Abstract
BackgroundThis study aimed at analyzing the economic burden and disease-specific health-related quality of life (HRQOL) of patients with spinal muscular atrophy (SMA) in Germany. SMA is a so far non-curable neuromuscular disease of the anterior nerve cells that causes high rates of morbidity and mortality.MethodsIn a cross-sectional study we analyzed the cost of illness (COI) and factors that influence the direct, indirect and informal care costs of affected patients and their families by using standardized, self-developed questionnaires. We used the PedsQL™© Measurement Model to analyze the disease-specific HRQOL of patients.ResultsOne hundred eighty nine patients with SMA types I to III aged <1 to 73 years were enrolled. The average annual COI was estimated at €70,566 per patient in 2013. The highest cost resulted in SMA I with significant lower costs for the milder phenotypes. Inversely, the self-estimated HRQOL increased from SMA I to SMA III. Major cost drivers were informal care cost and indirect cost incurred by patients and their caregivers.ConclusionsAlthough SMA requires high standards of care, there has been a distinct lack of health services research on SMA. Accordingly, our results significantly contribute to a more comprehensive insight into the current burden of SMA and quality of life status as related to SMA health services in Germany. In the light of innovative therapeutic interventions, our results suggest a notable potential for a reduction in overall COI and improvement of HRQOL if the therapeutic intervention leads to a less severe course of the disease.
Highlights
This study aimed at analyzing the economic burden and disease-specific health-related quality of life (HRQOL) of patients with spinal muscular atrophy (SMA) in Germany
In the light of innovative therapeutic interventions, our results suggest a notable potential for a reduction in overall cost of illness (COI) and improvement of HRQOL if the therapeutic intervention leads to a less severe course of the disease
Our study provides a detailed analysis of the cost of illness (COI) of Spinal muscular atrophy (SMA), differentiating between the SMA subtypes in Germany
Summary
This study aimed at analyzing the economic burden and disease-specific health-related quality of life (HRQOL) of patients with spinal muscular atrophy (SMA) in Germany. SMA is a so far non-curable neuromuscular disease of the anterior nerve cells that causes high rates of morbidity and mortality. Spinal muscular atrophy (SMA) is an autosomal recessive, inherited neuromuscular disease that affects the spinal anterior nerve cells. It leads to predominantly proximal muscle and diaphragm weakness and paralysis, along with respiratory distress, progressive disability, reduced working capacity and high health care needs. SMA is so far not curable; and patients need long-term symptomatic multidisciplinary medical care to maintain mobility, independency, ventilation and nutrition [5]. To be able to compare health care expenditures for the current symptomatic treatment and care with costs of future curative therapies, cost evaluations
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