When PCORI first proposed developing a national clinical research collaborative and community that would harness the power of partnerships and health data to improve patient outcomes, we knew we’d set out on an unprecedented and exciting path.
Today marks a major milestone on that path, with the approval by our Board of Governors of $142.5 million in awards to 34 research networks to participate in Phase II of PCORnet, the National Patient-Centered Clinical Research Network*. We thought it was a good time to reflect on what we accomplished in Phase I and on the opportunities and challenges that lie ahead.
PCORI has invested more than $100 million so far in developing PCORnet, a large “network of networks” designed to link researchers, patient communities, clinicians, and health systems. The goal is to allow the nation to conduct patient-centered comparative clinical effectiveness research (CER) more efficiently and less expensively than is possible now.
This resource will allow researchers to access, under strict privacy and security protections, large sets of health and healthcare data gathered in real-world settings, such as clinics and hospitals. The ability to use this information, representing tens of millions of people across the United States, should make it easier to conduct large studies that will help patients and those who care for them make better-informed decisions about their healthcare options. Potential projects include observational studies, which analyze data already collected by partner networks, and experimental studies, which randomly assign participants to one of two or more approaches. In both cases, the purpose of the study is to determine which approach to a health problem yields better outcomes for individual patients. Often these studies must be large to determine which choice is better, for whom, and under what circumstances.
Phase I: Building the Network
We set some ambitious goals for PCORnet during its 18-month development phase, and the report card is quite positive overall. We achieved our goal of creating a large, national research collaborative, led by the PCORnet Steering Committee and its Executive Committee. We refined the capabilities of the 29 partner networks and developed overall policies for data sharing and patient-centered governance. We built the data infrastructure: clinical data from electronic medical records, insurance claims, and other sources are now stored in identical datasets (the PCORnet Common Data Model) across the 29 networks and represent more than 60 million persons.
PCORnet’s organizational structure has taken shape, with individual networks building strong relationships with each other and the coordinating center providing overall technical and management support. And the pieces are now in place for research to begin; in fact, our first demonstration project is under way—a very large multi-site study of the best dose of aspirin to use to prevent heart attacks and strokes in people with heart disease.
Phase I of PCORnet included 11 Clinical Data Research Networks (CDRNs), which are based in healthcare systems such as hospitals or health plans and collect information during the course of routine patient care. These were joined by 18 Patient-Powered Research Networks (PPRNs), operated by groups of patients and their partners and focused on particular conditions. The 45-member PCORnet Steering Committee and its 12-member Executive Steering Committee, advised by the seven-member PCORnet Patient Council, oversees overall operations and develops policy.
During Phase I, we’re pleased to say that the CDRNs met their goals: each organized data from at least one million patients and standardized those data to conform to the PCORnet Common Data Model (CDM), which maps each network’s data, no matter how it was originally recorded, to a consistent format (such as describing patient characteristics and vital signs in exactly the same way). All CDRNs have implemented governance plans that include patients and clinicians.
The PPRNs have met many of their goals as well, including setting up governing structures that include patients, collecting data on at least 80 percent of the patients they’ve identified, and transforming their data according to the PCORnet CDM. But some have had challenges in recruiting the target numbers of patients willing to participate in the studies they’ll manage.
Specifically, the PPRNs’ goal was to recruit 0.5 percent of the US population with the disease or condition they proposed to study. Generally, the PPRNs that are focused on rare diseases met that goal, but those that address more common illnesses have had more difficulty. This raises important questions about what that goal should be and what it means for a patient to be enrolled as a participant. We’re looking carefully at how to address this challenge—one current consideration is whether PCORnet might offer different levels of patient involvement, enabling patients to choose the extent of information that they share with researchers.
Phase II: Ready for Research
Our vision for PCORnet during the next three years is to sharply ramp up capacity to serve as a platform for large-scale, patient-centered research and to conduct many high-quality studies, quickly and efficiently. Under the newly approved awards, PCORnet will expand its basic structure of CDRNs and PPRNs, again supported by the coordinating center. It will also include two PPRNs centered on communities rather than diseases.
During Phase II, the seven newly funded networks—two CDRNs and five PPRNs—will develop their data resources, and PCORnet will continue to streamline administrative processes, such as contracting and Institutional Review Board approvals, to speed the initiation of research projects. We will finalize our governance policies, which include patient engagement, leadership, decision making, and data privacy and security.
During Phase II, we expect the partner networks to conduct individual and joint research projects, for which they may supplement PCORI funding with other funds. Also, during Phase II, we expect to begin allowing outside researchers, within clear and stringent rules and safeguards, to use PCORnet data for a wide range of studies. We have already begun development of transparent “front door” policies for handling research requests. Creating a common culture to transform clinical research and serve the nation’s investigators and funders will help establish PCORnet as a true collaborative network.
Harnessing Patient Power
One of the aspects of PCORnet that has, from the start, made it such an exciting and potentially transformative opportunity is its focus on bringing patient communities into the very fabric of the network’s development and ongoing operations. As with all of the work that we do at PCORI, our intent here is to harness the power of true patient-researcher partnerships to improve health care and outcomes.
In its first 18 months, PCORnet already has made substantial progress toward this goal. Patients take a leadership role in all of the PPRNs, and along with researchers, clinicians, and health system leadership, they participate meaningfully in governance of all of the CDRNs. Eight patients are members of the PCORnet Steering Committee, and two serve on its Executive Committee. A patient recommended the creation of the Patient Council to ensure that patient perspectives are reflected in all PCORnet-wide policies.
In Phase II, we plan to accelerate this patient power by establishing ways for the PPRNs to efficiently share knowledge, expertise, and infrastructure and empowering those networks and their communities to participate in key national discussions about health research. The goal is to start a new kind of conversation about what it means to participate in research and to take charge of own one’s health.
Making PCORnet Successful and Sustainable
We also have begun discussions of how PCORnet can become a sustainable national resource in years to come. Over time, PCORI’s support for PCORnet will decrease with the expectation of increased funding from agencies such as the National Institutes of Health and Food and Drug Administration. PCORnet may evolve into a consortium or an individual entity, but seeded by PCORI’s initial investment, we envision a long life for this initiative. Preparing the network for transition to independence will be an important task throughout Phase II.
We know, however, that whatever our plans, PCORnet can’t succeed if there is not something in it for all of the key players—researchers, patients, health systems, clinicians, and funders. We’re committed to ensuring that each of these communities sees the value in this initiative and joins us in determining the most effective way for it to function to improve patient outcomes by harnessing the power of partnerships and health data.
*Totals were revised following Board approval of the awards. The new totals as of September 2015 are $141.3 million for 33 networks.