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Abstract Details

The Economic Burden of Spinal Muscular Atrophy Patients in a Commercially-Insured Population in the United States
Child Neurology and Developmental Neurology
P1 - Poster Session 1 (5:30 PM-6:30 PM)
7-054
To assess the economic burden associated with the treatment of Spinal Muscular Atrophy (SMA) patients in the US. 
In addition to its high psychosocial impact, SMA can place a considerable economic burden on patients and their caregivers. However, despite the high clinical burden, limited information exists on the economic burden of the disease.
A retrospective analysis of Truven MarketScan® Commercial and Medicare Supplemental Databases was conducted (January 2012 – March 2017) to identify SMA patients by ≥2 diagnosis codes (ICD-9: 335.0, 335.1x; ICD-10: G12.0, G12.1, G12.8, G12.9) on inpatient or outpatient medical claims >30 days apart. Patients were categorized in age groups, based on the index date of first claim (<2 years, 2-18 years, and >18 years). Patients were followed from index date until the end of continuous enrollment, or the end of the study period, whichever occurred earlier. All-cause and disease-specific healthcare resource utilization, and associated direct costs were captured from the claims databases, and were annualized. Costs were adjusted to 2017 US dollars. 
A total of 1,120 patients were included. Overall, 60.6% patients visited a neurologist or pulmonologist, 73.5% utilized home health services, 51.8% had emergency room visits, and 41.1% were hospitalized. Procedures with the highest utilization across the cohort were gastrostomy (21.5%) and tracheostomy (17.8%). Durable medical equipment with frequent use included CPAP/BiPAP (28.1%), oxygen machines (25.1%), and suction devices (23.0%). Utilization of healthcare and supportive care resources was higher among pediatric patients. Mean (SD) all-cause annual direct healthcare costs were $65,490 ($135,268), and were highest for SMA patients aged <2 years ($159,227 [$313,121]). Outpatient medical services accounted for 52.8% of costs across the study cohort. Disease-specific costs accounted for 49.4% of total costs, which on average (SD) totaled $32,332 ($93,687).
In a commercially-insured population, SMA patients had high economic burden driven by outpatient medical spend.  
Authors/Disclosures

PRESENTER
No disclosure on file
No disclosure on file
No disclosure on file
No disclosure on file
Kristen Johnson, PhD (Novartis) No disclosure on file