Comparative cost of illness analysis and assessment of health care burden of Duchenne and Becker muscular dystrophies in
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RESEARCH
Open Access
Comparative cost of illness analysis and assessment of health care burden of Duchenne and Becker muscular dystrophies in Germany Olivia Schreiber-Katz1†, Constanze Klug2†, Simone Thiele1, Elisabeth Schorling2, Janet Zowe2, Peter Reilich1, Klaus H Nagels2† and Maggie C Walter1*†
Abstract Background: Our study aimed to determine the burden of illness in dystrophinopathy type Duchenne (DMD) and Becker (BMD), both leading to progressive disability, reduced working capacity and high health care utilization. Methods: A micro-costing method was used to examine the direct, indirect and informal care costs measuring the economic burden of DMD in comparison to BMD on patients, relatives, payers and society in Germany and to determine the health care burden of these diseases. Standardized questionnaires were developed based on predefined structured interview guidelines to obtain data directly from patients and caregivers using the German dystrophinopathy patient registry. The health-related quality of life (HRQOL) was analyzed using PedsQL™ Measurement Model. Results: In total, 363 patients with genetically confirmed dystrophinopathies were enrolled. Estimated annual disease burden including direct medical/non-medical, indirect and informal care costs of DMD added up to € 78,913 while total costs in BMD were € 39,060. Informal care costs, indirect costs caused by loss of productivity and absenteeism of patients and caregivers as well as medical costs of rehabilitation services and medical aids were identified as the most important cost drivers. Total costs notably increased with disease progression and were consistent with the clinical severity; however, patients’ HRQOL declined with disease progression. Conclusion: In conclusion, early assessments of economic aspects and the disease burden are essential to gain extensive knowledge of a distinct disease and above all play an important role in funding drug development programs for rare diseases. Therefore, our results may help to accelerate payer negotiations such as the pricing and reimbursement of new therapies, and will hopefully contribute to facilitating the efficient translation of innovations from clinical research over marketing authorization to patient access to a causative treatment. Keywords: Duchenne muscular dystrophy, Becker muscular dystrophy, Direct costs, Indirect costs, Informal care costs, Cost of illness (COI), Burden of illness, Health care burden, Health-related quality of life (HRQOL), Socio-economic evaluation
Background Duchenne muscular dystrophy (DMD) is a hereditary X-linked neuromuscular disorder due to mutations in the dystrophin gene with a worldwide incidence of approximately 1:5,000 male newborns [1,2] leading to progressive muscle atrophy and weakness. First symptoms are * Correspondence: [email protected] † Equal contributors 1 Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University of Munich, Ziemssenstrasse 1, 80336 Munich, Germany Full list of author information is available
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