To identify and evaluate insurance coverage barriers for FDA-approved SMA treatments and supportive care.

Spinal muscular atrophy (SMA) is a neuromuscular disease characterized by progressive muscle weakness and atrophy. Patients and caregivers regularly face insurance-related barriers to accessing FDA-approved treatments and multidisciplinary care. These restrictions cause delays in essential services and may result in negative health outcomes and unnecessary patient and caregiver burden.  

A survey was developed and distributed from August 2024 – October 2024 to adults living with SMA and caregivers of children 17 or younger living with SMA within a non-profit owned membership database.

332 unique responses from adults living with SMA and caregivers were included within this analysis. 54.6% of adults and 54.5% of caregivers reported at least one insurance denial for an FDA-approved treatment for SMA. Socioeconomic and SMA-related demographics were similar between those that experienced denial(s) and those that did not. Of those who experienced insurance denial(s) for SMA treatment, 69.5% had private/employer-based insurance and 23.0% had Medicare coverage. Among those who had not experienced denial(s) 48.3% had private/employer-based insurance and 40.0% had Medicare. Though appeals were often successful (80.9%), treatment delay varied from less than 1 week to more than 8 weeks. Among those that experienced insurance denial(s) 49.1% reported experiencing a barrier to scheduling that was due to their health plan authorization being delayed, compared to 29.7% of those who had not experienced insurance denial(s) for SMA treatment.

This research reveals the impact of insurance restrictions on treatment access for the SMA community. Findings suggest that insurance plans that restrict access to FDA-approved SMA treatments may also include harmful barriers to other SMA-related care. Industry, healthcare providers, and patient advocacy groups may leverage this data to engage payors regarding burdens for the SMA community due to insurance-related barriers.