Patient-Powered Research: Keynote Speech at the Inaugural Learning Health Network Research Conference

(June 24, 2021) I am honored to be the keynote speaker at the Inaugural Research Conference of the Learning Health Networks.

Is it possible that the pace of change in medicine and health care delivery could be fundamentally changed? Is there another gear that could transform our performance and vastly improve outcomes?

I want to share an idea from a while ago that I thought might bring a change to research. The idea was to flip the balance of power in research through Patient-Powered Research Networks (PPRN) to transform the quantity and quality of research output. In essence, researchers would be working for and be accountable to patients. Research questions and goals would be generated by collaborative groups led by patients.

I was on the Patient-Centered Outcomes Research Institute (PCORI) board when I first pitched the idea.

For context, in 2010, the Affordable Care Act (ACA) established PCORI and I was fortunate to be a founding member of the Board of Governors. Remarkably, Congress established PCORI as an independent organization (“neither an agency nor establishment of the United States Government”). This independence meant that PCORI had funding for about a decade and could take some risks in forging a new path.

The legislation stated that PCORI was “to assist patients, clinicians, purchasers, and policy-makers in making informed health decisions by advancing the quality and relevance of evidence concerning how diseases, disorders, and other health conditions can effectively and appropriately be prevented, diagnosed, treated, monitored, and managed through research and evidence synthesis…” The important words here were that we were mandated to advance the quality and relevance of evidence to help people make more informed health decisions.

When the Board first met, we realized we had $3 billion and about a decade to fulfill that purpose, but little other guidance. We had no infrastructure or bylaws. The Comptroller General of the United States had appointed us, but we were free to chart our course.

Beyond administrative tasks, our first objective was to define PCORI. It had emerged from Congressional interest in supporting comparative effectiveness research — investigations that would provide insights about head-to-head comparisons of alternative approaches. I had heard that Congress developed the legislation on the idea that much of the waste in medicine is related to pursuing clinical strategies that may have little additional value beyond the less expensive alternatives.

As Congress negotiated the ACA, rumors of rationing and death panels led the Senate writers to seek a name that was more disarming than a Comparative Effectiveness Institute, which might have amplified concerns that the Government would use the evidence to restrict choice.

At the time, I was the Director of the Robert Wood Johnson Foundation Clinical Scholars Program at Yale, a training program that sought to develop future leaders by providing them with solid training in research methods, health policy, and leadership skills. In 2005, the Foundation mandated that we start a program in Community-Based Participatory Research (CBPR). CBPR is “a partnership approach to research that equitably involves community members, organizational representatives, researchers, and others in all aspects of the research process, with all partners contributing expertise and sharing in the decision-making.”

I had long fashioned myself as a patient advocate. My father was a physician and from an early age I would tag along on rounds. I witnessed the special nature of the bond between patients and their doctor, and the homespun wisdom of many of those patients as they coped with illness and uncertainty.

As an undergraduate, I spent a semester with the North Carolina Office of Rural Health Services. Led by Jim Bernstein, a former Peace Corps volunteer, this Office pioneered ways to partner with communities to bring primary care to under-served areas. After college, I took a year before medical school to study primary care delivery in other countries, focusing on how communities united to be a force for health. In medical school, I worked with community health centers and learned how they managed the mismatch between resources and health needs — and the importance of community engagement. My medical school graduation speech was about how doctors need to see through the eyes of their patients and my research throughout my professional career has focused on producing knowledge that improves health care and supports more informed patient choices.

And yet, the idea of CBPR took me by surprise.

I had been socialized into the hierarchical nature of medical research, with researchers at the top, making the decisions and not doing much sharing — socialization so intense that I am still unlearning it.

In a chapter from a book by Barbara Israel and colleagues on the Methods of Community-Based Participatory Research for Health, Nina Wallerstein and co-authors noted that “CBPR is described as supporting ‘collaborative, equitable partnerships in all phases of the research’ which will “promote co-learning and capacity building among partners.”

They write that “CBPR is dependent on partnerships, yet the skills and methods we need to develop and maintain research partnerships often are not taught or explored in academic settings.” That chapter, published in 2005, remains true today.

The traditional, hierarchical approach handicaps research by overlooking the involvement of the people it seeks to help. It neither taps their wisdom and experience nor respects and honors their role.

