Social/economic costs and health-related quality of life of mucopolysaccharidosis patients and their caregivers in Europe

Márta Péntek, László Gulácsi, Valentin Brodszky, Petra Baji, Imre Boncz (), Gábor Pogány, Julio López-Bastida, Renata Linertová, Juan Oliva (), Pedro Serrano-Aguilar, Manuel Posada- de-la-Paz, Domenica Taruscio, Georgi Iskrov, Arrigo Schieppati, Johann Matthias Graf Schulenburg, Panos Kanavos, Karine Chevreul, Ulf Persson and Giovanni Fattore
Additional contact information
László Gulácsi: Corvinus University of Budapest
Gábor Pogány: Hungarian Federation of People with Rare and Congenital Diseases (RIROSZ)
Julio López-Bastida: University of Castilla-La Mancha
Renata Linertová: Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC)
Pedro Serrano-Aguilar: Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC)
Manuel Posada- de-la-Paz: Institute of Rare Diseases Research, ISCIII, SpainRDR & CIBERER
Domenica Taruscio: National Centre for Rare Diseases, Istituto Superiore di Sanità (ISS)
Georgi Iskrov: Institute of Rare Diseases
Arrigo Schieppati: Centro di Ricerche Cliniche per Malattie Rare Aldo e Cele Daccò, Istituto di Ricerche Farmacologiche Mario Negri
Johann Matthias Graf Schulenburg: Leibniz Universität Hannover
Panos Kanavos: London School of Economics and Political Science
Karine Chevreul: URC Eco Ile de France, AP-HP, Hôtel Dieu
Ulf Persson: The Swedish Institute for Health Economics
Giovanni Fattore: Bocconi University

The European Journal of Health Economics, 2016, vol. 17, issue 1, No 9, 89-98

Abstract: 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.

Keywords: Mucopolysaccharidosis; Health-related quality of life; Cost-of-illness; Caregiver; EQ-5D (search for similar items in EconPapers)
JEL-codes: I19 (search for similar items in EconPapers)
Date: 2016
References: View references in EconPapers View complete reference list from CitEc
Citations: View citations in EconPapers (5)

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DOI: 10.1007/s10198-016-0787-0

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