CHICAGO, March 19, 2019 /PRNewswire/ -- Today, Cure SMA
and Parent Project Muscular Dystrophy (PPMD), two of the world's
leading rare disease patient advocacy organizations, announced
their strategic collaboration to advance clinical data aggregation
and reaffirmed their commitment to improving health outcomes for
those affected by spinal muscular atrophy (SMA) and Duchenne
muscular dystrophy (Duchenne). Both prominent organizations have
joined forces with Prometheus Research as their principal
informatics partner, enabling unprecedented levels of collaboration
on data and technology.
The Cure SMA clinical data registry collects electronic health
record based patient data from a growing set of sites in the newly
established Cure SMA Care Center Network; this data is being used
to facilitate a wide range of clinical research and quality
improvement initiatives. Launched in late-2018, the SMA Care Center
Network has quickly grown to include ten geographically diverse
clinical sites nationwide, with plans to add nationwide newborn
screening and more than double in size by the end of 2019. The
primary goals of the SMA Care Center Network are to understand how
life-changing SMA therapies are transforming the experience of SMA
and to identify an evidence-based standard of care that will allow
individuals to receive maximum benefit from these
therapies.
Established in 2007 and including patient-reported data from
nearly 5,000 registrants, PPMD's existing Duchenne Registry is the
largest, most comprehensive patient-reported registry for Duchenne
and Becker muscular dystrophy in the world. Data from The Duchenne
Registry's first decade of success was recently published in a
10-year report to inform clinical researchers and industry sponsors
on patient-reported outcomes. PPMD is now partnering with
Prometheus on a next-generation data infrastructure capable of
enhancing these patient-reports with real world data from clinical
systems (including electronic health records), administrative
claims, and social determinants. Like SMA, PPMD's registry will
integrate with dozens of specialty clinics through their
well-established Certified Duchenne Care Center program.
The Prometheus data solutions for both Cure SMA and PPMD have
been designed to integrate and repurpose data from disparate health
sources to help clinical researchers and industry sponsors address
common challenges, such as monitoring the changing natural history
of each disease and the outcomes these patients experience over
longer periods of time than traditional studies typically examine.
Underpinning these capabilities are Prometheus' nearly 20 years of
clinical research informatics expertise and RexRegistry, the
industry's first agile data hub that creates research-grade data
assets capable of minimizing burden on data partners while
dynamically supporting new combinations of data sources, data uses,
and stakeholders over time. With both organizations now using the
same underlying system, care sites can more effectively and
efficiently participate in these initiatives.
"With new treatments, the experience of SMA is rapidly shifting,
and healthcare providers need to adapt quickly to these changing
needs," said Kenneth Hobby,
president of Cure SMA. "The goal of the SMA Care Center Network is
to develop an evidence-based standard of care that will address
this changing landscape, expand access to approved SMA treatments,
and help accelerate and enhance insurance coverage and
reimbursement."
"Since a small group of parents and grandparents formed PPMD
almost 25 years ago, we have believed in the importance of
innovation and patient data in the fight to end Duchenne," said
Pat Furlong, Founding President
& CEO of PPMD. "We are thrilled to collaborate with Cure SMA,
working side by side to seize the power of patient-reported
outcomes and electronic health records in order to further our
missions to end these devastating diseases," she continued.
"This is a very exciting time in human health, and for rare
disease advocacy specifically," added David
Voccola, cofounder of Prometheus Research. "The advent of
modern data standards is unlocking potentially transformative
collaboration models. Prometheus is thrilled to be supporting these
two remarkable organizations as they lead the way to this promising
new future."
In the rapidly changing world of disease therapies, Cure SMA and
PPMD are steadfastly committed to shared learnings and working to
develop greater collaborations across and within the rare disease
space.
About Cure SMA
Cure SMA is dedicated to the treatment and cure of spinal
muscular atrophy (SMA)—a disease that takes away a person's ability
to walk, eat, or breathe. It is the number one genetic cause of
death for infants.
Since 1984, we've directed and invested in comprehensive
research that has shaped the scientific community's understanding
of SMA. We are currently on the verge of breakthroughs that will
strengthen bodies, extend life, and lead to a cure.
We have deep expertise in every aspect of SMA—from the
day-to-day realities to the nuances of care options—and until we
have a cure, we'll do everything we can to support and advocate for
those affected by the disease. Learn more about how you can help us
reach a treatment and cure at www.cureSMA.org and follow Cure SMA
on Facebook, Twitter, Instagram, and YouTube.
About Parent Project Muscular Dystrophy
Duchenne is a fatal genetic disorder that slowly robs people 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 people
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 people with Duchenne live longer, stronger lives. We
will not rest until every person 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.
Follow PPMD on Facebook, Twitter, and YouTube.
About Prometheus Research
Prometheus Research is the principal informatics partner to
renowned health organizations engaged in ambitious translational
research endeavors and quality improvement initiatives. Our agile
data platform, RexRegistry, empowers our partners to acquire,
integrate, and repurpose disparate sources of health data to
accelerate scientific discovery and patient access to new
therapies. Together with our consulting services, Prometheus helps
forward-thinking institutions build data assets today that will
continue advancing the state of care for generations to
come.
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SOURCE Cure SMA