In this traditional model, research interactions with people participating in studies are transactional, not relational. The study design and the workflows suit the researchers but not necessarily the participants — which ultimately hampers investigations.

Let’s consider the word ‘subject,’ as in research subject.

The word subject is from Latin, with “sub” meaning under and “jacere” meaning throw. It soon became “subjects,” which meant brought under, and by Middle English, the word “implied owing obedience. In the 14th Century, subject meant “person under the control or dominion of another.”

The word and what it signals are toxic.

In some of our early insights about CBPR, we reflected on our previous research and realized that often we had not engaged people before we started, rarely incorporated their suggestions during the investigation, and often did not tell them the results. When we finished the study, we could not remember sending thank-you notes or any other expression of gratitude for their participation. We had much to learn about being good partners — and that work continues.

A counterpoint to the traditional approach was our research to improve the timeliness of treatment for heart attacks. Some heart attack treatments — the ones that seek to re-open a closed coronary artery — are time dependent. The longer it takes to re-open the artery, either through medications or stenting, the less effective the treatment is. Which makes sense, as the longer that portions of the heart lack blood flow, the more irreversible injury that occurs — and at some point, it is just too late to re-open the artery to revive the damaged muscle.

We conducted an NIH-funded study to learn how to improve the timeliness of treatment. In this case, clinicians were our community and we incorporated them in every step of the research. We also started the study with a listening tour, visiting hospitals and talking with clinicians. We focused on exemplary performers and tried to learn what they were doing that enabled them to be positive outliers. We conducted qualitative research to elicit themes and understand challenges. Then we conducted a national study to elicit what worked best to produce the best outcomes.

The research was tuned from the beginning for translation. As we published the results, we turned to the American College of Cardiology to promote the translation of the knowledge. Within a year, national times to treatment dropped by nearly 50% with the adoption of evidence-based processes and hard-wired feedback on every patient. The comprehensive involvement of clinicians ensured that we were asking the right questions, collecting data in convenient ways, and producing actionable results — and helped set the stage for hospitals to abandon long-held practices that contributed to delays. Today, we can confidently say that wherever you are in the US, if you have a heart attack you can be assured that you will be treated rapidly. The change in performance has been remarkably durable.

By the time I started at PCORI, I had the experience of five years in a grand experiment at Yale to change research culture, at least within our Program. The grand idea was to work in partnership with patients and the communities we sought to serve with our research. We had much to learn about our community and the people who surrounded us. Community members became our teachers. We worked to be worthy of their trust. Gradually we did better, and launched projects that were drawn from community ideas and guided by community wisdom, with community members as co-investigators.

The initial idea I brought to PCORI was to seize the term “patient-centered” and embrace the idea that people would be at the center of what we did, including participation in selection panels, research prioritization, and research dissemination — and that we would only fund projects that genuinely engaged people. The idea took root immediately among an enthusiastic Board of Governors who genuinely sought to incorporate the ideas, energy, and perspectives of the people who faced the health challenges. The idea became ‘research done differently’ to convey that the knowledge could and would be coproduced.

But the problem was that the research ecosystem had substantial inertia and it was not easy to see how the system might change.

An even bolder idea was to test the concept that more fundamentally changing the role of patients in research funding and oversight would produce a positive transformative change. Francis Collins had famously said that working with researchers was like herding cats — and the only effective means of changing behavior was to move their food.

The inspiration for this bold new approach was inspired by my experience at a PCORI retreat at Stanford, where we had invited patient groups to present their perspectives. At one point, someone stood at the microphone to the side of a large room and introduced herself as someone associated with Autism Speaks.

Autism Speaks is a grassroots organization whose vision is a world where all people with autism can reach their full potential. Their mission is to “promote solutions across the spectrum and through the life span, for the needs of individuals with autism and their families.”

The speaker drew a breath and, in a moment, she described the need for knowledge to inform choices in support of people with autism, and made clear how insightful, capable, and wise they were about the condition and its challenges. I had the thought — what if these people, instead of being a target population for researchers, had the power to hire researchers to solve the problems that were important to them.

I was also aware of the Cystic Fibrosis Foundation, which raised considerable money, placed its bets on scientific research, and built patient registries to support trial enrollment. They developed a novel strategy of venture philanthropy that made payouts when they reached milestones. They did not care about publications; they cared about progress.

