Cumulative Inpatient Costs Per Child With SMA Type 1 Top $2.3 Million

Without disease-modifying treatment, mortality rates and health care costs for pediatric patients with spinal muscular atrophy (SMA) are extremely high, according to study results published in The European Journal of Health Economics.

“As expected, the costs are highest for patients with SMA type 1, the most severe form of the disease,” wrote a research team from the University of Hong Kong. “The 18-year cumulative inpatient costs in Hong Kong for identified cases of SMA type 1 exceed HK$18,500,000 (US$2,371,795) per patient. By comparison, the 18-year cumulative costs for SMA type 2 and SMA type 3 per patient were 17.5% and 1.7% of that for SMA type 1.”

The study include 71 patients with SMA who were born in Hong Kong in the year 2000 or later. Researchers followed patients until they died, left Hong Kong, reached 18 years, or initiated disease-modifying treatment.

Over a median follow-up of 6 years, the overall incidence rate of death was 5.422 per 100 person-years. Among the deaths, 67.7% occurred in patients with SMA type 1 and 11% in patients with SMA type 2. The median age of death was 0.8 years in patients with SMA type 1 and 10.9 years in patients with SMA type 2. No deaths were recorded in patients with SMA type 3, according to the study.

Cumulative direct medical costs averaged US$935,570 per patient overall. By SMA type, mean direct medical costs per patient were US$2,393,250 for SMA type 1, US$413,165 for SMA type 2, and US$40,735 for SMA type 3.

“These findings provide compelling and cost-effective evidence that support newborn screening for SMA…,” researchers wrote. “Through newborn screening and early disease-modifying treatments, the extremely high SMA-associated health care costs can be effectively minimized, and the significant disability and high mortality of the disease can also be prevented.”

Reference:
Chan SHS, Wong CKH, Wu T, et al. Significant healthcare burden and life cost of spinal muscular atrophy: real-world data. Eur J Health Econ. Published online November 20, 2022. doi:10.1007/s10198-022-01548-5