Multistakeholder Engagement in PCORnet, the National Patient-Centered Clinical Research Network.

OVERVIEW

The Patient-Centered Outcomes Research Institute (PCORI) was established to fund research that helps people make more informed health care decisions and improves the delivery of care by producing evidence-based information. PCORI-funded research is guided by patients, caregivers, and the health care community; at the core of PCORI’s mission is a commitment to meaningful multistakeholder engagement at all stages of the research process. The purpose of this article is to describe PCORI’s research network and the critical role that multistakeholder engagement plays.

To fulfill its mandate for advancing the Nation’s capacity to conduct patient-centered outcomes research (PCOR), PCORI-funded PCORnet, the National Patient-Centered Clinical Research Network. Since its inception in 2014, PCORnet has worked to build a resource that can meet an urgent need—to conduct PCOR faster, more efficiently, and at a lower cost.1 By leveraging the power of previously collected health data and unique partnerships with patients, clinicians, and delivery systems, PCORnet aims to conduct highly relevant clinical research by creating a Network of networks that engages these health care stakeholders in the development, execution, and dissemination of clinical research.

PCORnet is comprised of partner networks—Clinical Data Research Networks (CDRNs), Patient-Powered Research Networks (PPRNs), and a more recent addition of Health Plan Research Networks (HPRNs). The CDRNs are centered within care delivery systems, ranging from academic medical centers to integrated delivery systems, federally qualified health centers, and other community-based clinical networks that collect electronic health information at the point of care. The CDRNs include entire populations that receive some or all of their health care at participating systems. CDRNs draw patients from specific geographic regions, most covering multiple states and diverse patient populations.

The PPRNs are organizations operated and governed by groups of patients, caregivers, and community members, with a shared interest in driving research to better serve their communities and improve health outcomes; they include organizations dedicated to both common and rare diseases, families and caregivers of persons living with a disease, and communities not traditionally included in research. Two of the PPRNs are focused on vulnerable or underserved populations that are not defined by specific conditions. Across the community-based and condition-specific PPRNs, these networks involve patients, caregivers, and community members interested in participating in clinical research and in the governance of research activities.

The HPRNs are led by the research arms of 2 of the country’s largest private health plans and are working to facilitate the inclusion of claims data in PCORnet-based research to support the Network’s efforts to achieve more complete, longitudinal data and to incorporate the perspectives and interests of payers in PCORnet’s work. In addition to the technical linkage work, these HPRNs are actively engaged in data and research governance activities within PCORnet.

The goals of PCORnet are to support a range of study designs, including large longitudinal observational studies, pragmatic clinical trials conducted within participating delivery systems, and rapid-cycle research such as exploratory prevalence and surveillance studies. It is envisioned that PCORnet will serve as a national resource to support large, multinetwork research projects funded by PCORI, as well as other private and governmental funders and sponsors. By investing in a reusable research infrastructure, PCORnet hopes to address some of the challenges of traditional clinical research, such as the high costs associated with one-off approaches to conducting single studies, lack of adequate data on rarer conditions or on underserved populations, and limited generalizability of study results.

PCORnet is focused on improving the efficiency of clinical research while also shifting the culture from one directed by researchers to one driven by the true needs of patients, caregivers, clinicians, and communities. As part of this cultural shift, PCORnet aims to set a precedent for effective multistakeholder and community engagement within a large national research network. With the increasing use of electronic health records in the United States and the capacity to collect health-related information from people directly, networks have the potential to combine data from clinical care settings and lived experience of patients and community members, providing opportunities for innovation in both research and clinical care.2 However, to meet the real-world needs of these stakeholders and to legitimately use the health data of patients, this research must involve stakeholders in the research and clinical improvement processes to a degree not seen previously in clinical research.

A key emphasis of PCORI’s investment in PCORnet has been the inclusion of robust principles of patient and stakeholder engagement in network activities, including building off of resources such as the PCORI Engagement Rubric, which serves as a framework for operationalizing engagement in all phases of research. The engagement of the entire range of stakeholders, including patients/participants, caregivers, families, communities, clinicians, and host health systems and health plans will, we believe, contribute to the Network’s long-term sustainability, legitimacy, and capacity to produce meaningful results. PCORI requires incorporation of stakeholders and their perspectives into the overall infrastructure and governance of PCORnet, and the research they conduct. Each stakeholder group provides a unique perspective that can enhance the research by helping to identify specific knowledge gaps and direct the research toward filling those gaps.

During its first 2 years (2014 and 2015), PCORnet engagement strategies were largely developed locally to fit the circumstances and capacities of the initial 29 networks. PCORnet encouraged partner networks to share and build upon individual networks’ accomplishments and successes in community engagement. As the Network has evolved, an Engagement Committee was formed to guide and coordinate PCORnet’s growing engagement processes. Activities of the Engagement Committee include developing engagement policies, disseminating best/promising practices and tools, and compiling standardized engagement-related terms and definitions. This Committee—1 of 3 committees that provide decision-making and leadership across PCORnet—continues to work to improve and oversee PCORnet’s engagement of patients, clinicians, researchers, health system leaders, and other health stakeholders.3

During these past 3 years, the CDRNs and PPRNs have continued to enjoy latitude in the development and implementation of individual network-level engagement strategies, with a resulting diversity of arrangements by which stakeholders are engaged in many aspects of network governance. Stakeholders serve as network principal investigators, on Executive Committees, and several networks include a stakeholder or community-focused committee within their central governance. Networks support multistakeholder engagement in preresearch and research processes including research topic identification and prioritization, proposal development, research design and implementation, and dissemination. These processes also include infrastructure components such as citizen-scientist training programs, web-based platforms for eliciting research ideas directly from stakeholders, and stakeholder-driven proposal review committees, as well as study-specific engagement efforts.4,5 The PCORnet Commons serves as a platform for sharing such individual network engagement tools and practices to allow for peer-to-peer learning across PCORnet.

Over these first 5 years, PCORnet has continued to improve governance structures and engagement practices on the basis of lessons learned. Some of these lessons are detailed in this Special Issue. Beginning in 2016, several research demonstration projects have been serving to test different engagement strategies and their efficacies in the context of the Network. PCORnet partner networks have faced challenges and piloted innovative strategies with regard to engagement in the development of a research network, including patient privacy, data sharing, project review, prioritization and approval, recruitment, and informed consent. PCORnet and the papers in this Special Issue provide insight into these engagement practices and describe tools that can be leveraged more broadly in the context of data networks and patient-centered research.

As a national consortium of clinical and patient-powered groups working together to carry out stakeholder-driven research more effectively and efficiently, PCORnet provides a unique ecosystem for building on multistakeholder engagement to create a sustainable national research infrastructure. In 2018, PCORnet investigators participated in the creation of the People-Centered Research Foundation (PCRF), a not-for-profit corporation that will serve to carry PCORnet into the future, coordinating network activities and serving as a central contact for potential research sponsors. Engagement and a focus on the practical questions of patients and other stakeholders are central tenets of PCRF’s Articles of Incorporation and Board Charter. PCORnet and PCRF will continue to learn much about the practice, challenges, and outcomes of engagement in the context of a multisponsor, national clinical research network that must balance the goals of efficiency, relevance, and sustainability.