For 7 years, DuchenneConnect, a patient-report registry for Duchenne and Becker muscular dystrophies (DBMD), has collected longitudinal patient-report data that is used by industry, clinicians, and academic researchers. A parent-led foundation, Parent Project Muscular Dystrophy, founded and supports the registry.
Working with PatientCrossroads, Geisinger Health System, and the University of California, Los Angeles (UCLA) in the PCORnet project, DuchenneConnect will develop new approaches to balancing robust data collection with reducing the burden and increasing the benefits for registrants. We will integrate data from electronic health records, allow evaluation of patient-report outcome (PRO) accuracy, and facilitate the answering of questions of community interest. We will improve PRO standardization, while continuing to be responsive to emerging research questions. We will also improve coding and standardized information exchange. We will implement a multifaceted outreach approach to reach a more demographically representative—and larger proportion of—DBMD patients. The registry is guided by a multidisciplinary committee that includes individuals with DBMD and parents of patients, and we will systematically engage advisors, registrants, and the DBMD community in posing questions of interest, identifying innovative efforts to increase participant empowerment, improving care provision, and better defining DBMD natural history.
This project allows us to extend and leverage existing collaborations; for example, DuchenneConnect and the UCLA Department of Human Genetics have engaged in comparative outcomes research using registry data. UCLA collaborators will advise the project on improving data standardization and export. They will also work with us to explore use of large data sources to reach a more representative registrant population. Collaborators from PatientCrossroads and Geisinger Health System will identify and solve technical problems related to integration of electronic health information. PatientCrossroads will implement improvements to the data collection interface, as well as new capabilities for registry access and data input. Geisinger experts will advise on ethical and legal issues. Through the PatientCrossroads model, many of DuchenneConnect’s achievements will become available to other disease registries.