Social/economic costs and health-related quality of life of mucopolysaccharidosis patients and their caregivers in Europe |
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| Authors: | Márta Péntek László Gulácsi Valentin Brodszky Petra Baji Imre Boncz Gábor Pogány Julio López-Bastida Renata Linertová Juan Oliva-Moreno Pedro Serrano-Aguilar Manuel Posada-de-la-Paz Domenica Taruscio Georgi Iskrov Arrigo Schieppati Johann Matthias Graf von der Schulenburg Panos Kanavos Karine Chevreul Ulf Persson Giovanni Fattore BURQOL-RD Research Network |
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| Affiliation: | 1.Department of Health Economics,Corvinus University of Budapest,Budapest,Hungary;2.Institute for Health Insurance, University of Pécs,Pécs,Hungary;3.Hungarian Federation of People with Rare and Congenital Diseases (RIROSZ),Budapest,Hungary;4.University of Castilla-La Mancha,Talavera de la Reina,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 & CIBERER,Madrid,Spain;10.National Centre for Rare Diseases, Istituto Superiore di Sanità (ISS),Rome,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.Centro di Ricerche Cliniche per Malattie Rare Aldo e Cele Daccò, Istituto di Ricerche FarmacologicheMario Negri,Ranica (Bergamo),Italy;14.Centre for Health Economics Research Hannover (CHERH),Leibniz Universit?t 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, H?tel Dieu,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;20.Centre for Research on Health and Social Care Management (CERGAS),Bocconi University,Milan,Italy |
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| Abstract: | ObjectivesTo 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.MethodsIn 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.ResultsAltogether, 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).ConclusionsMPS 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. |
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