248740 Cost of Amyotrophic Lateral Sclerosis, Muscular Dystrophy, and Spinal Muscular Atrophy

Wednesday, November 2, 2011: 8:50 AM

Wenya Yang, MPA, MA , The Lewin Group, Falls Church, VA
Paul Hogan, MS , Federal National Security and Emergency Preparedness, The Lewin Group, Falls Church, VA
Carol Simon, PhD , Lewin Group, Falls Church, VA
Yiduo Zhang, PhD , Federal National Security and Emergency Preparedness, The Lewin Group, Falls Church, VA
Anjali Jain, MD , Federal Health, The Lewin Group, Falls Church, VA
Annie Kennedy , National Advocacy Office, Muscular Dystrophy Association, Washington, D.C., DC
Valerie Cwik, MD , Research and Medical, Muscular Dystrophy Association, Tucson, AZ
Elizabeth Habeeb-Louks, MS , Translational Research, Muscular Dystrophy Association, Tucson, AZ
Jane Larkindale, DPhil , Translational Research, Muscular Dystrophy Association, Tucson, AZ
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.

Learning Areas:
Advocacy for health and health education
Biostatistics, economics
Public health or related public policy
Public health or related research

Learning Objectives:
1. Estimate direct medical cost associated with three main types of neuromuscular diseases that rare yet devastating to patients and families through claims data analysis 2. Estimate direct non-medical cost associated with these diseases to patients and families via a cost-of-illness survey 3. Assess indirect cost associated with the three diseases in terms of productivity loss, income loss, and opportunity cost via a cost-of-illness survey 4. Determine the total economic burden of these diseases on families, payers, and society as a whole

Keywords: Public Health, Healthcare Costs

Presenting author's disclosure statement:

Qualified on the content I am responsible for because: This study is funded by the Muscular Dystrophy Association.
Any relevant financial relationships? Yes

Name of Organization Clinical/Research Area Type of relationship
Muscular Dystrophy Association neurological sciences Independent Contractor (contracted research and clinical trials)

I agree to comply with the American Public Health Association Conflict of Interest and Commercial Support Guidelines, and to disclose to the participants any off-label or experimental uses of a commercial product or service discussed in my presentation.

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