Cure SMA and Parent Project Muscular Dystrophy Announce Strategic Collaboration with Prometheus Research

Wednesday, Mar 20, 2019

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.



Other News