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

描述（由申请人提供）：目前对MD经济负担的了解受到用于估计成本的方法的限制。我们将使用肌营养不良监测、跟踪和研究网络（MD STARnet）收集的纵向、基于人群的监测数据，以确定和估计确诊为MD和具有良好自然病史的患者的手术成本。MD STARnet成立于2002年，对杜氏和贝克肌营养不良症（DBMD）病例进行基于人群的监测和长期随访;自2004年以来一直在进行DBMD的纵向监测。2011年，扩展了MD STARnet，以包括另外7种MD（先天性、远端、Emery-Dreifuss、面肩肱、肢带、肌强直和眼咽）的横断面监测;这些MD的监测将扩展到进行纵向监测。我们将通过对医疗服务的纵向监测、私人和公共索赔数据库以及自我报告的医疗服务疾病成本调查来评估这些MD的经济负担。我们已成功整合了爱荷华州主要合作伙伴（如爱荷华州神经肌肉项目）提供的临床、项目和转诊资源，以及爱荷华州先天性和遗传性疾病登记处的基于人群的活跃资源，以进行DBMD的纵向监测和其他MD的横断面监测。此外，我们与生殖分子流行病学研究和教育计划以及爱荷华州大学公共卫生统计中心的合作伙伴关系将提供经验丰富的调查人员进行数据分析和发布疾病成本数据。为了实现我们的研究目标，我们的目标是：1）对DBMD和其他选定的MD进行疾病成本分析; 2）为选定的MD制定和实施医疗保健服务疾病成本调查; 3）估计选定的MD遵守护理考虑的成本;以及4）向利益相关者和科学界传播研究结果。可用性的人口为基础的治疗费用估计建议在医疗MD的护理将是一个有用的资源，由医疗保健从业者和家庭的财务规划在诊断时，随着疾病的进展。

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.