Marianna Cavazza1, Yllka Kodra2, Patrizio Armeni3, Marta De Santis2, Julio López-Bastida4,5, Renata Linertová5,6, Juan Oliva-Moreno5,7, Pedro Serrano-Aguilar5,8, Manuel Posada-de-la-Paz9, Domenica Taruscio2, Arrigo Schieppati10, Georgi Iskrov11,12, Márta Péntek13, Johann Matthias Graf von der Schulenburg14, Panos Kanavos15, Karine Chevreul16,17,18, Ulf Persson19, Giovanni Fattore3. 1. Centre for Research on Health and Social Care Management (CERGAS), Bocconi University, Via Roentgen 1, 20136, Milan, Italy. marianna.cavazza@unibocconi.it. 2. National Centre for Rare Diseases, Istituto Superiore di Sanità (ISS), Rome, Italy. 3. Centre for Research on Health and Social Care Management (CERGAS), Bocconi University, Via Roentgen 1, 20136, Milan, Italy. 4. University of Castilla-La Mancha, Talavera de la Reina, Toledo, Spain. 5. Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC), Madrid, Spain. 6. Fundación Canaria de Investigación Sanitaria (FUNCANIS), Las Palmas de Gran Canaria, Spain. 7. University of Castilla-La Mancha, Toledo, Spain. 8. Evaluation and Planning Service at Canary Islands Health Service, Santa Cruz de Tenerife, Spain. 9. Institute of Rare Diseases Research, ISCIII, SpainRDR and CIBERER, Madrid, Spain. 10. Centro di Ricerche Cliniche per le Malattie Rare "Aldo e Cele Daccò", Mario Negri Institute for Pharmacological Research, Ranica, Bergamo, Italy. 11. Institute of Rare Diseases, Plovdiv, Bulgaria. 12. Department of Social Medicine and Public Health, Faculty of Public Health, Medical University of Plovdiv, Plovdiv, Bulgaria. 13. Department of Health Economics, Corvinus University of Budapest, Budapest, Hungary. 14. Centre for Health Economics Research Hannover (CHERH), Leibniz Universität Hannover, Hannover, Germany. 15. Department of Social Policy and LSE Health, London School of Economics and Political Science, London, UK. 16. URC Eco Ile de France, AP-HP, Paris, France. 17. Université Paris Diderot, Sorbonne Paris Cité, ECEVE, UMRS 1123, Paris, France. 18. INSERM, ECEVE, U1123, Paris, France. 19. The Swedish Institute for Health Economics, Lund, Sweden.
Abstract
OBJECTIVE: The aim of this study was to determine the economic burden from a societal perspective and the health-related quality of life (HRQOL) of patients with Duchenne muscular dystrophy (DMD) in Europe. METHODS: We conducted a cross-sectional study of patients with DMD from Bulgaria, France, Germany, Hungary, Italy, Spain, Sweden, and the UK. Data on demographic characteristics, healthcare resource utilization, informal care, labor productivity losses, and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire. Costs have been estimated from a societal perspective adopting a bottom-up approach. RESULTS: A total of 422 questionnaires were included in the study; 268 of which were collected from patients with DMD and 154 from caregivers. The average annual cost per person in 2012 ranged from €7657 in Hungary to €58,704 in France. Direct non-healthcare costs are the main component of whole costs and informal care is the main driver of non-healthcare costs. Costs are also shown to differ between children and adults. With regard to HRQOL of adult patients, the EQ-5D VAS score and EQ-5D index scores were 50.5 and 0.24, respectively. The corresponding EQ-5D VAS and EQ-5D index scores for caregivers were 74.7 and 0.71, respectively. CONCLUSIONS: We have estimated the average annual cost per patient with DMD in eight European countries adopting a social perspective, and to our knowledge this is the first study with such a wide perspective. The results on costs show a considerable gap between Eastern and Western European countries. Non-healthcare costs range from 64 to 89 % of overall costs and informal care is to a great extent the main driver of this cost category. The HRQOL of people with DMD is much lower than that of the general population.
OBJECTIVE: The aim of this study was to determine the economic burden from a societal perspective and the health-related quality of life (HRQOL) of patients with Duchenne muscular dystrophy (DMD) in Europe. METHODS: We conducted a cross-sectional study of patients with DMD from Bulgaria, France, Germany, Hungary, Italy, Spain, Sweden, and the UK. Data on demographic characteristics, healthcare resource utilization, informal care, labor productivity losses, and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire. Costs have been estimated from a societal perspective adopting a bottom-up approach. RESULTS: A total of 422 questionnaires were included in the study; 268 of which were collected from patients with DMD and 154 from caregivers. The average annual cost per person in 2012 ranged from €7657 in Hungary to €58,704 in France. Direct non-healthcare costs are the main component of whole costs and informal care is the main driver of non-healthcare costs. Costs are also shown to differ between children and adults. With regard to HRQOL of adult patients, the EQ-5D VAS score and EQ-5D index scores were 50.5 and 0.24, respectively. The corresponding EQ-5D VAS and EQ-5D index scores for caregivers were 74.7 and 0.71, respectively. CONCLUSIONS: We have estimated the average annual cost per patient with DMD in eight European countries adopting a social perspective, and to our knowledge this is the first study with such a wide perspective. The results on costs show a considerable gap between Eastern and Western European countries. Non-healthcare costs range from 64 to 89 % of overall costs and informal care is to a great extent the main driver of this cost category. The HRQOL of people with DMD is much lower than that of the general population.
Entities:
Keywords:
Duchenne muscular dystrophy; Health-related quality of life; Rare diseases; Social burden; Societal costs
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