{Reference Type}: Journal Article
{Title}: Economic evaluation of Motor Neuron Diseases: a nationwide cross-sectional analysis in Germany.
{Author}: Heinrich F;Cordts I;Günther R;Stolte B;Zeller D;Schröter C;Weyen U;Regensburger M;Wolf J;Schneider I;Hermann A;Metelmann M;Kohl Z;Linker RA;Koch JC;Radelfahr F;Schönfelder E;Gardt P;Mohajer-Peseschkian T;Osmanovic A;Klopstock T;Dorst J;Ludolph AC;Schöffski O;Boentert M;Hagenacker T;Deschauer M;Lingor P;Petri S;Schreiber-Katz O;
{Journal}: J Neurol
{Volume}: 270
{Issue}: 10
{Year}: 2023 Oct 25
{Factor}: 6.682
{DOI}: 10.1007/s00415-023-11811-1
{Abstract}: <B>OBJECTIVE: </B>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.<BR><B>METHODS: </B>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.<BR><B>RESULTS: </B>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.<BR><B>CONCLUSIONS: </B>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.