People like Sharon Terry had also inspired me. Doctors diagnosed her two children with a rare genetic disease, pseudoxanthoma elasticum, and she pivoted to become a genetic researcher and a leader in open science. She helped forge a central repository of blood and tissue so she and her collaborators could force the scientists to be collaborative.

With PCORI’s freedom to innovate, I wondered… what if we truly gave the reins to patients to provide oversight of the funding of some of the research? What if we could bring together people facing health challenges to lead how we invested the money in research, how we held people accountable, and how we ensured that the data would help propel advances through open science? These people could also set the rules of engagement, requiring that the research involved patients in partnership. They could set the rules on selection process. PCORI could provide infrastructure, training, and scientific support.

In my dreams, a selection process could be quite different than the traditional approach. Imagine a diverse group of patients and caregivers who have a particular health challenge. They have worked with their respective communities to prioritize research topics and engage with PCORI to assemble applications. Venture philanthropy experts would help them put together a plan that distributes funds based on performance. The application has two parts. Part one describes the aims and methods and would be vetted by scientific experts for rigor and risk. Part two was where it got creative. Applicants would submit an imagined product of the research in the form of a press release and a magazine article that described the journey of the research and the outcome for the public. Then there would be an interview in which the patients determined which researchers deserved their support. They would get to look the researchers in the eyes and determine their commitment to the research and to partnering with patients. The budget process would align with quarterly milestones and the release of funds would correspond to accomplishments.

In this new construct, the people with the most at stake are the decision-makers. They would depend on experts to vet the promise of the science. They would look at what the researchers imagine they could deliver to determine if the project is worth the investment, with payment dependent on performance. The performance would not be not about the result but about achievement of the milestones.

Patients are now positioned as savvy consumers, eager to purchase knowledge. They are not paying for research; they are seeking a return on investment that could tangibly make a difference for people in their position. Of note, we had previously published that as many as half of NIH-funded trials that are completed do not report their results (for reasons that are unclear, and without consequence). I don’t think that would happen if payments were tied to milestones, like reporting results and sharing data. And what if researchers had to face patients and explain why they opted not to share their results — the findings from the experiments they did on people and with funding to produce knowledge that depends on those studies?

I believe that this approach would be good for researchers too. The innovation of putting patients in charge would get people thinking differently about their work and the endgame. The emphasis is focused on what patients view as important — the process and outcome of the research.

Unfortunately, the experiment never really got off the ground. There were calls for PPRNs and funds that were disbursed. But the efforts were underfunded and PCORI could not fully get behind the vision of this approach to empowering patients. In the end, I think it was just too radical of an idea. To me, it was not extreme. The model would have required iteration for proper optimization, while ensuring co-production by handing the funding power to patients (in tandem with the Board, which had fiduciary responsibility). It seemed like a sound approach.

I want to pause on that word co-production and credit Paul Batalden, another of the inspiring figures in my career, along with Gene Nelson and others who have promoted the importance of collaboration in the journey to do better in medicine.

I recommend Paul’s writings, as they have broad applicability. He wrote that “coproduction of health describes the inter-dependent work of users and professionals who are creating, designing, producing, delivering, assessing and evaluating the relationships and actions that contribute to the health of individuals and populations.” “Rethinking healthcare as a coproduced service adds depth to our understanding of how we might better design and make services, improve them, and ultimately increase their contribution to better health.” This has prompted me to think about research as a co-produced service too.

Too often, I hear people say that patients are not capable of engaging — that they lack knowledge, expertise, and even ability. I know that is not true.

Before the pandemic, I produced a podcast called “Never Delegate Understanding.” The premise was that patients should not defer to others but realize that they can understand the critical considerations in decisions that affect their health. I interviewed many of my health care heroes: Sara Riggare, Gregg Gonsalves, Liz Salmi, Hugo Campos, e-Patient Dave, Dana Lewis, Andrea Downing, Bray Patrick-Lake, Christie Mangir, Sue Sheridan, and Patrick Gee. They taught me so much about courage and persistence.

In the podcast with Gregg Gonsalves, he related a story that he heard from Eric Mama, a physician with Doctors without Borders. Mama was seeing a South African woman attending an HIV clinic in a poor township. She said, “Dr. Eric, you have to take me off of D4T. I am getting all this peripheral neuropathy in my feet. I think you should switch me to Tenofovir. I know there are issues around bone density, but I think it’s the right thing to do.” This young woman who didn’t go to high school but had been part of community education projects in that township showed the level of comprehension possible when we spend the time engaging people.

