Comparative cost of illness analysis and assessment of health care burden of Duchenne and Becker muscular dystrophies in Germany

Orphanet Journal of Rare Diseases

Maggie C Walter²  Klaus H Nagels¹  Peter Reilich²  Janet Zowe¹  Elisabeth Schorling¹  Simone Thiele²  Constanze Klug¹  Olivia Schreiber-Katz² 

[1] Institute for Healthcare Management and Health Sciences, University of Bayreuth, Prieserstrasse 2, Bayreuth, 95444, Germany;Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University of Munich, Ziemssenstrasse 1, Munich, 80336, Germany

关键词: Socio-economic evaluation;    Health-related quality of life (HRQOL);    Health care burden;    Burden of illness;    Cost of illness (COI);    Informal care costs;    Indirect costs;    Direct costs;    Becker muscular dystrophy;    Duchenne muscular dystrophy;   

Others  :  1138573
DOI  :  10.1186/s13023-014-0210-9

 received in 2014-09-30, accepted in 2014-12-05,  发布年份 2014

【 摘 要 】

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.

【 授权许可】

   
2014 Schreiber-Katz et al.; licensee BioMed Central.

【 图 表 】

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