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High Healthcare Resource Use in Hospitalized Patients with a Diagnosis of Spinal Muscular Atrophy Type 1 (SMA1):
Jessica Cardenas1, Melissa Menier1, Marjet D Heitzer1
1Department of Clinical Development, AveXis, Inc, 2275 Half Day Road, Suite 200, Bannockburn, IL, 60015, USA.
Insights
Spinal muscular atrophy type 1 (SMA1) hospitalizations incur significantly higher costs and resource use than other conditions. Previous cost estimates likely underestimate the true financial burden of SMA1 due to frequent readmissions.
Area of Science:
- Pediatric Health Economics
- Neuromuscular Disorders
- Healthcare Resource Utilization
Background:
- Spinal muscular atrophy (SMA) leads to high healthcare resource use (HRU) due to complications from muscle atrophy.
- Existing cost estimates may understate direct costs for spinal muscular atrophy type 1 (SMA1).
Purpose of the Study:
- To analyze healthcare resource utilization (HRU) in hospitalizations for SMA1.
- To compare HRU for SMA1 hospitalizations with those for complex chronic conditions (CCC) and no CCC.
Main Methods:
- Retrospective analysis of a subset of the 2012 Kids' Inpatient Database (KID).
- Comparison of hospitalizations for children aged <3 years across three groups: SMA1, other CCC, and no CCC.
Main Results:
- SMA1 admissions had significantly higher mean total charges (US$150,921) versus no CCC (US$19,261).
- SMA1 admissions involved more procedures (81.9%) and longer stays (15.1 days) compared to no CCC admissions.
- SMA1 hospitalizations incurred higher costs per admission than both no CCC and other CCC groups.
Conclusions:
- Average total charges for SMA1 admissions exceed those for the no CCC group.
- Frequent hospitalizations for SMA1 suggest previous cost estimates significantly underestimate direct costs.
- Further research is needed on indirect costs and societal impacts of SMA1.
Background:
Patients with spinal muscular atrophy (SMA) have high healthcare resource use (HRU) due to respiratory and nutritional complications resulting from progressive muscle atrophy. While previous studies estimate the direct costs to be US$113,000 to US$121,682 per year in the US, they potentially understate costs for type 1 SMA (SMA1). This study analyzed HRU in hospitalizations with a diagnosis of SMA1 and compared it with hospitalizations with complex chronic conditions (CCC) other than SMA1 or those with no CCC.
Methods:
This retrospective analysis of a defined subset of the 2012 Kids' Inpatient Database (KID) compared a nationally estimated number of hospitalizations of children (aged < 3 years) categorized into three groups: (1) SMA1 (n = 237 admissions), (2) no CCC (n = 632,467 admissions), and (3) other CCC (n = 224,953 admissions).
Results:
Mean total charges were higher for SMA1 admissions compared with admissions with no CCC (US$150,921 vs US$19,261 per admission, respectively; costs: US$50,190 vs $5862 per admission, respectively; both p < 0.0001). A larger proportion of SMA1 admissions were billed for one or more procedure codes (81.9%) than in the no CCC group (39.4%) or other CCC group (70.1%; both p ≤ 0.0003). SMA1 admissions had a longer length of stay compared with admissions with no CCC (15.1 vs 3.4, respectively; p < 0.0001).
Conclusions:
The average total charges for a single SMA1 admission were higher than those of the no CCC group. Because most infants with SMA1 require multiple hospitalizations per year, previous estimates may dramatically underestimate the direct costs associated with HRU. Further studies are required to determine the indirect costs and societal impacts of SMA1.