Even on a tough day, it’s hard not to smile while chatting with patient representative Neely Williams. Maybe it’s the passion she brings to patient-centered research as a community partner consultant at the National Institutes of Health and the patient co-principal investigator for the PCORnet Bariatric Study. Maybe it’s her knack for talking about research in a frank, down-to-earth manner that makes it accessible and unintimidating to lay communities. At any rate, Neely is undeniably a strong and positive force in the PCORnet community, and now that she’s joined the PCORnet Steering Committee as one of three patient representatives, her light shines even brighter. Here is her perspective on this role and why it matters:

How did you get involved in clinical research?

I began collaborating with PCORnet’s network partners in the course of doing my ministry work. My background is as a minister, a community advocate, and a community organizer, so I am always looking for new ways to bring communities together and improve them. Clinical research seemed like an enterprise that needed a better bridge to communities. As an African-American woman living in the Deep South who has battled obesity for most of my adult life, I have personally experienced the frustrations of being part of a community seeking answers to an under-researched disease. I saw the problem across my community too—when people talked about research, it was always what they didn’t trust about it. PCORnet’s collaborators, inspired by the Network’s initial funder PCORI, were trailblazing a patient-centered research model to resolve that disconnect, and I was excited to join the cause and share my voice. If you know me, you know I don’t do things halfway, so it wasn’t too long before I was serving as a co-investigator and member of the stakeholder advisory board for the STAR-CRN (formerly Mid-South), a PCORnet partner network. 

How do you view the changing role of patients in clinical research?

Not so long ago, the notion of patient engagement essentially amounted to what I call “nodding head” involvement. Patients were offered a gift card to listen to a protocol, so investigators could tick off a “patient involvement” checkbox. Researchers weren’t too interested in getting real input, and patients just wanted to get their gift card and go home. Neither saw the value.

Today, something different is happening. Patients have pulled back the blinds of clinical research, and they realize they have a lott to contribute, that they can be changemakers in this research process; they want to be empowered in their health. The PCORnet community and others have recognized the value of the community voice in research and opened doors to let these voices in. As a result, the system is changing.

We know now that research is never going to be effective and meaningful when it is conducted in a silo without real collaboration. We know we need patients and other community stakeholders as a balancing force. Once we have all the voices at the table, we can start building protocols that will not only be effectively executed, but also will deliver meaningful insights that matter to patients and their communities. This is how we build trust and improve patient care, which is, at the end of the day, what both communities and researchers want to see.

What contributions have you made as a patient representative?

We meet once a week to review protocols and brainstorm ideas for building better research or sharing outcomes. We’ve made a lot of progress within PCORnet at integrating the patient voice in studies.

Studies that use PCORnet resources are patient-centered as a rule, but the larger clinical research community would benefit from more patient input as well. One area where I feel I can add value as a patient representative is around ensuring that the engagement efforts being built into a study are strong, realistic, and executable. I remember looking at one study proposal and asking the other members of the Committee where the budget line item for engagement was. The study had great ideas for engaging patients, but I couldn’t see how they would be funded. The response from the author of the protocol was that the engagement budget was built into several other areas and they would pull funds from those areas as needed. I said, “Tell me something. Would you feel okay about baking a cake and not adding in the salt because it’s already included in the butter? Of course not! Patient engagement needs a line item in the budget or it won’t happen.” That got a laugh, but they understood my point and built it in!

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

Right now, three out of 11 Steering Committee members are patient representatives, and I think that’s a good balance. However, we need to keep bringing in fresh insights, so I envision those members changing. We are working now to cultivate other patient representatives who will serve. There is a great energy at the grassroots level of the research process in places like STAR and other PCORnet partner networks. Many people would love to be in this role, but may not know how to begin that process. Kiely [Law], Henry [Cruz], and I were the first to open these doors to the Committee, and now it is our job to mentor and hold open that door to make room for others to come in, too.

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. Not everyone wants to be invested at the level I am, and that’s okay! Any bridge between communities and researchers is a worthwhile investment, even if it is just responding to a five-minute survey. If the research community has a responsibility to follow PCORnet’s lead and open the door to patients and caregivers, then we, likewise, have a responsibility to walk through it.

This is part two of an ongoing series highlighting patient partners on PCORnet’s Steering Committee. Check out this interview  with Henry Cruz. Watch this video of Neely Williams and PCORnet coordinating center principal investigator Adrian Hernandez discussing how the engagement of patients has contributed to the network’s initiatives.

A recent JAMA Cardiology paper describes several design elements that make ADAPTABLE, a demonstration study of PCORnet®, the National Patient-Centered Clinical Research Network, a novel pragmatic clinical trial. The manuscript’s authors detailed these trial elements with an aim to inspire efficiency and patient-centricity in future trials.

“A strong need has arisen to develop and execute more streamlined, pragmatic trials leveraging real-world data and technologies to generate real-world evidence to answer important clinical questions that have a direct impact on public health,” the paper’s authors write.