By now, you may understand the why. It is not just the right thing to include patients in research; it’s a competitive advantage. People can keep researchers grounded, correctly oriented, and accountable. In academia, you are rewarded for intermediate milestones like papers, citations, grants; but patients care about what your work has produced for them. Also, people facing health challenges are repositories of great wisdom about their condition and its manifestations.

So there is little question about when we should adopt this approach. Now. We should have done it already. And it is being done in some places; we just need more.

And I have hope. I have hope when I work with colleagues like Carley Riley, Brita Roy, Erica Spatz, Oyere Onuma, and Yuan Lu, for whom working with patients and communities is what seemed right to them from the beginning. They inspire me about the future.

I have hope when I see what is happening in the long COVID community. Survivor Corps, a leading grassroots COVID patient group, is producing research from its 150K+ Facebook group that is catalyzing progress. We, at Yale, are partnering with them on a study of the effect of vaccination on long COVID. The Patient-Led Research Collaborative, a group of people with long COVID who are conducting research and making contributions, also helped us with our surveys, as did individual patients. In these cases, the patient groups are leading the researchers and it is happening organically.

I have hope when I see this organization, the Learning Health Networks, putting patients first at every step and seeing unprecedented results.

But how do we get there? We need to address a culture that does not welcome co-production. We need to address inertia that maintains an under-performing status quo. Technology and data will be part of this change, but I chose to focus this talk on the cultural shift that could enable us to leverage the technology and data in the best possible way.

We need to think of ways to open avenues of collaboration and to bring in the wisdom of patients as an integral part of clinical and population health research and improvement.

We also need to emphasize and reward breakthroughs in implementation that address structural racism and inequities that undermine health.

We need to invest in infrastructure that aligns discovery, improvement, and accountability in medicine with patients’ perspectives at the center.

We need prototypes that test approaches. This group is an incubator for ideas and a testbed for patient-powered research.

I do want to share another example at Yale that is exciting, the OPTIMUM study, led by Elizabeth Claus, a leading neurosurgeon and biostatistician. The best thing I can do is share what patient advocate Liz Salmi wrote me about the evolution of this study and her observations of it.

Liz said that she enrolled in Claus’ low-grade glioma research registry in 2014. She described the sign-up process as “super complicated — it took me 4 weeks just to get everything submitted. If I wasn’t a big nerd, I might have given up after just 1 hour of friction.” She then met Elizabeth at a brain tumor conference and told her how hard it was to participate in the study. A few years later, Elizabeth asked Liz to be an advisor on a major grant and Liz said she did not want to be an advisor — she wanted to be an investigator. She wanted to help design the messaging, recruitment, and feedback process for other patients like her living with low-grade glioma. Liz had already been working with Bethany Kwan, who had a project requiring multi-stakeholder engagement. In the end, Elizabeth and Liz began working together, with Bethany joining too, to craft a truly innovative approach to brain research that will bring in patients as partners — and some, as researchers. Liz says, “I was inspired and emboldened by patient-researchers like Dana Lewis, who doesn’t want to be studied, she wants to lead the study.”

I asked Liz what changed when a patient becomes a co-investigator. She said it starts with how people talk. She wrote to me that “When we first started plotting the project, one of the bench scientists referred to both human and canine tumors in the same sentence as if we were equal participants. And I guess, without a ‘patient in the room,’ we would continue to be just as involved as dogs.” She also noted that the project has a unit that she leads that is focused on the experience of participants.

Liz conveyed the importance of the early adopters of this approach. The work of the early adopters can embolden people like Elizabeth Claus to trust patients as valued members of the team, beginning a wave of adoption among more traditional investigators.

So, kudos to the Learning Health Networks. You are the pioneers. Your work will de-risk these approaches for those who follow you. I was on a call this week, and Paul Wicks was quoting something from Susannah Fox… something like … people say… that’s crazy, that’s crazy, that’s crazy… oh, that was obvious. We are closing in on the day when people will see what you are doing not as radical or fringe, but so obviously beneficial that they will wonder what took so long.

Thank you all for what you are doing.