DuchenneConnect Approved for Funding Award from Patient-Centered Outcomes Research Institute to Become Integral Part of New National Clinical Research Network

19 Feb, 2014, 12:53 ET from Parent Project Muscular Dystrophy

HACKENSACK, N.J., Feb. 19, 2014 /PRNewswire-USNewswire/ -- DuchenneConnect, a program of Parent Project Muscular Dystrophy (PPMD), is thrilled to announce receiving a funding award of $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.  

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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, PhD at Geisinger Health Systems; Stanley Nelson, MD, Nancy Halnon, MD and Richard Wang, PhD 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. 

Nationwide benefits: 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.

DuchenneConnect benefits: Ms. Peay is excited about what this award means for DuchenneConnect: "I'm so proud of what we have accomplished with DuchenneConnect in the last six years, but we are ready to take this registry to the next level. With the funding from this award and with our great collaborators at UCLA, Geisinger, and PatientCrossroads, we are excited to enter our next period of growth with a focus on improving the registry experience, increasing our enrollment, exploring information needs of our community, and working to address those needs through collaborative research that is efficient and cost-effective. We are extremely enthusiastic about maturing the registry with input and support from the PCORnet partners, while concurrently sharing our experiences (and ultimately data sets) with the Network partners. 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.

PCORI has awarded a total of $464.4 million since it began funding CER in 2012. For more information about PCORI funding, visit http://pcori.org/funding-opportunities. For more information about PCORnet, visit http://www.pcornet.org.  To learn more about DuchenneConnect, visit http://duchenneconnect.org.

About Parent Project Muscular Dystrophy
Duchenne is a fatal genetic disorder that slowly robs young men of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne.

We invest deeply in treatments for this generation of young men affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite, and educate the global Duchenne community.

Everything we do—and everything we have done since our founding in 1994—helps boys with Duchenne live longer, stronger lives. We will not rest until every young man has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne.

About DuchenneConnect
Early in 2007, a group of thought leaders in the Duchenne muscular dystrophy community began discussing the need for a new kind of resource that would connect and serve the needs of the entire community. What they envisioned was a central hub that would bring together those living with the disease, along with their families, friends and caregivers, to connect them with medical research, clinical care, clinical trials and each other. At the same time, it would also be a resource for researchers and industries with an interest in Duchenne, allowing access to aggregated, de-identified information provided by patients and their families—information that could prove vital to advances in care and treatment. This resource, a breakthrough multidirectional information portal for Duchenne and Becker muscular dystrophy, would help connect these stakeholders, while providing them with resources never before available in one place. Today, the result of this endeavor is DuchenneConnect, a robust and cutting-edge website and registry.

The Patient-Centered Outcomes Research Institute (PCORI) is an independent, non-profit organization authorized by Congress in 2010. Its mission is to fund research that will provide patients, their caregivers and clinicians with the evidence-based information needed to make better-informed health care decisions. PCORI is committed to continuously seeking input from a broad range of stakeholders to guide its work. More information is available at www.pcori.org.

SOURCE Parent Project Muscular Dystrophy