Parent Project Muscular Dystrophy launches Electronic Health Record (EHR) Study

Parent Project Muscular Dystrophy has announced it will collect RWD directly from electronic health records to complement its established registry of patient-reported outcomes for building the most comprehensive dataset on Duchenne and Becker muscular dystrophy. 

Non-profit patient advocacy group Parent Project Muscular Dystrophy (PPMD) has announced the launch of its Electronic Health Record (EHR) Study. People with Duchenne and Becker muscular dystrophy are being enrolled at PPMD’s Certified Duchenne Care Centers (CDCCs) so that RWD from their EHRs will automatically upload to the PPMD Duchenne Outcomes Research Interchange. In this Interchange, the clinician-recorded EHR data will be combined with PPMD’s established resource of patient-reported outcomes (PRO) in the organization’s Duchenne Registry. Together these complementary RWD will build the most comprehensive dataset to help clinicians and researchers better understand and treat Duchenne and Becker muscular dystrophy.  

PPMD is a parent-founded group that is leading the fight to end muscular dystrophy. As well as investing deeply in treatments for the current generation of people living with Duchenne — they have been involved in five FDA approvals — they invest heavily in research that will benefit future generations. 

Pat Furlong, PPMD’s Founding President and CEO, explained: “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.” 

PPMD launched the Duchenne Outcomes Research Interchange in 2018 with Prometheus Research, an IQVIA company industry leader in health data informatics. PPMD’s VP of Community Research and Genetic Services, Ann Martin, said: “For the last several years, PPMD has been expanding and enhancing the Duchenne Registry, our comprehensive 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.” 

In an effort to collect clinical data to inform care and research without adding additional burden on patients and families, PPMD’s EHR Study is set up such that important information is automatically uploaded from a person’s EHR to the Interchange once they have consented to participate in the study. The data collected will include diagnosis, medications, laboratory tests, and procedures. Retrospective data will be uploaded, and prospective data will be gathered until the study ends or the patient requests to be withdrawn from the study. 

“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,” detailed Megan Freed, PPMD’s Director of Data and Technology Strategy.  

Arkansas Children’s Hospital is the first CDCC to take part in PPMD’s EHR Study, with initial patients enrolled. Several more CDCCs across the US are in the process of joining the study and PPMD will invite all centers within its network to participate over the coming years. “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, child neurologist and Director of the CDCC for children with Duchenne and Becker at Arkansas Children’s Hospital, and Assistant Professor of Pediatrics in the University of Arkansas for Medical Sciences College of Medicine.