PCORI's first Annual Meeting brought together more than 1,100 researchers, patients, caregivers, clinicians and others for three days of sessions that highlighted efforts to build a patient-centered comparative clinical effectiveness research (CER) community.

If you weren’t able to attend, or if you’d like to catch up on anything you missed while you were there, please visit our meeting archives page. You’ll find video highlights, archives of the plenary webcasts, photos, and more. We’re also posting slides from plenary and breakout sessions to the meeting agenda and recordings of sessions will also be added soon. Watch a brief video featuring some of the “voices” from the meeting below and continue reading for some highlights of the ideas, progress reports, and enthusiasm shared during the meeting. 

Reports of Progress, Promise, and Work Ahead

Throughout the meeting, speakers and attendees emphasized the gains made in advancing PCORI’s vision of “research done differently,” where patients and other stakeholders are partners in all aspects of the research process. Many noted it was just five years ago that PCORI set out to fund studies comparing how well different care approaches work with a focus on engagement and outcomes important to patients and those who care for them.

Victor Montori, MD, professor of medicine at the Mayo Clinic, spoke for many when he said during his keynote address that “PCORI has made engagement fundamental … It’s difficult to imagine research without engaging patients and caregivers and other stakeholders anymore.”

Elizabeth Cox, MD, PhD, a PCORI-funded researcher at the University of Wisconsin, Madison, made it more personal, saying she found it hard “to imagine ever doing another study where I don’t start out with patients and families first. It absolutely changes the way you look at a project and the way you do business, and I hope I don’t ever do another one without patient and family engagement.”

Growing Impact of Patient-Centered Outcomes Research

Presenters suggested a range of reasons during the meeting that patient-centered outcomes research (PCOR) is taking hold. “If the treatment, although it may be clinically sound, is not aligned with your personal circumstances or goals, we’re simply throwing care at you that is irrelevant,” noted Marc Boutin, JD, CEO of the National Health Council.

A number of researchers cited early evidence of how engagement can have a positive impact on some of the fundamental aspects of research. For example, Cox’s project surveys its teenage study participants regularly and reports a very high response rate, which she credits to the input of the teen patients and their parents on the project's advisory board. Another awardee, Katherine Deans, MD, MHSc, of Nationwide Children’s Hospital in Columbus, Ohio, added that her study's recruitment and retention rates increased significantly after the research team made changes based on input from their patient and stakeholder partners.

“What drives us every day is the key words in our name … ‘patient-centered,’” Grayson Norquist MD, MSPH, Chairperson of PCORI’s Board of Governors, said in his welcoming remarks. “If we haven’t made a difference in the lives of patients, we have failed.”

Challenges Remain

But for all of the progress PCORI and the PCOR community have made, there is still work to do, speakers also noted. Several laid out direct calls for action.

Lewis Sandy, MD, FACP, of UnitedHealth Group, challenged PCORI “to move faster … at the speed at which the patients demand it, [at which] the system is changing, and the speed at which payers need to make their decisions… We have more that we can do than we have really thought about doing, so please continue to explore and don’t get stuck in legacy thinking. Be as innovative as you can.”

Montori, meanwhile, identified collaboration as “the next big challenge.” Competition in clinical research poses a huge obstacle to getting the evidence and answers we need, he said. “We now need to make collaboration fundamental. That's going to give us the big science that we need,” he continued, pointing to initiatives such as PCORnet, the national, collaborative initiative of 34 health data and patient-powered networks developed through PCORI support, as models for such cooperation.

Session Highlights

Breakout sessions and summits featured research teams, many with their patient and stakeholder partners, sharing their progress and results. Visit the event website for the full agenda with links to slides of individual sessions. Audio recordings will be posted soon.

Engagement as a Path to Better Research

Many presentations focused on efforts to engage patients, caregivers, clinicians, and other stakeholders to see that the studies PCORI funds produce useful results more likely to be adopted in practice. 

Investigator-patient partner teams described experiences both positive and challenging. One was Kenneth Wells, MD, MPH, of the University of California, Los Angeles, and patient partner Loretta Jones, who heads Healthy African American Families II and the National Community Centers of Participatory Research Excellence. In their session, they noted how their efforts to overcome barriers to studying mental health issues in the African-American community include frank discussions of the historic legacy of mistrust in research. A guiding principle, Jones said, is “change can happen if you make sure everyone is sitting at the table. Who’s missing and how do we get them to the table?”

