DuchenneConnect (DCN) is a program of Parent Project Muscular Dystrophy (PPMD), the largest and most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne and Becker muscular dystrophy (DBMD). DBMD is a set of rare, progressive neuromuscular disorders that cause progressive muscle weakness and early death. Onset is typically in the pediatric years.
DCN is a self-report registry that began collecting data in 2007 as a community resource to facilitate and speed research in DBMD. The current size of the network is over 3,000, and we estimate adding approximately 50 new registrants a month in Phase II. The fact that DCN is a program of an advocacy organization has strongly influenced the registry’s goals, processes, and governance. Patient and caregiver engagement is at the heart of advocacy organizations and is deeply ingrained in their culture.
DCN has successfully achieved Phase I goals of patient and caregiver engagement and governance, enhanced data collection, and rigorous but streamlined informed consent. We have improved recruitment with a continued focus on outreach to underserved communities. Our data are analysis-ready in compliance with the common data model. We have confirmed our ability and willingness to participate in collaborative PCORnet research with other clinical data research networks (CDRNs), patient-powered research networks (PPRNs), and outside organizations. DCN successfully engaged our communities to identify resource priorities, prioritize topic domains, assess face validity of existing or purposively developed patient-reported outcomes, and rapidly contact our registrants for purposes of recruitment into clinical trials and research studies. We will continue to collaborate with PatientCrossroads and Geisinger Health Systems to achieve Phase II milestones, and with the University of California, Los Angeles through implementing shared priorities. In addition, DCN is developing robust, mutually beneficial relationships with CDRNs and PPRNS where the needs and priorities of patients and caregivers are actualized through collaborative research and decision support tools.
New opportunities in Phase II include the integration of PPMD’s Certified Duchenne Care Centers (CDCCs). These CDCCs represent a robust, engaged network of expert clinicians who are proven partners in addressing community-identified research priorities. DCN will also implement a community-engaged request for proposals, which will contribute to the identification of research priorities, the development of multi-stakeholder research groups, the leveraging of PPMD resources to support protocol and proposal development, DCN mentoring, and avenues to sustainability funding.
DCN will continue to contribute to PCORnet as a PPRN that is directly accountable to patients and caregivers; as a network with years of collaborative research experience with academia, industry, and patients and families; in our emerging capacity to integrate electronic health record data; and most importantly as an engaged network responsive to the needs of our stakeholders. DCN is anticipating an unparalleled opportunity to answer questions of high importance and identify and address unmet needs related to our progressive, fatal pediatric disorder. We continue to value the PCORnet philosophy and experience and anticipate continued efforts to engage fully with our esteemed network partners.
View more about this project on PCORnet.org.
^Holly Peay, MS and Ann Lucas, MS, CGS were the original principal investigators on this project.