OBJECTIVES: Cure SMA is a patient advocacy organization that provides support for spinal muscular atrophy (SMA) research and care. In 2018, Cure SMA created a registry to inform development of evidence-based standards of care (SOC). The SMA Clinical Data Registry (CDR) ingests monthly submissions of structured electronic medical record (EMR) data from 23 participating U.S. clinical care sites into a shared data mart. High-priority data unavailable from discrete EMR fields are collected via linked electronic case report forms. The CDR comprises data from >1,050 individuals with SMA and is among the largest EMR-based SMA registries worldwide. Ensuring high-quality data across participating institutions is challenging, given heterogeneity in data availability, reliability, infrastructure, submission methods, and extraction/mapping. As an important use of the CDR is to inform SOC development, processes that ensure high confidence in its data are critical. Therefore, Cure SMA aims to develop a customized quality framework to ensure data quality to inform SOC development and enable additional use cases.

METHODS: The CDR quality framework pulls from existing published frameworks, follows “ALCOA+” (Attributable, Legible, Contemporaneous, Original, and Accurate) principles, and has two components: (1) foundational checks that focus on data conformance, plausibility, and completeness; and (2) field- and patient-level checks within specific populations that confirm data align with anticipated patterns. The framework provides quality checks at various levels (e.g., registry, site, table, field), including comparisons to uploads from previous months to enable further identification of inconsistencies.

RESULTS: The framework criteria and results from the CDR will be presented.

CONCLUSIONS: Real-world data (RWD) are increasingly relied upon to inform all aspects of medical care and outcomes. The CDR quality framework will ensure relevant and extensive RWD of high quality and completeness are available to create evidence for future SOC guidelines and other use cases, which collectively is expected to improve care for individuals with SMA.