Using the broad and vast resources of PCORnet, ADAPTABLE is positioned to deliver answers in a way that is less expensive, more efficient, and less burdensome for both patients and clinicians. A key PCORnet-enabled design element of the trial included large-scale recruitment using EHRs from members of the Network. One of the goals of the trial was to test PCORnet capabilities for patient identification, recruitment, and follow-up. ADAPTABLE successfully recruited over 15,000 participants from among its 40 participating sites.

Patient engagement has been carefully planned and implemented throughout the trial, including constitution of a Patient Review Board for the study comprised of “Adaptors,” the patient leaders engaged to help lead ADAPTABLE. By design, one Adaptor has been identified from each of the seven participating PCORnet Clinical Data Research Networks in order to represent patient viewpoints from the geographic region of the sites. Through their participation, the Adaptors have reviewed various aspects of study policy and a variety of specific patient-facing study materials. They have contributed both reactively as well as proactively, providing many specific suggestions and also new unsolicited ideas about how to approach and engage participants in a way that will help the trial succeed.

In an accompanying commentary on this paper, Patrick T. O’Gara, MD, from the Cardiovascular Division, Department of Medicine, Brigham and Women’s Hospital, Harvard Medical writes that “JAMA Cardiology has not previously published trial design articles. The editors believe the methods incorporated in the ADAPTABLE trial represent important next steps in the more rapid and less expensive generation of reliable real-world evidence to enable better decision-making. The efficacy and safety outcomes of this 15 000-patient study are awaited with interest. In the interim, the design features should spur further efforts to improve the clinical research ecosystem.”

PCORnet Bariatric Study publishes latest clinical findings based on data from tens of thousands of bariatric surgery recipients

With its latest findings in JAMA Surgery on how many adults see their Type 2 diabetes go away and come back following weight-loss surgery, the PCORnet Bariatric Study now provides patients and their doctors with a more complete picture about the comparative benefits and risks of the two most commonly used surgical procedures. This real-world evidence enables patients in consultation with their providers to make more personalized decisions about which option would be best for them.

Drawing from the resources of PCORnet^®, the National Patient-Centered Clinical Research Network, the PCORnet Bariatric Study is the largest study to date to compare the longer-used Roux-en-Y gastric bypass procedure and the newer and somewhat simpler approach called sleeve gastrectomy. Its findings are based on analyses of data from tens of thousands of bariatric recipients securely accessed via PCORnet.

The study, funded by the Patient-Centered Outcomes Research Institute (PCORI), previously reported findings on how these two procedures compare in weight loss and maintenance among adult patients in Annals of Internal Medicine along with rates of short-term health risks. Findings about rates of long-term problems requiring reoperations and other interventions appeared in JAMA Surgery.

Now with PCORI funding, the PCORnet Bariatric Study’s findings are being incorporated into a decision aid to empower patients, in consultation with their clinicians, to make personalized decisions about weight-loss surgery.

“More people with severe obesity should be having conversations about the role of bariatric surgery, which has been underused because of concerns about safety and weight regain,” said the study’s lead co-principal investigator David Arterburn, M.D., M.P.H., an internist and senior investigator at Kaiser Permanente Washington Health Research Institute in Seattle. “The results of the PCORnet Bariatric Study show that the overwhelming majority of patients maintain successful weight loss long term, especially after bypass. But bypass patients had a higher risk of subsequent operation and hospitalization.”

“As with many health care options, no one weight-loss surgical procedure is the best choice for every patient because tradeoffs are associated with each,” added co-principal investigator Kathleen McTigue, M.D., M.P.H., M.S., an associate professor of medicine and epidemiology at the University of Pittsburgh. “The choice requires personalized decision making that involves weighing the evidence about the pros and cons along with each person’s individual circumstances and preferences.”

Because of the large sample sizes that the researchers could assess through PCORnet, they could examine subgroups of people to assess whether certain characteristics improved or worsened their chance of weight loss success. They found that men, African Americans, Hispanics, people age 65 and older, and people with diabetes or lower body mass index (less than 50) tended to lose less weight than did other people in the study.

But these differences between patient groups were small—less than 3 percent differences in weight lost at 1, 3, and 5 years across groups—which was much less than the differences between the procedures.

Both PCORnet and PCORI emphasize patient guidance and partnership in studies. People who have had weight-loss surgery served as co-investigators and helped to refine the study question, select outcomes to study, interpret the findings and present the results. Neely Williams, M.Div., who has served as patient co-principal investigator for the study, noted that having gastric bypass has helped her, but this study’s results would have been helpful at the time of her surgery decision because she was not sure which procedure was best for her. Her pre-surgery screening did not offer details, and she didn’t know what questions to ask. As a partner in the study, she was able to call attention to things that may or may not have been considered by the people who were doing the research.

Developed with funding from PCORI, PCORnet harnesses the power of real-world data drawn from electronic health records, claims data and other sources to generate real-world evidence. The network enables researchers to access aggregated, de-identified data to conduct research on large sample sizes.