{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,16]],"date-time":"2026-10-16T06:11:36Z","timestamp":1792131096590,"version":"4.4.0"},"reference-count":56,"publisher":"Springer Science and Business Media LLC","issue":"10","license":[{"start":{"date-parts":[[2023,6,25]],"date-time":"2023-06-25T00:00:00Z","timestamp":1687651200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2023,6,25]],"date-time":"2023-06-25T00:00:00Z","timestamp":1687651200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"DOI":"10.13039\/501100010275","name":"Deutsche Gesellschaft f\u00fcr Muskelkranke","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100010275","id-type":"DOI","asserted-by":"publisher"}]},{"name":"Medizinische Hochschule Hannover (MHH)"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["J Neurol"],"published-print":{"date-parts":[[2023,10]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec>\n                <jats:title>Background and objectives<\/jats:title>\n                <jats:p>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.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Methods<\/jats:title>\n                <jats:p>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.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Results<\/jats:title>\n                <jats:p>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\u20ac in ALS, 206,856\u20ac in SMA and 27,074\u20ac 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\u20ac\/QALY for ALS, 525,033\u20ac\/QALY for SMA, and 49,573\u20ac\/QALY for HSP. The main predictors of the high cost of illness and low HRQoL were disease progression and loss of individual autonomy.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Conclusion<\/jats:title>\n                <jats:p>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.<\/jats:p>\n              <\/jats:sec>","DOI":"10.1007\/s00415-023-11811-1","type":"journal-article","created":{"date-parts":[[2023,6,25]],"date-time":"2023-06-25T11:01:14Z","timestamp":1687690874000},"page":"4922-4938","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":13,"title":["Economic evaluation of Motor Neuron Diseases: a nationwide cross-sectional analysis in Germany"],"prefix":"10.1007","volume":"270","author":[{"ORCID":"https:\/\/orcid.org\/0009-0007-0981-6486","authenticated-orcid":false,"given":"Felix","family":"Heinrich","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2078-1997","authenticated-orcid":false,"given":"Isabell","family":"Cordts","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0329-5644","authenticated-orcid":false,"given":"Ren\u00e9","family":"G\u00fcnther","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2774-067X","authenticated-orcid":false,"given":"Benjamin","family":"Stolte","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3393-5657","authenticated-orcid":false,"given":"Daniel","family":"Zeller","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carsten","family":"Schr\u00f6ter","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ute","family":"Weyen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2172-7386","authenticated-orcid":false,"given":"Martin","family":"Regensburger","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joachim","family":"Wolf","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7109-2542","authenticated-orcid":false,"given":"Ilka","family":"Schneider","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7364-7791","authenticated-orcid":false,"given":"Andreas","family":"Hermann","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8670-6148","authenticated-orcid":false,"given":"Moritz","family":"Metelmann","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4147-8866","authenticated-orcid":false,"given":"Zacharias","family":"Kohl","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8740-3106","authenticated-orcid":false,"given":"Ralf 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Heinrich reports no disclosures relevant to the manuscript. I. Cordts reports no disclosures relevant to the manuscript. R. G\u00fcnther reports no disclosures relevant to the manuscript. B. Stolte received speaker honoraria from Biogen without relation to ALS. D. Zeller received compensation for participation on an advisory board of Biogen, as well as for consultancy work from Novartis. C. Schr\u00f6ter reports no disclosures relevant to the manuscript. U. Weyen received compensation for activities with Merz and Desitin. M. Regensburger reports no disclosures relevant to the manuscript. J. Wolf reports no disclosures relevant to the manuscript. I. Schneider received personal\/speaker fees from Sanofi-Genzyme, Biogen, and from the German Neuromuscular Society \u201cDeutsche Gesellschaft fuer Muskelkranke\u201d (DGM e.V.) without relation to ALS. A. Hermann received compensation for consultant work for Biogen, Desitin, and Roche. Additionally, A.\u00a0Hermann received research funding from BMBF, GBA-Innovations Fund, Helmholtz Gesellschaft, NOMIS-Foundation, and \u201cHermann und Lilly-Schilling Stiftung fuer medizinische Forschung\u201d im Stifterverband. M. Metelmann reports no disclosures relevant to the manuscript. Z. Kohl reports no disclosures relevant to the manuscript. R.A. Linker received compensation for activities with Biogen, Celgene, Merck, Novartis, and Roche, as well as research support from DFG, Else-Kroener-Fresenius Stiftung, Biogen, and Novartis. J.C. Koch received personal fees from Biogen, Roche, Avexis, Abbvie, and Ipsen, and funding by the \u201cDeutsche Gesellschaft fuer Muskelkranke\u201d (DGM e.V.) outside of the submitted work. F. Radelfahr reports no disclosures relevant to the manuscript. P. Schischlevskij reports no disclosures relevant to the manuscript. E. Sch\u00f6nfelder reports no disclosures relevant to the manuscript. T. Mohajer-Peseschkian reports no disclosures relevant to the manuscript. A. Osmanovic received honoraria as a speaker\/consultant from the German Neuromuscular Society \u201cDeutsche Gesellschaft fuer Muskelkranke\u201d (DGM e.V.), Biogen, and Impulze. T. Klopstock reports no disclosures relevant to the manuscript. J. Dorst reports no disclosures relevant to the manuscript. A.C. Ludolph received honoraria from Desitin, Novartis, Biogen, Roche, and the German Society of Neurology (Deutsche Gesellschaft fuer Neurologie e.V.). O.Sch\u00f6ffski reports no disclosures relevant to the manuscript. M. Boentert received honoraria from UCB Pharma, Desitin, L\u00f6wenstein Medical, Sanofi-Genzyme, and Biogen, as well as financial research support from Sanofi-Genzyme and L\u00f6wenstein Medical. T.\u00a0Hagenacker received honoraria from Biogen, Roche, Novartis, and Alexion without relation to ALS, and received research funding from Biogen, AveXis, and Roche without relation to ALS. M. Deschauer received personal fees as a speaker\/consultant from Biogen, Roche, Sanofi-Genzyme, and Alnylam Pharmaceuticals. P. Lingor received honoraria from ITF-Pharma and Desitin with relation to ALS. S. Petri received honoraria as speaker\/consultant from Biogen GmbH, Roche, Novartis, Teva, Cytokinetics Inc., Desitin, Italfarmaco, Amylyx, and Zambon; and grants from DGM e.V, Federal Ministry of Education and Research, German Israeli Foundation for Scientific Research and Development, EU Joint Program for Neurodegenerative Disease Research. O. Schreiber-Katz\u00a0received honoraria as a speaker\/consultant and\/or funding for travel expenses from the German Neuromuscular Society \u201cDeutsche Gesellschaft fuer Muskelkranke\u201d (DGM e.V.), Novartis, Biogen GmbH, Biermann Verlag GmbH, and\u00a0MK\u2009+\u2009S\u2014Medizin, Kommunikation & Service GmbH.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of interest"}},{"value":"The Ethics Board of Hannover Medical School approved the study (no. 7922_BO_K_2018, date: July and November 2018), as well as the local Ethics Boards of the individual MND Network centers.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval"}},{"value":"Written informed consent was obtained from all participants involved in the study and before study enrolment.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent to participate"}}]}}