Cost of Amyotrophic Lateral Sclerosis, Muscular Dystrophy, and Spinal Muscular Atrophy

Background: Amyotrophic Lateral Sclerosis (ALS), Muscular Dystrophy (MD), and Spinal Muscular Atrophy (SMA) are the main types of neuromuscular diseases. The economic burden of these rare diseases is largely unknown in the US. We estimate the direct and indirect, medical and non-medical costs associated with these diseases.

Study Design: The cross-sectional study has two components:

1) Claims Analysis: Sample was identified based on a disease diagnosis from a large commercial claims database and a 5% sample of Medicare beneficiaries. Patients had to be continuous covered for both medical and pharmacy benefits in any of the three years 2007 – 2009 (commercial claims) and in year 2008 (Medicare). Study outcomes include healthcare use and cost by types of insurance (i.e. commercial vs. Medicare), types of care (e.g. inpatient vs. outpatient) and patient characteristics.

2) A Cost-of-Illness Survey: Survey questionnaire included 56 questions on disease history, employment status, and out-of-pocket expenses. A stratified random sample was selected out of the members of the Muscular Dystrophy Association.

We conducted descriptive analysis on the family burden of these diseases by family and patient characteristics. Multivariate regression analysis will be used to evaluate the impact of these diseases on family income by comparing data from MDA families to controls obtained from national surveys such as the American Community Survey using a human capital approach.

Results: We identified 3,076 patients with ALS, 8,783 with MD, and 1,914 with SMA from the commercial claims data during 2007 to 2009. Another 800 patients were identified from Medicare data for ALS and MD respectively. Commercial claims analysis suggests that ALS ($27,527), MD ($22,118), and SMA ($19,829) are associated with significant direct annual medical costs and so does the analysis from Medicare data. Survey is being implemented and results will be available later. Conclusions: This is the first study that quantifies the overall economic burden of ALS, MD, and SMA in the U.S. Findings suggest that the significant costs related to these diseases drain resources from families and payers alike. A breakthrough medical treatment is urgently needed.