Spinal muscular atrophy: an update for managed care pharmacists

The cost burden of patients with SMA is considerable, and is estimated to be approximately $4 million to $5 million over 10 years in patients with early-onset SMA. This cost is 54.2 times greater than an otherwise healthy population. The utilization of medication, resources, and cost differs between different types of SMA and is more intensive in infantile-onset SMA type 1. Patients often require supportive physical aides, ventilation, and other services to treat sequalae of muscle weakness. Early diagnosis and treatment initiation are necessary to maximize benefit of treatment. Genetic newborn screening has allowed for early diagnosis. With the approval of novel pharmacotherapy options for SMA, timely treatment initiation may help to decrease healthcare burden and costs associated with early-onset SMA. Current options are effective in improving mobility, but maximum benefit has yet to be seen as this population is still growing. Due to the cost of treatment, managed care pharmacists should consider appropriate utilization management and innovative outcomes-based payment models to decrease risk while maximizing outcomes.