DuchenneConnect, a program of Parent Project Muscular Dystrophy (PPMD), has been awarded $970,000 by the Patient-Centered Outcomes Research Institute (PCORI) to take part in developing a health data network. DuchenneConnect will be part of PCORnet: the National Patient-Centered Clinical Research Network. The DuchenneConnect network is one of 29 networks that were approved for PCORnet. The formation of the new national PCORnet resource aims to boost the efficiency of health research.
This is especially exciting for rare disorders like Duchenne and Becker muscular dystrophy, where health data networks may provide a pathway to faster, more effective clinical research to answer questions important to the Duchenne and Becker muscular dystrophy community.
DuchenneConnect is an established patient-report registry for Duchenne and Becker muscular dystrophies. With seven years of experience collecting longitudinal patient-report data that is utilized by industry, clinicians and academic researchers for advances in Duchenne and Becker muscular dystrophy, DuchenneConnect has been a model for rare-disease registries. The DuchenneConnect team is led by principal investigator Holly Peay, MS CGC, vice president of Education and Outreach for PPMD; and project director Ann Martin, MS CGC, DuchenneConnect Curator. The DuchenneConnect project benefits from key collaborations with W. Andrew Faucett, MS CGC, and F. Daniel Davis, Ph.D., at Geisinger Health Systems; Stanley Nelson, M.D., Nancy Halnon, M.D., and Richard Wang, Ph.D., at UCLA; and Kyle Brown and Jud Rhode at PatientCrossroads.
The PCORnet project comes with anticipated benefits for the DuchenneConnect community, and for the nation. PCORI envisions PCORnet to be a secure, national data network that improves the speed, efficiency and use of patient-centered comparative effectiveness research (CER). By integrating data available in the 29 individual networks, PCORnet aims to provide access to a large amount of diverse, nationally representative health information that can support a range of study designs. It will reduce the time and effort needed to launch new studies and focus research on questions and outcomes especially useful to patients and those who care for them.
Peay said, “Many of DuchenneConnect's achievements through this program will become available to other disease registries under the PatientCrossroads model, thereby extending the reach of PCORnet to a broader range of patient networks."
PCORnet will join together networks operated by both patient communities and health systems and will require patients' and other stakeholders' involvement in all aspects of the collection and use of the data. By enabling researchers and patients, clinicians and other end-users of study results to interact directly and jointly determine research priorities, such as the selection specific studies to support, PCORnet aims to advance the shift in clinical research from investigator-driven to patient-centered studies.
DuchenneConnect was selected through a review process in which patients, caregivers, and other stakeholders joined scientists to evaluate the proposals. Applications were assessed for the capacity of their network to collect complete, comprehensive clinical data; how well they will engage patients and other stakeholders; and their ability to maintain data security and patient privacy among other criteria.
All awards are approved pending completion of a business and programmatic review by PCORI staff and issuance of a formal award contract.