The cost-effectiveness of early noninvasive ventilation for ALS patients.
BACKGROUND
Optimal timing of noninvasive positive pressure ventilation (NIPPV) initiation in patients with amyotrophic lateral sclerosis (ALS) is unknown, but NIPPV appears to benefit ALS patients who are symptomatic from pulmonary insufficiency. This has prompted research proposals of earlier NIPPV initiation in the ALS disease course in an attempt to further improve ALS patient quality of life and perhaps survival. We therefore used a cost-utility analysis to determine a priori what magnitude of health-related quality of life (HRQL) improvement early NIPPV initiation would need to achieve to be cost-effective in a future clinical trial.


METHODS
Using a Markov decision analytic model we calculated the benefit in health-state utility that NIPPV initiated at ALS diagnosis must achieve to be cost-effective. The primary outcome was the percent utility gained through NIPPV in relation to two common willingness-to-pay thresholds: 50,000 dollars and 100,000 dollars per quality-adjusted life year (QALY).


RESULTS
Our results indicate that if NIPPV begun at the time of diagnosis improves ALS patient HRQL as little as 13.5%, it would be a cost-effective treatment. Tolerance of NIPPV (assuming a 20% improvement in HRQL) would only need to exceed 18% in our model for treatment to remain cost-effective using a conservative willingness-to-pay threshold of 50,000 dollars per QALY.


CONCLUSIONS
If early use of NIPPV in ALS patients is shown to improve HRQL in future studies, it is likely to be a cost-effective treatment. Clinical trials of NIPPV begun at the time of ALS diagnosis are therefore warranted from a cost-effectiveness standpoint.
Gruis
Kirsten L
KL
Department of Neurology, University of Michigan Health System, Ann Arbor, Michigan, USA. kgruis@med.umich.edu
Chernew
Michael E
ME
Brown
Devin L
DL
D000690:Amyotrophic Lateral Sclerosis / Q000150:complications / Q000191:economics / Q000628:therapy*; D001294:Attitude to Health; D003362:Cost-Benefit Analysis / Q000706:statistics & numerical data; D003663:Decision Trees; D005245:Fee Schedules; D005102:Health Expenditures; D006801:Humans; D008390:Markov Chains; D006278:Medicare; D017059:Models, Econometric; D011175:Positive-Pressure Respiration / Q000191:economics*; D011788:Quality of Life; D019057:Quality-Adjusted Life Years; D012131:Respiratory Insufficiency / Q000209:etiology / Q000628:therapy*; D014481:United States