Cure SMA is pleased to announce the launch of an expanded Phase 3 of the Real World Evidence Collaboration. The collaboration was established to leverage the experience, expertise and resources of pharmaceutical and biotechnology companies and nonprofit organizations involved in development of SMA therapeutics to guide the future direction of real world evidence collection and use in SMA. Current members include Biogen, Genentech/Roche, and Novartis Gene Therapies.
The primary goal of the Real World Evidence Collaboration is to improve the quality and volume of real world data collection in SMA. Activities include supporting the infrastructure of the Cure SMA Clinical Data Registry (CDR), and the continued expansion of the SMA Care Center Network (CCN).
Since 2018, Cure SMA has partnered with hospitals across the U.S. with the goal to improve healthcare for people with SMA. Every CCN site submits consented patient information and data to the CDR. The CDR is an institutional review board governed database of healthcare information about individuals living with SMA. Data comes from electronic medical records at CCN sites. This real world data provides the core for determining what healthcare should look like for every person with SMA. In collaboration with healthcare providers and people with SMA, Cure SMA will study this CDR data to drive care improvements throughout the community.
Real World Evidence Collaboration Phase 3 Initiatives
As the Real World Evidence Collaboration embarks on its third year, we will build upon previous achievements to improve real world evidence data collection via the CDR, study the characteristics of Care Centers within the CCN to understand the care delivery landscape for SMA, and work to enhance patient engagement across the sites. The collaboration is sub-divided into “Topic Groups” each with a particular focus and specific goals. Every year, each Topic Group outlines the projects it will complete to achieve those goals. A description of the Phase 3 goals and priorities for each Topic Group are provided below; please click each Topic Group name to expand the content.
Real World Evidence Collaboration Phase 2 Achievements
During Phase 2, the Real World Evidence Collaboration drove improvements to real world data collection for the SMA community. The Collaboration’s accomplishments include:
- Conducting a CDR gaps assessment to identify opportunities for continued data improvement
- Establishment of goals to support the improvement of quality metrics in the CDR
- Refinement of the Cure SMA Newborn Screening Registry survey to improve participant experience and allow for more efficient longitudinal data capture
- Development of a survey to better understand the real-world structural components of SMA care delivery within the Care Center Network
About the Real World Evidence Collaboration
The Cure SMA Real World Evidence Collaboration was established to leverage the experience, expertise and resources of pharmaceutical and biotechnology companies and nonprofit organizations involved in development of SMA therapeutics to guide the future direction of real world evidence collection and use in SMA. Current members include Biogen, Genentech/Roche, and Novartis Gene Therapies.