Economic evaluation of Motor Neuron Diseases: a nationwide cross-sectional analysis in Germany.
Cost of illness (COI)
Cost-utilities
Health-related Quality of Life (HRQoL)
Motor Neuron Disease (MND)
Quality-adjusted life years (QALYs)
Socio-economic burden
Journal
Journal of neurology
ISSN: 1432-1459
Titre abrégé: J Neurol
Pays: Germany
ID NLM: 0423161
Informations de publication
Date de publication:
Oct 2023
Oct 2023
Historique:
received:
19
03
2023
accepted:
07
06
2023
revised:
06
06
2023
medline:
21
9
2023
pubmed:
25
6
2023
entrez:
25
6
2023
Statut:
ppublish
Résumé
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. 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. 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. 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.
Sections du résumé
BACKGROUND AND OBJECTIVES
OBJECTIVE
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
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
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
CONCLUSIONS
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.
Identifiants
pubmed: 37356024
doi: 10.1007/s00415-023-11811-1
pii: 10.1007/s00415-023-11811-1
pmc: PMC10511618
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
4922-4938Informations de copyright
© 2023. The Author(s).
Références
Neuromuscul Disord. 2018 Feb;28(2):103-115
pubmed: 29290580
J Neurol Sci. 1999 Oct 31;169(1-2):13-21
pubmed: 10540002
Cerebellum. 2017 Apr;16(2):525-551
pubmed: 27271711
Drugs. 2019 Jul;79(11):1255-1262
pubmed: 31270752
Orphanet J Rare Dis. 2014 Dec 18;9:210
pubmed: 25519771
Brain Sci. 2021 Mar 14;11(3):
pubmed: 33799476
J Neurol Neurosurg Psychiatry. 2022 May 16;:
pubmed: 35577511
Muscle Nerve. 2021 May;63(5):668-677
pubmed: 33501671
Gesundheitswesen. 2015 Jan;77(1):53-61
pubmed: 25025287
Eur J Neurol. 2012 Jan;19(1):168-71
pubmed: 21631647
Amyotroph Lateral Scler Frontotemporal Degener. 2013 Jan;14(1):20-5
pubmed: 22871079
N Engl J Med. 2020 Jul 9;383(2):109-119
pubmed: 32640130
Muscle Nerve. 2014 Mar;49(3):431-8
pubmed: 23836444
J Neurol. 2017 Apr;264(4):749-757
pubmed: 28220290
J Mark Access Health Policy. 2021 Feb 28;9(1):1889841
pubmed: 33708361
Value Health. 2019 Nov;22(11):1257-1265
pubmed: 31708062
Neurology. 2019 Apr 23;92(17):e2027-e2037
pubmed: 30918088
J Clin Neuromuscul Dis. 2014 Dec;16(2):104-5
pubmed: 25415523
Brain Sci. 2021 Jun 04;11(6):
pubmed: 34200087
N Engl J Med. 2020 Sep 3;383(10):919-930
pubmed: 32877582
Gesundheitswesen. 2005 Oct;67(10):736-46
pubmed: 16235143
Orphanet J Rare Dis. 2017 Jul 4;12(1):124
pubmed: 28676062
Int J Environ Res Public Health. 2020 Dec 02;17(23):
pubmed: 33276656
Gesundheitswesen. 2010 Dec;72(12):917-33
pubmed: 20865653
Orphanet J Rare Dis. 2016 May 04;11(1):58
pubmed: 27145956
J Neuromuscul Dis. 2021;8(4):553-568
pubmed: 33749617
Front Neurol. 2022 Apr 21;13:864339
pubmed: 35528743
Eur J Neurol. 2012 Mar;19(3):360-75
pubmed: 21914052
Pharmacoeconomics. 2019 Jun;37(6):845-865
pubmed: 30714083
Qual Life Res. 2011 Dec;20(10):1727-36
pubmed: 21479777
Health Qual Life Outcomes. 2021 Jul 20;19(1):181
pubmed: 34284776
Value Health. 2012 Jul-Aug;15(5):708-15
pubmed: 22867780
Am J Med. 2019 Jan;132(1):32-37
pubmed: 30075105
Cost Eff Resour Alloc. 2020 Oct 06;18:41
pubmed: 33041673
Neuromuscul Disord. 2018 Mar;28(3):197-207
pubmed: 29305137
Drugs. 2020 Nov;80(17):1853-1858
pubmed: 33044711
Brain. 2012 Mar;135(Pt 3):847-52
pubmed: 22271664
Acta Neuropathol. 2013 Sep;126(3):307-28
pubmed: 23897027
Neuroepidemiology. 2014;42(3):174-83
pubmed: 24603320
Drugs. 2017 Mar;77(4):473-479
pubmed: 28229309
Int J Surg. 2014 Dec;12(12):1495-9
pubmed: 25046131
Orphanet J Rare Dis. 2020 Jun 12;15(1):149
pubmed: 32532288
Orphanet J Rare Dis. 2020 Aug 24;15(1):217
pubmed: 32838797
Neurol Sci. 2021 Mar;42(3):883-894
pubmed: 33439395
Amyotroph Lateral Scler Frontotemporal Degener. 2014 Jun;15(3-4):279-84
pubmed: 24720420