Another presentation described SMART DOCS, a randomized controlled trial of a coordinated care management approach for sleep medicine. Stakeholder advisors to the study led by Clete Kushida, MD, MS, PhD, of the Stanford University School of Medicine, include patients, clinicians, nurses, scientists, hospital administrators, professional association leaders, and industry leaders. Stakeholder engagement extends throughout the trial, during design, recruitment, analysis, and dissemination planning. As stakeholder partner Deborah Nichols noted, “We’re trying to make a change in the future of how sleep medicine is practiced for everyone.” 

During a session on patient-centered approaches to managing symptoms and side effects in cancer care, Mary Cooley, PhD, RN, FAAN, of the Dana Farber Cancer Institute, and Richard Boyajian, NP, a patient partner, described their work on an app to help patients deal with side effects of medication. “When I was discharged from the hospital after cancer treatment, it was ‘go home, come back for checkups,’ and it was up to me to figure out what to do, when to call the doctor,” Boyajian said. Interactions with patients and clinical experience led the team to concentrate on three symptoms to determine when to call the doctor in developing a decision support system, now being tested. 

In a related presentation, researcher Jun J. Mao, MD, MSCE, of the University of Pennsylvania, described a study comparing treatments for a problem that affects between 30 percent and 50 percent of patients with cancer – insomnia. As Mao’s patient partner Jodi MacLeod recounted, her difficulty sleeping made every other problem and symptom worse as she adjusted to life as a breast cancer survivor. As a study partner, MacLeod said it was “thrilling to see PCOR up close, to see how highly the patient experience was valued.” She said she was “looking forward to delivering study results to those who would most benefit. It’s been an empowering experience.”

A session on the Eugene Washington PCORI Engagement Awards highlighted a number of projects designed to advance knowledge and build training and development capacity for patients and others not traditionally involved in research. Angela Smith, MD, MS, from the University of North Carolina-Chapel Hill, described how her project is leveraging an online community to create an infrastructure for bladder cancer patients and their caregivers.  “One of the lessons learned” through the projects survey “is that many patients were disillusioned with research, so we must re-educate them with PCORI’s mission.” 

PCORnet Awardees Report on Progress and Future Plans

In a day-long summit, researchers and patient and community partners involved in PCORI’s PCORnet initiative shared updates on their progress in building this national resource to improve the nation’s capacity to conduct patient-centered outcomes studies. 

Mitchell Lunn, MD, a principal investigator of PRIDEnet, one of PCORnet's new Patient-Powered Research Networks (PPRNs), described how his team developed and is using an app to allow members of the LGBT community to become involved in research via their iPhones. Participants can give consent (signing with a fingerprint), provide health information, and participate in a discussion forum. In just 14 weeks, nearly 15,000 people have enrolled using the app. REACHnet, formerly the Louisiana Clinical Data Research Network (CDRN), also has used an app, deployed via 226 tablets in 16 clinics, to help gain 2,500 participants’ consent in the first six months of recruitment, noted principal investigator Tom Carton, PhD, MS. 

Elizabeth Cope, PhD, principal investigator of NephCure, a PPRN focused on nephrotic syndrome, told attendees that those involved in developing PPRNs “work together to answer questions we couldn’t answer by ourselves.” Patient-reported data, claims data, and clinical data together form a very rich picture, she said. But not to be overlooked, she emphasized, is the importance of capturing patients' reports on their feelings about participating in a PPRN and their reasons for dropping out. Practical suggestions to make participation easier can be particularly valuable, she said. 

Richard Colletti, MD, president and executive network director of the ImproveCareNow (ICN) PPRN, described the transformation of ICN from a consortium of clinics and centers into a collaborative network of patients, caregivers, clinicians, and researchers studying ways to improve the care of children with inflammatory bowel disease. “To go fast you work alone, but to go far, we work together,” he said. ICN, which started in 2007, now includes 81 medical centers and covers 22,500 patients. With the help of PCORI funding, it has expanded its activities to include retrospective observational research, comparative effectiveness research, and randomized controlled trials. 

Looking Toward 2016

Even though our first Annual Meeting just ended, we’re already planning the next one, reviewing evaluations from attendees and speakers for suggestions as to how we might make next year’s gathering even better than this year’s. We invite you to join that conversation, by posting a comment below or sending an email to [email protected].