PCORnet®: Accelerating Patient-Centered Comparative Clinical Effectiveness Research

This special issue presents manuscripts reflecting the many services and uses of PCORnet®, the National Patient-Centered Clinical Research Network. PCORnet is a large, federated “network of networks” that facilitates innovative patient-centered comparative clinical effectiveness research (CER) and other health research at a national scale. As a flagship infrastructure program funded by the Patient-Centered Outcomes Research Institute® (PCORI)®, an independent, nonprofit research organization, PCORnet is unique among national networks given the scope and scale of the network, with linkages to 47 million unique patients and more than 13,000 sites of care. Research expertise, strong governance, and a commitment to keeping the patient at the center of all activities enables PCORnet to be used for a range of study types, from pragmatic trials to observational studies. Having just celebrated its 10th year, PCORnet® Network Partners have demonstrated that infrastructure integrating high-quality data, research expertise, and patient partnership contributes substantially to national-scale health research. PCORnet has been used to conduct more than 300 research projects in the US, including notable CER studies such as ADAPTABLE, the aspirin study, Greenlight Plus, BP Lab, as well as PREVENTABLE.1–5 PCORnet is open to all interested investigators, partners, and funders, and enables broad access to research resources through the PCORnet® Front Door.6 Given this stage of maturity, now is an appropriate juncture to reflect on lessons learned using PCORnet for the conduct of research done differently. This special issue was guided by an exceptional Technical Expert Panel (TEP) (Table 1) who advised on all aspects of the issue. In the fall of 2024, abstracts were invited for commentaries, empirical papers, case studies, and methodological papers. Themes include uses of PCORnet for patient-centered CER and other health research; engagement and patient partnership; infrastructure enhancements, and uses of PCORnet to accelerate progress toward an integrated learning health system. All abstracts were required to focus on uses of PCORnet to enhance the culture and capacity for patient-centered CER in the service of a transformed health research ecosystem. Perspectives from patients and other partners on all themes were explicitly invited. Forty-six abstracts were received. TABLE 1 - Technical Expert Panel (TEP) Participants Name Affiliation Melissa Bronson Patient Partner participating in PCORnet Sarah Dunsmore Program DirectorOffice of Translational MedicineNational Institute of Health Laura Forsythe Chief Planning & Evaluation OfficerPCORI Crispin Goytia Patient Partner participating in PCORnet Adrian Hernandez Vice Dean and Executive DirectorDuke Clinical Research Institute Erin Holve Chief OfficerResearch Infrastructure and InnovationPCORI Mike Kappelman Associate ProfessorPediatric GastroenterologyUNC Chapel Hill Kathleen McTigue Professor of MedicineUniversity of Pittsburgh Mark Weiner ProfessorClinical Population Health SciencesWeill Cornell Medicine Adam Wilcox Professor of MedicineDirector of the Center for Applied Clinical InformaticsWashington University in St. Louis Jennifer Wiltz CAPT, US Public Health ServiceChief Medical Officer, HHS Office of Health PolicyPediatric Faculty, Uniformed Services University Among the articles in this special issue, the papers reflect key dimensions of the PCORnet ecosystem. The papers review PCORI’s role in funding the PCORnet infrastructure to enhance patient-centered health research, and reflect on progress in achieving these aims. Others address specific innovations—both social and technical—using PCORnet. These include PCORnet® Network Partner experiences collaborating with patient leaders, health plans, and community health centers, as well as pathways for early and mid-career investigators. The final group of papers sets directions for the future of PCORnet in several areas of health research. In addition to this introduction by the Special Editors, 3 papers provide an orientation to the network, including a brief history of PCORnet and ways the PCORnet infrastructure has grown. The first of these, by Marschhauser et al,7 provides a review of PCORI’s rationale for funding PCORnet as an infrastructure capable of supporting innovation in clinical design and CER. Two manuscripts by Cohen et al,8 and Hernandez et al,9 review research innovations and research powered by PCORnet®. Five manuscripts address engagement and partnership using PCORnet. These include Merritt et al's10 reflections on patient leaders’ experiences participating in PCORnet, including a call to action for network partners to enhance careful, kind, and connected research; and John et al's lessons on patient engagement from the CHI-RON study, which aimed to increase the diversity of patients participating in the Congenital Heart Initiative.11 Bensksen et al12 provide a case study of the ADVANCE Clinical Research articulates the accomplishments, ongoing challenges, and importance of community health center research. Nauman et al13 review experiences collaborating with health plans to create more robust and flexible data linkages with health plans to facilitate work on shared research priorities with national and regional plans. Pagidipati offers one clinician researcher’s career pathway using the PCORnet infrastructure to share lessons for investigators’ interested in working with PCORnet.14 Three manuscripts consider innovations in technical infrastructure. Koscielniak et al15 present an approach to conceptualizing complex research networks using a generalizable method for network infrastructure mapping. Two papers by Marsolo et al16 and Nelson et al17 consider lessons from PCORnet® Network Partners’ implementation of new technologies, including Fast Healthcare Interoperability Resources (FHIR) standard, and using centralized text messaging for participant recruitment. Five papers provide insights from studies powered by PCORnet. Several of these characterize patient populations and priorities for research that can be addressed with the scale of PCORnet. Conditions ranging from pregnancy18 and peripartum care19 to cardiovascular disease and HIV,20 and COVID-19,21,22 illustrating the breadth and depth of data and research expertise across PCORnet. The papers offer strategies and methods using PCORnet to study a range of populations, conditions, and treatments. Finally, 3 papers set the stage for future opportunities using PCORnet, including accelerating progress toward an integrated learning health system. Abbasi et al23 draw upon experiences in leadership within the Food and Drug Administration to elevate specific opportunities for PCORnet to advance postmarket evidence generation. Franklin et al24 draw on their experience developing a roadmap for investigators to accelerate research in intellectual and developmental disabilities using PCORnet, and Rothman et al25 describe opportunities for collaboration between PCORnet® Network Partners and the NIH Genomic Learning Health System. As demonstrated by the breadth of the content and volume of the special issue, the PCORnet ecosystem is highly dynamic and brings together high-quality data, patient partnership, and deep clinical and research expertise. This special issue complements nearly one thousand research manuscripts that have been developed based on CER and other health research using PCORnet.6 Perhaps most import, the breadth of public resources on www.pcornet.org demonstrate that PCORnet is a learning system that can support research teams’ ability to design the next generation of high-impact studies. This special issue marks a point of inflection for PCORnet, reflecting substantial progress, shared learning, and future directions for collaboration. As the network continues to evolve, the papers are a useful snapshot of the unique features of PCORnet and opportunities for research teams to use PCORnet for national-scale CER and other health research.