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

Title data

Schreiber-Katz, Olivia ; Klug, Constanze ; Thiele, Simone ; Schorling, Elisabeth ; Zowe, Janet ; Reilich, Peter ; Nagels, Klaus ; Walter, Maggie C.:
Comparative cost of illness analysis and assessment of health care burden of Duchenne and Becker muscular dystrophies in Germany.
In: Orphanet Journal of Rare Diseases. Vol. 9 (2014) . - p. 210.
ISSN 1750-1172
DOI: https://doi.org/10.1186/s13023-014-0210-9

Abstract in another language

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.

Further data

Item Type: Article in a journal
Refereed: Yes
Institutions of the University: Faculties > Faculty of Law, Business and Economics > Lehrstuhl Medizinmanagement und Versorgungsforschung > Lehrstuhl Medizinmanagement und Versorgungsforschung - Univ.-Prof. Dr. Klaus Nagels
Faculties
Faculties > Faculty of Law, Business and Economics
Faculties > Faculty of Law, Business and Economics > Lehrstuhl Medizinmanagement und Versorgungsforschung
Result of work at the UBT: Yes
DDC Subjects: 300 Social sciences > 300 Social sciences, sociology and anthropology
300 Social sciences > 330 Economics
600 Technology, medicine, applied sciences > 610 Medicine and health
Date Deposited: 08 Sep 2017 08:15
Last Modified: 25 Sep 2017 14:59
URI: https://eref.uni-bayreuth.de/id/eprint/39407