THE ECONOMIC BURDEN ON FAMILIES OF CHILDREN WITH TYPE 1 SPINAL MUSCULAR ATROPHY IN BRAZIL
Author(s)
André Soares Motta-Santos, PhD1, Kenya Valéria Micaela de Souza Noronha, PhD2, Carla de Barros Reis, MSc2, Daniela Almeida Freitas, MSc, PhD3, Lélia Maria de Almeida Carvalho, MSc4, Lívio Matheus Oliveira da Silva, MSc5, Ney Cristian Amaral Boa-Sorte, PhD5, Juliana Gurgel Giannetti, PhD6, Mônica Viegas Andrade, PhD2.
1PhD Candidate, Universidade Federal de Minas Gerais and University of Southern Queensland, Belo Horizonte, Brazil, 2Department of Economics, Universidade Federal de Minas Gerais, Belo Horizonte, Brazil, 3Universidade José do Rosário Vellano, Belo Horizonte, Brazil, 4Center for Health Technology Assessment, Hospital das Clínicas da Universidade Federal de Minas Gerais, Belo Horizonte, Brazil, 5Health Technology Assessment Centre, Hospital Universitário Prof. Edgar Santos da Universidade Federal da Bahia, Salvador, Brazil, 6Department of Pediatrics, Universidade Federal de Minas Gerais, Belo Horizonte, Brazil.
1PhD Candidate, Universidade Federal de Minas Gerais and University of Southern Queensland, Belo Horizonte, Brazil, 2Department of Economics, Universidade Federal de Minas Gerais, Belo Horizonte, Brazil, 3Universidade José do Rosário Vellano, Belo Horizonte, Brazil, 4Center for Health Technology Assessment, Hospital das Clínicas da Universidade Federal de Minas Gerais, Belo Horizonte, Brazil, 5Health Technology Assessment Centre, Hospital Universitário Prof. Edgar Santos da Universidade Federal da Bahia, Salvador, Brazil, 6Department of Pediatrics, Universidade Federal de Minas Gerais, Belo Horizonte, Brazil.
OBJECTIVES: Spinal muscular atrophy (SMA) is a rare, progressive neuromuscular disease that severely impacts patients and their families. Although Brazil provides subsidies for high-cost therapies, families caring for children with type I SMA still face substantial financial hardship. This study aims to estimate the household-level economic burden of SMA in Brazil, highlighting the hidden and catastrophic costs borne by families.
METHODS: We conducted a cross-sectional study with face-to-face interviews administered by trained healthcare professionals using a structured instrument. Data collection included caregivers’ intangible and indirect costs, household direct medical costs, and direct non-medical costs. Comparative analyses employed the Student’s t-test and Kruskal-Wallis test for continuous variables, and the χ2 test for categorical variables. All costs were converted to PPP-USD, and analyses were performed in R.
RESULTS: A total of 43 patients from 42 households were included. Caregivers experienced a measurable loss in health-related quality of life (utility: 0.957 vs. 0.846, p<0.10) and reduced income (USD1,151 vs. USD566 monthly, p<0.10) after the child’s birth. Major cost drivers included special diets (USD868.5/month), legal representation (USD13,401 total), and home adaptations (USD10,907 total). Over two years, the cumulative household cost reached USD139,109.6, despite government coverage of direct medical treatment.
CONCLUSIONS: SMA imposes a catastrophic and multidimensional economic burden on families, driven primarily by non-medical and indirect costs. These findings underscore the urgent need for comprehensive support policies that go beyond subsidizing medicines to protect families from financial devastation.
METHODS: We conducted a cross-sectional study with face-to-face interviews administered by trained healthcare professionals using a structured instrument. Data collection included caregivers’ intangible and indirect costs, household direct medical costs, and direct non-medical costs. Comparative analyses employed the Student’s t-test and Kruskal-Wallis test for continuous variables, and the χ2 test for categorical variables. All costs were converted to PPP-USD, and analyses were performed in R.
RESULTS: A total of 43 patients from 42 households were included. Caregivers experienced a measurable loss in health-related quality of life (utility: 0.957 vs. 0.846, p<0.10) and reduced income (USD1,151 vs. USD566 monthly, p<0.10) after the child’s birth. Major cost drivers included special diets (USD868.5/month), legal representation (USD13,401 total), and home adaptations (USD10,907 total). Over two years, the cumulative household cost reached USD139,109.6, despite government coverage of direct medical treatment.
CONCLUSIONS: SMA imposes a catastrophic and multidimensional economic burden on families, driven primarily by non-medical and indirect costs. These findings underscore the urgent need for comprehensive support policies that go beyond subsidizing medicines to protect families from financial devastation.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
EE380
Topic
Economic Evaluation
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
SDC: Neurological Disorders, SDC: Pediatrics, SDC: Rare & Orphan Diseases