Article ; Online: Economic evaluation of Motor Neuron Diseases: a nationwide cross-sectional analysis in Germany.
2023 Volume 270, Issue 10, Page(s) 4922–4938
Abstract: Background and objectives: Motor Neuron Diseases (MND) are rare diseases but have a high impact on affected individuals and society. This study aims to perform an economic evaluation of MND in Germany.: Methods: Primary patient-reported data were ... ...
Abstract | Background and objectives: Motor Neuron Diseases (MND) are rare diseases but have a high impact on affected individuals and society. This study aims to perform an economic evaluation of MND in Germany. Methods: Primary patient-reported data were collected including individual impairment, the use of medical and non-medical resources, and self-rated Health-Related Quality of Life (HRQoL). Annual socio-economic costs per year as well as Quality-Adjusted Life Years (QALYs) were calculated. Results: 404 patients with a diagnosis of Amyotrophic Lateral Sclerosis (ALS), Spinal Muscular Atrophy (SMA) or Hereditary Spastic Paraplegia (HSP) were enrolled. Total annual costs per patient were estimated at 83,060€ in ALS, 206,856€ in SMA and 27,074€ in HSP. The main cost drivers were informal care (all MND) and disease-modifying treatments (SMA). Self-reported HRQoL was best in patients with HSP (mean EuroQoL Five Dimension Five Level (EQ-5D-5L) index value 0.67) and lowest in SMA patients (mean EQ-5D-5L index value 0.39). QALYs for patients with ALS were estimated to be 1.89 QALYs, 23.08 for patients with HSP and 14.97 for patients with SMA, respectively. Cost-utilities were estimated as follows: 138,960€/QALY for ALS, 525,033€/QALY for SMA, and 49,573€/QALY for HSP. The main predictors of the high cost of illness and low HRQoL were disease progression and loss of individual autonomy. Conclusion: As loss of individual autonomy was the main cost predictor, therapeutic and supportive measures to maintain this autonomy may contribute to reducing high personal burden and also long-term costs, e.g., care dependency and absenteeism from work. |
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MeSH term(s) | Humans ; Quality of Life ; Cost of Illness ; Cross-Sectional Studies ; Amyotrophic Lateral Sclerosis ; Cost-Benefit Analysis ; Surveys and Questionnaires ; Health Care Costs ; Germany/epidemiology ; Muscular Atrophy, Spinal |
Language | English |
Publishing date | 2023-06-25 |
Publishing country | Germany |
Document type | Journal Article |
ZDB-ID | 187050-6 |
ISSN | 1432-1459 ; 0340-5354 ; 0012-1037 ; 0939-1517 ; 1619-800X |
ISSN (online) | 1432-1459 |
ISSN | 0340-5354 ; 0012-1037 ; 0939-1517 ; 1619-800X |
DOI | 10.1007/s00415-023-11811-1 |
Database | MEDical Literature Analysis and Retrieval System OnLINE |
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