Cure SMA Patient Data Collection: Comparing the Cure SMA Clinical Data Registry, Membership Database and US SMA Population


Clinical Management

Poster Number: Virtual


Lisa Belter, MPH, Cure SMA, Sarah Whitmire, MS, Cure SMA, Alesa Monk, MBA, Cure SMA, Mary Schroth, MD, Cure SMA

Cure SMA, a patient advocacy organization that provides support and funding for care and treatment of spinal muscular atrophy (SMA), maintains a membership database that is one of the largest SMA patient-reported data repositories worldwide, with 9431 individuals. In addition, the Clinical Data Registry (CDR), is comprised of electronic medical record (EMR) data, representing 621 individuals from 19 care sites, as well as clinician-entered survey data, developed to gather additional metrics not routinely found in discrete EMR data fields. Here we evaluated both Cure SMA-managed databases to determine whether they are representative of the full SMA population.

A comparative analysis was performed to evaluate similarities/differences between the membership database, CDR, and the full US SMA population, based on published literature and US Census data. The breakdowns of sex, race/ethnicity, age, and treatment status with an SMA disease modifying therapy (DMT) were compared. Patients with an unknown SMA type or non-5Q SMA were removed from the analysis, along with international and deceased patients. The breakdown of males was 46.5%, 51.4% and 49.0% for the membership database, CDR, and US SMA population, respectively. There was a greater percentage of Caucasians in the membership database compared with the CDR and US SMA populations: 75.2% vs 62.4% and 70.0%, respectively. There was a greater proportion of children (under the age of 18) in the CDR vs the membership database and the US SMA populations: 73.1% vs 51.1% and 43.2%. Finally, the CDR reported greater use of DMT’s 82.7% vs 62.0% that were estimated in the US SMA population. Additional analyses will be presented.

Exploring reasons for differences in the three data sources will help Cure SMA adjust outreach and support underserved populations.

Thank you to the Care Center Network.

This work was funded by the Cure SMA Real World Collaboration with members Biogen, Novartis Gene Therapies, and Genentech.