Márta Péntek1, László Gulácsi2, Valentin Brodszky2, Petra Baji2, Imre Boncz3, Gábor Pogány4, Julio López-Bastida5,6, Renata Linertová6,7, Juan Oliva-Moreno6,8, Pedro Serrano-Aguilar6,9, Manuel Posada-de-la-Paz10, Domenica Taruscio11, Georgi Iskrov12,13, Arrigo Schieppati14, Johann Matthias Graf von der Schulenburg15, Panos Kanavos16, Karine Chevreul17,18,19, Ulf Persson20, Giovanni Fattore21. 1. Department of Health Economics, Corvinus University of Budapest, Budapest, Hungary. marta.pentek@uni-corvinus.hu. 2. Department of Health Economics, Corvinus University of Budapest, Budapest, Hungary. 3. Institute for Health Insurance, University of Pécs, Pécs, Hungary. 4. Hungarian Federation of People with Rare and Congenital Diseases (RIROSZ), Budapest, Hungary. 5. University of Castilla-La Mancha, Talavera de la Reina, Toledo, Spain. 6. Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC), Madrid, Spain. 7. Fundación Canaria de Investigación Sanitaria (FUNCANIS), Las Palmas de Gran Canaria, Spain. 8. University of Castilla-La Mancha, Toledo, Spain. 9. Evaluation and Planning Service at Canary Islands Health Service, Santa Cruz de Tenerife, Spain. 10. Institute of Rare Diseases Research, ISCIII, SpainRDR & CIBERER, Madrid, Spain. 11. National Centre for Rare Diseases, Istituto Superiore di Sanità (ISS), Rome, Italy. 12. Institute of Rare Diseases, Plovdiv, Bulgaria. 13. Department of Social Medicine and Public Health, Faculty of Public Health, Medical University of Plovdiv, Plovdiv, Bulgaria. 14. Centro di Ricerche Cliniche per Malattie Rare Aldo e Cele Daccò, Istituto di Ricerche Farmacologiche Mario Negri, Ranica (Bergamo), Italy. 15. Centre for Health Economics Research Hannover (CHERH), Leibniz Universität Hannover, Hannover, Germany. 16. Department of Social Policy and LSE Health, London School of Economics and Political Science, London, UK. 17. URC Eco Ile de France, AP-HP, Hôtel Dieu, Paris, France. 18. Université Paris Diderot, Sorbonne Paris Cité, ECEVE, UMRS 1123, Paris, France. 19. INSERM, ECEVE, U1123, Paris, France. 20. The Swedish Institute for Health Economics, Lund, Sweden. 21. Centre for Research on Health and Social Care Management (CERGAS), Bocconi University, Milan, Italy.
Abstract
OBJECTIVES: To assess the health-related quality of life (HRQOL) of patients with mucopolysaccharidosis (MPS) and their caregivers and to quantify the disease-related costs from a societal perspective. METHODS: In the context of a multi-country study of rare diseases (BURQOL-RD project), a cross-sectional survey was performed among MPS patients in seven European countries. Data on demographic characteristics, health resource utilization, informal care, and loss of labor productivity were collected. The EQ-5D, Barthel index (BI), and Zarit burden interview (ZBI) questionnaires were used to assess patients' and their informal caregivers' quality of life, patients' functional ability, and caregivers' burden, respectively. RESULTS: Altogether, 120 patients (children 62 %, females 40 %) and 66 caregivers completed the questionnaire. Patients' mean age was 16.5 years and median age at diagnosis was 3 years. Adult patients' average EQ-5D and EQ VAS scores varied across countries from 0.13 to 0.43 and 30.0 to 62.2, respectively, mean BI was 46.7, and ZBI was 32.7. Mean informal care time was 51.3 h/week. The mean total annual cost per patient (reference year 2012) was €24,520 in Hungary, €25,993 in France, €84,921 in Italy, €94,384 in Spain, and €209,420 in Germany. Costs are also shown to differ between children and adults. Direct costs accounted for most of the costs in all five countries (80, 100, 99, 98, and 93 %, respectively). CONCLUSIONS: MPS patients experience substantial loss of HRQOL and their families take a remarkable part in their care. Although utilization of health and social care resources varies significantly across countries, MPS incurs considerable societal costs in all the countries studied.
OBJECTIVES: To assess the health-related quality of life (HRQOL) of patients with mucopolysaccharidosis (MPS) and their caregivers and to quantify the disease-related costs from a societal perspective. METHODS: In the context of a multi-country study of rare diseases (BURQOL-RD project), a cross-sectional survey was performed among MPSpatients in seven European countries. Data on demographic characteristics, health resource utilization, informal care, and loss of labor productivity were collected. The EQ-5D, Barthel index (BI), and Zarit burden interview (ZBI) questionnaires were used to assess patients' and their informal caregivers' quality of life, patients' functional ability, and caregivers' burden, respectively. RESULTS: Altogether, 120 patients (children 62 %, females 40 %) and 66 caregivers completed the questionnaire. Patients' mean age was 16.5 years and median age at diagnosis was 3 years. Adult patients' average EQ-5D and EQ VAS scores varied across countries from 0.13 to 0.43 and 30.0 to 62.2, respectively, mean BI was 46.7, and ZBI was 32.7. Mean informal care time was 51.3 h/week. The mean total annual cost per patient (reference year 2012) was €24,520 in Hungary, €25,993 in France, €84,921 in Italy, €94,384 in Spain, and €209,420 in Germany. Costs are also shown to differ between children and adults. Direct costs accounted for most of the costs in all five countries (80, 100, 99, 98, and 93 %, respectively). CONCLUSIONS:MPSpatients experience substantial loss of HRQOL and their families take a remarkable part in their care. Although utilization of health and social care resources varies significantly across countries, MPS incurs considerable societal costs in all the countries studied.
Entities:
Keywords:
Caregiver; Cost-of-illness; EQ-5D; Health-related quality of life; Mucopolysaccharidosis
Authors: Kenneth I Berger; Simone C Fagondes; Roberto Giugliani; Karen A Hardy; Kuo Sheng Lee; Ciarán McArdle; Maurizio Scarpa; Martin J Tobin; Susan A Ward; David M Rapoport Journal: J Inherit Metab Dis Date: 2012-11-15 Impact factor: 4.982
Authors: Isaac Aranda-Reneo; Luz María Peña-Longobardo; Juan Oliva-Moreno; Svenja Litzkendorf; Isabelle Durand-Zaleski; Eduardo F Tizzano; Julio López-Bastida Journal: Int J Environ Res Public Health Date: 2020-12-02 Impact factor: 3.390