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

Background and objectivesMotor 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.MethodsPrimary 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.Results404 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 83,060euro in ALS, 206,856euro in SMA and 27,074euro 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: 138,960euro/QALY for ALS, 525,033euro/QALY for SMA, and 49,573euro/QALY for HSP. The main predictors of the high cost of illness and low HRQoL were disease progression and loss of individual autonomy.ConclusionAs 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.

Automated summary

This study conducted an economic evaluation of Motor Neuron Diseases (MND) in Germany, finding that these rare diseases have a significant impact on patients and society. Therapeutic and supportive measures can reduce the personal burden and long-term costs for patients.