Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), today announced the launch of the organization’s Electronic Health Record (EHR) Study with the first patients consented to participate.
| Research Study Will Collect Information Directly From Patients’ Electronic Health Records to Help Researchers and Clinicians Better Understand Duchenne and Becker Muscular Dystrophy WASHINGTON, Dec. 6, 2022 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), today announced the launch of the organization’s Electronic Health Record (EHR) Study with the first patients consented to participate. PPMD’s EHR Study will extract data from several of the organization’s Certified Duchenne Care Centers (CDCCs) across the country to collect clinical data to inform research and care without adding additional burden on patients and families. Arkansas Children’s Hospital is the first CDCC to participate, with several more CDCCs in the process of joining PPMD’s EHR Study later this year.
As part of the study, data from consented patients’ health records will be securely sent to PPMD’s Duchenne Outcomes Research Interchange (‘the Interchange’), a patient- and clinician-reported data warehouse which can combine EHR data with data from The Duchenne Registry, as well as data provided by industry partners with approved therapies. “For the last several years, PPMD has been expanding and enhancing The Duchenne Registry, our comprehensive patient-reported outcomes (PRO) registry for Duchenne and Becker muscular dystrophy. As a new therapeutic landscape emerged, we saw a clear need to combine PRO data with clinician-reported outcomes data. Recent advances in technology around shared data have enabled PPMD to combine these data sets within a centralized data interchange that will benefit the entire community – from patients, to clinicians and scientists exploring therapeutics and treatments. PPMD’s Interchange provides this central repository for all types of Duchenne and Becker data and ‘real world evidence’ to be collected and analyzed,” said Ann Martin, MS, CGC, PPMD’s Vice President of Community Research & Genetic Services. PPMD launched the Interchange in 2018 with Prometheus Research (an IQVIA company), an industry leader in health data informatics. All data is stored securely and in accordance with strict industry standards and patient privacy laws. The Interchange will only allow data to be used for research purposes and to improve care for Duchenne and Becker patients. In an effort to collect clinical data without adding additional burden on patients and families, once a patient consents to participate in PPMD’s EHR Study, the study will automatically collect important information from doctor visits and send it to the Interchange. “Collecting data directly from the EHR capitalizes on recent advances in health data interoperability, positioning patient advocacy groups to gather data more easily, without asking either the clinician or the patient to enter the data themselves. It is faster and more efficient for everyone, with less possibility for errors,” explains Megan Freed, MPH, PPMD’s Director of Data and Technology Strategy. “As physicians treating Duchenne and Becker patients, the more information we have about this rare, multi-system disease, the more informed decisions we can make about clinical care. The ability to view in the Interchange population-level data from our clinic compared to other Duchenne and Becker clinics in the CDCC Network is incredibly valuable to advancing the science,” said Dr. Aravindhan Veerapandiyan, MD, an assistant professor of Pediatrics in the University of Arkansas for Medical Sciences (UAMS) College of Medicine who practices as a child neurologist at Arkansas Children’s Hospital and directs the Certified Duchenne Care Center / Dystrophinopathy Clinic for children with Duchenne and Becker muscular dystrophies. While patients do not need to be registered with The Duchenne Registry to participate in PPMD’s EHR Study, the ultimate goal is to combine this data within the Interchange, building the most comprehensive dataset on Duchenne and Becker muscular dystrophy. Pat Furlong, PPMD’s Founding President & CEO, explains that these data sources fit together like puzzle pieces to create a more complete picture of the natural history of the disorder: “Since a small group of parents and grandparents formed PPMD over 25 years ago, we have believed in the importance of innovation and patient data in the fight to end Duchenne. The Duchenne Registry remains a critical tool in Duchenne research, as it will always be important to get information directly from patients about how they are feeling and what they are able to do. And now, powered with 15 years of robust data thanks to our incredible community, it is ready to expand and evolve. With the addition of EHR data, PPMD’s Interchange will provide a more complete picture of Duchenne and Becker, allowing researchers and clinicians to improve care and develop treatments faster.” Over the coming years, PPMD will invite all CDCCs within its network to participate in the EHR Study. Patients who attend participating CDCCs will be invited to join the study during an upcoming clinic or phone visit. If the patient consents to participate, the clinic will securely push data from the EHR to the Interchange. The data will include diagnosis, medications, laboratory tests, and procedures. Retrospective data will be pushed, and all prospective data will be pushed until the study ends or the patient requests to be withdrawn from the study. To learn more about PPMD’s EHR Study, click here. ABOUT PARENT PROJECT MUSCULAR DYSTROPHY: Duchenne is a fatal genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) fights every single battle necessary to end Duchenne. We demand optimal care standards and ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. We invest deeply in treatments for this generation of Duchenne patients and in research that will benefit future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and won five FDA approvals. Everything we do—and everything we have done since our founding in 1994—helps those with Duchenne live longer, stronger lives. We will not rest until we end Duchenne for every single person affected by the disease. Join our fight against Duchenne at EndDuchenne.org. Follow PPMD on Facebook, Twitter, Instagram, and YouTube.
SOURCE Parent Project Muscular Dystrophy (PPMD) |
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