Comp D-Cost of Illness Analysis of MD and Neuromuscular Disorders

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DESCRIPTION (provided by applicant): The current knowledge of the economic burden of MDs is limited by the methodologies used to estimate cost. We will use longitudinal, population-based surveillance data collected by the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet) to identify and estimate costs of procedures for patients with confirmed MDs and well-characterized natural histories. The MD STARnet was established in 2002 to conduct population-based surveillance and long-term follow-up of cases with Duchenne and Becker muscular dystrophies (DBMD); longitudinal surveillance for DBMD has been ongoing since 2004. In 2011, the MD STARnet was expanded to include cross-sectional surveillance for seven additional MDs (congenital, distal, Emery-Dreifuss, facioscapulohumeral, limb-girdle, myotonic, and oculopharyngeal); surveillance of these MDs will be expanded to conduct longitudinal surveillance. We will evaluate the economic burden of these MDs from longitudinal surveillance of health care services received, private and public claims databases, and a self-report Health Care Services Cost-of-Illness survey. We have successfully integrated clinical, programmatic, and referral resources provided by key partners in Iowa, such as the Iowa Neuromuscular Program, with the active, population-based resources of the Iowa Registry for Congenital and Inherited Disorders to conduct longitudinal surveillance of DBMD and cross-sectional surveillance for the additional MDs. Also, our partnerships with the Reproductive Molecular Epidemiology Research and Education Program and the Center for Public Health Statistics at The University of Iowa will provide experienced investigators to conduct data analyses and publish cost-of-illness data. To accomplish our study objectives, we aim to: 1) conduct cost-of- illness analyses for DBMD and other selected MDs; 2) develop and implement a Health Care Services Cost-of-Illness survey for selected MDs; 3) estimate cost of compliance with care considerations for selected MDs; and 4) disseminate research findings to stakeholders and the scientific community. The availability of population-based cost estimates of treatments recommended in the care of MDs will be a useful resource for financial planning by health care practitioners and families at the time of diagnosis and as disease progresses.