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

Research output: Contribution to journalArticleResearchpeer review

Authors

  • BURQOL-RD Research Network

External Research Organisations

  • Corvinus University of Budapest
  • University of Pecs
  • Hungarian Federation of People with Rare and Congenital Diseases (RIROSZ)
  • Universidad de Castilla-La Mancha
  • Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC)
  • Canary Islands Foundation for Health Research (FUNCANIS)
  • Canary Islands Health Service
  • Istituto Superiore di Sanita
  • Institute of Rare Diseases
  • Medical University of Plovdiv
  • Institute of Pharmacological Research Mario Negri IRCCS
  • London School of Economics and Political Science
  • Hopital Hotel-Dieu AP-HP
  • Institut national de la santé et de la recherche médicale (INSERM)
  • The Swedish Institute for Health Economics
  • Università Commerciale Luigi Bocconi
  • Universite Paris 7
  • Institute of Health Carlos III (ISCIII)
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Details

Original languageEnglish
Pages (from-to)89-98
Number of pages10
JournalEuropean Journal of Health Economics
Volume17
Early online date9 Apr 2016
Publication statusPublished - Apr 2016

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

    Caregiver, Cost-of-illness, EQ-5D, Health-related quality of life, Mucopolysaccharidosis

ASJC Scopus subject areas

Sustainable Development Goals

Cite this

Social/economic costs and health-related quality of life of mucopolysaccharidosis patients and their caregivers in Europe. / BURQOL-RD Research Network.
In: European Journal of Health Economics, Vol. 17, 04.2016, p. 89-98.

Research output: Contribution to journalArticleResearchpeer review

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title = "Social/economic costs and health-related quality of life of mucopolysaccharidosis patients and their caregivers in Europe",
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{\textquoteright} and their informal caregivers{\textquoteright} quality of life, patients{\textquoteright} functional ability, and caregivers{\textquoteright} burden, respectively. Results: Altogether, 120 patients (children 62 %, females 40 %) and 66 caregivers completed the questionnaire. Patients{\textquoteright} mean age was 16.5 years and median age at diagnosis was 3 years. Adult patients{\textquoteright} 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 = "Caregiver, Cost-of-illness, EQ-5D, Health-related quality of life, Mucopolysaccharidosis",
author = "{BURQOL-RD Research Network} and M{\'a}rta P{\'e}ntek and L{\'a}szl{\'o} Gul{\'a}csi and Valentin Brodszky and Petra Baji and Imre Boncz and G{\'a}bor Pog{\'a}ny and Julio L{\'o}pez-Bastida and Renata Linertov{\'a} and Juan Oliva-Moreno and Pedro Serrano-Aguilar and Manuel Posada-de-la-Paz and Domenica Taruscio and Georgi Iskrov and Arrigo Schieppati and {von der Schulenburg}, {Johann Matthias Graf} and Panos Kanavos and Karine Chevreul and Ulf Persson and Giovanni Fattore",
note = "Funding Information: Supported by the Social/Economic Burden and Health-Related Quality of Life in Patients with Rare Diseases in Europe Project, which received funding from the European Union within the framework of the Health Programme [Grant A101205]. The Executive Agency of the European Union is not responsible for any use that may be made of the information contained herein. ",
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language = "English",
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Download

TY - JOUR

T1 - Social/economic costs and health-related quality of life of mucopolysaccharidosis patients and their caregivers in Europe

AU - BURQOL-RD Research Network

AU - Péntek, Márta

AU - Gulácsi, László

AU - Brodszky, Valentin

AU - Baji, Petra

AU - Boncz, Imre

AU - Pogány, Gábor

AU - López-Bastida, Julio

AU - Linertová, Renata

AU - Oliva-Moreno, Juan

AU - Serrano-Aguilar, Pedro

AU - Posada-de-la-Paz, Manuel

AU - Taruscio, Domenica

AU - Iskrov, Georgi

AU - Schieppati, Arrigo

AU - von der Schulenburg, Johann Matthias Graf

AU - Kanavos, Panos

AU - Chevreul, Karine

AU - Persson, Ulf

AU - Fattore, Giovanni

N1 - Funding Information: Supported by the Social/Economic Burden and Health-Related Quality of Life in Patients with Rare Diseases in Europe Project, which received funding from the European Union within the framework of the Health Programme [Grant A101205]. The Executive Agency of the European Union is not responsible for any use that may be made of the information contained herein.

PY - 2016/4

Y1 - 2016/4

N2 - 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.

AB - 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.

KW - Caregiver

KW - Cost-of-illness

KW - EQ-5D

KW - Health-related quality of life

KW - Mucopolysaccharidosis

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U2 - 10.1007/s10198-016-0787-0

DO - 10.1007/s10198-016-0787-0

M3 - Article

C2 - 27062257

AN - SCOPUS:84964030231

VL - 17

SP - 89

EP - 98

JO - European Journal of Health Economics

JF - European Journal of Health Economics

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