Research Network. Its goal is to use the experiences of frontline health workers to develop fast knowledge to keep these workers and their communities safe and healthy in the COVID-19 pandemic.

"We are thrilled to see the HERO Registry continue to grow, a clear indication that frontline health workers across the nation are united in their commitment to fight COVID-19, both in and out of the hospital setting," said Emily O’Brien, principal investigator for the HERO Registry. "Their experiences are tremendously valuable in helping the research community better understand the physical and mental toll of this virus, which will improve COVID-19 care across the board."

The partnership with PCORnet Network Partners has been central to the registry’s success. Network Partners have ties to a well-established community of healthcare systems with experience collaborating on large clinical studies engaging patients and clinicians alike. This community’s support ensures that any interested healthcare or emergency worker can easily join the registry.

Targeting the next phase of COVID-19: long-term vaccine safety

An advantage of real-world registries is their ability to evolve naturally as the pandemic progresses. For example, now that the U.S. Food and Drug Administration (FDA) has issued Emergency Use Authorization of COVID-19 vaccines, the HERO Registry is serving as a key tool in understanding the real-world experiences of healthcare workers taking vaccines.

In December, Pfizer funded the HERO-Together study, which is using the HERO Registry to follow healthcare workers for two years to assess their experiences after receiving one of the COVID-19 vaccines such as the Pfizer-BioNTech COVID-19 vaccine. Like previous studies that leveraged the HERO Registry, HERO-TOGETHER is making participation easy, allowing vaccinated health workers to quickly and securely provide information about their health and any unexpected medical care they have received.

What’s next for the HERO Registry?

The HERO Registry will continue to evolve in the coming years, and its community of engaged healthcare workers are certain to help answer more important questions about the short- and long-term impacts of COVID-19 within this essential community.

Patient Reps Power PCORnet’s Steering Committee: A Q&A With Nadine Zemon

Posted on by Jodi Snare

Nadine Zemon can be described as a natural explorer, someone who has sought out new insights and experiences throughout her life. She now brings that relentless curiosity to PCORnet in her role as the newest patient representative on the PCORnet Steering Committee. Never one to be boxed into a single interest area, Nadine spent her younger years exploring dual passions: art and medicine. She has worked as a pediatric emergency room nurse, managed an employee health unit and, with an interest in integrative medicine, practiced massage therapy that incorporated guided imagery techniques of psychosynthesis. Her longtime interest in art led to an appreciation for the value of the arts in medicine and to becoming a graphic designer later in life. Now, Nadine is using her voice on the Committee to infuse the patient perspective into PCORnet-enabled work. Here’s her perspective on this role and why it matters:

What sparked your interest in clinical research?

My mother died of rheumatic heart disease when I was a teenager, which was made all the more tragic because of the fact that she suffered greatly due to a lack of available treatments. As time went on and cardiac care evolved, I marveled at how much more we know now versus then. It made a strong imprint on me about the power of clinical research. My mother’s quality of life could have been completely different if she had experienced her disease today as opposed to 50 years ago. How much more can we help patients 50 years from today?

How did you get involved in clinical research?

I heard about patient-centered research from a friend who was forming the first Citizen Scientists group at the University of Florida. I learned the program was recruiting patients to be involved with the Translational Science program, and I decided to sign up. At the time, the concept of patients having a seat at the research table was so novel, and yet it also made so much sense. I was excited to take part.

What excites you about PCORnet?

The whole idea of patient-centered research excites me. This notion that we should have symbiotic relationships between patients, providers, and researchers feels like it could be a pathway to a whole new era of therapeutic development—one that transforms the “old school” way of conducting research in the best way possible.

How has your role changed as a patient representative in clinical research?

When I first started participating in research, I often doubted my instincts. For example, I might think I was the only one who didn’t understand something, so I wouldn’t speak up. Over time, I’ve learned that if I don’t understand it, most other patients probably won’t either. My voice has become much stronger, and as part of the PCORnet Steering Committee, the patient representatives have actually formalized the process of identifying confusing topics and presenting them to the other Committee members. After each weekly Steering Committee meeting, the patient representatives meet to list areas where we—and the patient community at large—will need more clarity. The Committee is always receptive to this feedback. It is a big part of the value we bring and why we are here!

How do you see the PCORnet Steering Committee evolving in the coming years? 

Now that we patient representatives have found our footing on the Steering Committee, we want to find better ways to communicate with other current or prospective patient reps. We are launching a pilot project to connect with patient partners at Clinical Research Networks to open the communication channels. We want to know their thoughts on PCORnet and the Steering Committee, and we also want to share ways they can get involved in research. Our hope is that by starting these conversations, we can engage with new ideas and maybe even inspire the influential patient representatives of the future.

What would you like other patients looking to engage in research to know? 

We need your voice in research, and there are so many ways to get involved. Also, you don’t have to have a medical degree to be an effective patient representative; you just have to be willing to share you perspective toward the betterment of research.