Julio López-Bastida1,2, Renata Linertová3,4, Juan Oliva-Moreno3,5, Manuel Posada-de-la-Paz6, Pedro Serrano-Aguilar3,7, Panos Kanavos8, Domenica Taruscio9, Arrigo Schieppati10, Georgi Iskrov11,12, Petra Baji13, Claudia Delgado14, Johann Matthias Graf von der Schulenburg15, Ulf Persson16, Karine Chevreul17,18,19, Giovanni Fattore20. 1. University of Castilla-La Mancha, Talavera de la Reina, Toledo, Spain. julio.lopezbastida@uclm.es. 2. Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC), Madrid, Spain. julio.lopezbastida@uclm.es. 3. Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC), Madrid, Spain. 4. Canary Islands Foundation for Health Research (FUNCANIS), Las Palmas de Gran Canaria, Spain. 5. University of Castilla-La Mancha, Toledo, Spain. 6. Institute of Rare Diseases Research (IIER), Spain RDR and CIBERER, Madrid, Spain. 7. Evaluation and Planning Service at Canary Islands Health Service, Santa Cruz de Tenerife, Spain. 8. London School of Economics and Political Science-Health Research Centre, London, UK. 9. National Center for Rare Diseases, Istituto superiore di sanità (ISS), Rome, Italy. 10. Centro di Ricerche Cliniche per Malattie Rare Aldo e Cele Daccò, Istituto di Ricerche Farmacologiche Mario Negri, 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. Federación Española de Enfermedades Raras (FEDER), Madrid, Spain. 15. Centre for Health Economics/Institute for Risk and Insurance, Leibniz University, Hannover, Germany. 16. The Swedish Institute for Health Economics, Lund, Sweden. 17. URC Eco Ile de France, AP-HP, Paris, France. 18. Université Paris Diderot, Sorbonne Paris Cité, ECEVE, UMRS 1123, Paris, France. 19. INSERM, ECEVE, U1123, Paris, France. 20. Centre for Research on Health and Social Care (CERGAS), Bocconi University, Milan, Italy.
Abstract
OBJECTIVE: The aim of this study was to determine the economic burden from a societal perspective and health-related quality of life (HRQOL) of patients with Prader-Willi syndrome (PWS) in Europe. METHODS: We conducted a cross-sectional study of patients with PWS from Spain, Bulgaria, Hungary, Germany, Italy, the UK, Sweden and France. Data on demographic characteristics, healthcare resource utilisation, informal care, labour productivity losses and HRQOL were collected from questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire. RESULTS: A total of 261 patients completed the questionnaire. The average annual costs ranged from € 3937 to € 67,484 between countries; the reference year for unit prices was 2012. Direct healthcare costs ranged from € 311 to € 18,760, direct non-healthcare costs ranged from € 1269 to € 44,035, and loss of labour productivity ranged from € 0 to € 2255. Costs were also shown to differ between children and adults. The mean EQ-5D index score for adult PWS patients ranged between 0.40 and 0.81 and the mean EQ-5D visual analogue scale score ranged between 51.25 and 90.00. CONCLUSION: The main strengths of this study lie in our bottom-up approach to costing and in the evaluation of PWS patients from a broad societal perspective. This type of analysis is very scarce in the international literature on rare diseases in comparison with other illnesses. We conclude that PWS patients incur considerable societal costs and experience substantial deterioration in HRQOL.
OBJECTIVE: The aim of this study was to determine the economic burden from a societal perspective and health-related quality of life (HRQOL) of patients with Prader-Willi syndrome (PWS) in Europe. METHODS: We conducted a cross-sectional study of patients with PWS from Spain, Bulgaria, Hungary, Germany, Italy, the UK, Sweden and France. Data on demographic characteristics, healthcare resource utilisation, informal care, labour productivity losses and HRQOL were collected from questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire. RESULTS: A total of 261 patients completed the questionnaire. The average annual costs ranged from € 3937 to € 67,484 between countries; the reference year for unit prices was 2012. Direct healthcare costs ranged from € 311 to € 18,760, direct non-healthcare costs ranged from € 1269 to € 44,035, and loss of labour productivity ranged from € 0 to € 2255. Costs were also shown to differ between children and adults. The mean EQ-5D index score for adult PWSpatients ranged between 0.40 and 0.81 and the mean EQ-5D visual analogue scale score ranged between 51.25 and 90.00. CONCLUSION: The main strengths of this study lie in our bottom-up approach to costing and in the evaluation of PWSpatients from a broad societal perspective. This type of analysis is very scarce in the international literature on rare diseases in comparison with other illnesses. We conclude that PWSpatients incur considerable societal costs and experience substantial deterioration in HRQOL.
Entities:
Keywords:
Cost of illness; Economic burden; Health-related quality of life; Prader-Willi syndrome
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