Journal Article

Economic evaluation of Motor Neuron Diseases: a nationwide cross-sectional analysis in Germany

Carsten Schröter, Susanne Petri, Felix Heinrich, Zacharias Kohl, René Günther, Erik Schönfelder, Marcus Deschauer, Thomas Klopstock, Benjamin Stolte, Albert C. Ludolph, Pavel Gardt, Andreas Hermann, Ralf A. Linker, Tim Hagenacker, Florentine Radelfahr, Daniel Zeller, Jan Christoph Koch, Oliver Schöffski, Tara Mohajer-Peseschkian, Ilka Schneider, Joachim Wolf, Matthias Boentert, Ute Weyen, Johannes Dorst, Alma Osmanovic, Paul Lingor, Isabell Cordts, Martin Regensburger, Moritz Metelmann, Olivia Schreiber-Katz

2023

Overview

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.

• Publisher: Springer Science and Business Media LLC,Springer Berlin Heidelberg,Springer Nature B.V
• Subject: Amyotrophic Lateral Sclerosis / Autonomy / Cost of Illness / Cost of illness (COI) / Cost-Benefit Analysis / Cost-utilities / Costs / Cross-Sectional Studies / epidemiology [Germany] / Germany / Health Care Costs / Health-related Quality of Life (HRQoL) / Hereditary spastic paraplegia / Humans / Medicine / Medicine & Public Health / Medizin / Motor Neuron Disease (MND) / Motor neuron diseases / Muscular Atrophy, Spinal / Neurology / Neuromuscular diseases / Neuroradiology / Neurosciences / Original Communication / Original Communication ; Motor Neuron Disease (MND) ; Cost of illness (COI) ; Health-related Quality of Life (HRQoL) ; Quality-adjusted life years (QALYs) ; Socio-economic burden ; Cost-utilities ; Medical and Health Sciences ; Economics / Patients / Quality of Life / Quality-adjusted life years (QALYs) / Socio-economic burden / Spinal muscular atrophy / Surveys and Questionnaires