Significant healthcare burden and life cost of spinal muscular atrophy: real-world data

Sophelia H. S. Chan (), Carlos K. H. Wong (), Tingting Wu, Wilfred Wong, Michael K. L. Yu, Ivan C. H. Au and Godfrey C. F. Chan
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Sophelia H. S. Chan: The University of Hong Kong
Carlos K. H. Wong: The University of Hong Kong
Tingting Wu: The University of Hong Kong
Wilfred Wong: The University of Hong Kong
Michael K. L. Yu: The University of Hong Kong
Ivan C. H. Au: The University of Hong Kong
Godfrey C. F. Chan: The University of Hong Kong

The European Journal of Health Economics, 2023, vol. 24, issue 8, No 10, 1373-1382

Abstract: Abstract Objectives The aim of this study is to quantify the mortality rate, direct healthcare costs, and cumulative life costs of pediatric patients with spinal muscular atrophy (SMA) type 1, type 2, and type 3 born in Hong Kong. Methods Data were collected from genetically confirmed SMA patients born in or after 2000 from the Hospital Authority medical database. Patients were followed up from birth until they died, left Hong Kong, reached 18 years, or initiated disease-modifying treatment. Study outcomes included incidence risks of mortality, cumulative direct medical costs—attendances of special outpatient clinics, emergency department, allied health services, and mean length of stay in hospitals over time. Total direct medical costs were calculated as unit costs multiplied by utilization frequencies of corresponding healthcare services at each age. Results Seventy-one patients with SMA were included. Over a median follow-up period of 6 years, the overall incidence rate of death was 5.422/100 person-years (95%CI 3.542–7.945/100 person-years). 67.7% and 11% of deaths occurred in SMA1 and SMA2 groups, respectively. The median age of death was 0.8 years in SMA1 and 10.9 years in SMA2. The mean cumulative direct medical costs in overall SMA, SMA1, SMA2 and SMA3 groups per patient were US$935,570, US$2,393,250, US$413,165, and US$40,735, respectively. Interpretation: Our results confirmed a significantly raised mortality and extremely high healthcare burden for patients with SMA especially SMA type 1 and 2 without disease-modifying treatment. Study evaluating health and economic impact of newborn screening and early treatment is needed.

Keywords: Spinal muscular atrophy; Healthcare burden; Mortality; Newborn screening; Disease modifying treatment (search for similar items in EconPapers)
Date: 2023
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DOI: 10.1007/s10198-022-01548-5

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