Health-Related Quality of Life of Patients With Epidermolysis Bullosa in Hungary: Results With the EQ-5D-5L and EQ-5D-Y-3L Preference-Based Measurement Tools
Author(s)
Marta Pentek, MD, DSc1, Márta Medvecz, MD, PhD2, László Gulácsi, MD, DSc1, Zsombor Zrubka, MD, MBA, PhD1, Renata Linertová, MSc3, Áron Hölgyesi, PharmD, PhD1.
1Health Economics Research Center, University Research and Innovation Center, Obuda University, Budapest, Hungary, 2Department of Dermatology, Venereology and Dermatooncology, Faculty of Medicine, Semmelweis University, Budapest, Hungary, 3Canary Islands Health Research Institute Foundation (FIISC), Las Palmas de Gran Canaria, Spain.
1Health Economics Research Center, University Research and Innovation Center, Obuda University, Budapest, Hungary, 2Department of Dermatology, Venereology and Dermatooncology, Faculty of Medicine, Semmelweis University, Budapest, Hungary, 3Canary Islands Health Research Institute Foundation (FIISC), Las Palmas de Gran Canaria, Spain.
OBJECTIVES: As part of the BUR-EB project (https://www.bur-eb.com/) on the social and economic impact of epidermolysis bullosa (EB), we aimed to assess the health status utility values of the EB patients in Hungary to feed health economic evaluations of new innovative therapies with country-specific data.
METHODS: A cross-sectional online survey was performed in 2024. Recruitment was conducted by DEBRA Hungary (patient organisation) and a university hospital-based EB centre. A resource utilisation survey was designed, and patients were asked to complete the Hungarian version of the EQ-5D-5L (for adults) and EQ-5D-Y-3L (children) questionnaires. Tariffs for Hungary were used to calculate EQ-5D index values. Ethical approval was obtained from Medical Research Council, Hungary (BM/26385-3/2023). The study has been supported by National Research, Development, and Innovation Fund of Hungary, financed under the 2019-2.1.7-ERA-NET-2022-00055 funding scheme and EJP RD JTC 2021.
RESULTS: Altogether 9 adults (female 44.4%) participated in the survey and out of the 13 child (female 53.8%) participants EQ-5D-Y-3L was applicable for 7 children. The number of adults reporting no problem on the five domains of the EQ-5D-5L descriptive system was: Mobility 1 (11.1%), Self-care 5 (55.6%), Usual Activities 2 (22.2%), Pain/discomfort 1 (11.8%), Anxiety/depression 4 (44.4%). These numbers for the EQ-5D-Y-3L among children were 2 (28.6%), 3 (42.9%), 2 (28.6%) 2 (28.6%) and 7 (100%), respectively. EQ-5D-5L index values of adults was mean 0.47 (0.5) and of children was 0.71 (0.2). These values are substantially lower than of the age and sex matched values of the general population of Hungary.
CONCLUSIONS: This first EB study with the EQ-5D instruments in Hungary show a significant burden of EB on patients’ health-related quality of life. Our data can be used to estimate utility scores but further studies involving larger samples of different EB subtypes are encouraged to confirm and refine our results.
METHODS: A cross-sectional online survey was performed in 2024. Recruitment was conducted by DEBRA Hungary (patient organisation) and a university hospital-based EB centre. A resource utilisation survey was designed, and patients were asked to complete the Hungarian version of the EQ-5D-5L (for adults) and EQ-5D-Y-3L (children) questionnaires. Tariffs for Hungary were used to calculate EQ-5D index values. Ethical approval was obtained from Medical Research Council, Hungary (BM/26385-3/2023). The study has been supported by National Research, Development, and Innovation Fund of Hungary, financed under the 2019-2.1.7-ERA-NET-2022-00055 funding scheme and EJP RD JTC 2021.
RESULTS: Altogether 9 adults (female 44.4%) participated in the survey and out of the 13 child (female 53.8%) participants EQ-5D-Y-3L was applicable for 7 children. The number of adults reporting no problem on the five domains of the EQ-5D-5L descriptive system was: Mobility 1 (11.1%), Self-care 5 (55.6%), Usual Activities 2 (22.2%), Pain/discomfort 1 (11.8%), Anxiety/depression 4 (44.4%). These numbers for the EQ-5D-Y-3L among children were 2 (28.6%), 3 (42.9%), 2 (28.6%) 2 (28.6%) and 7 (100%), respectively. EQ-5D-5L index values of adults was mean 0.47 (0.5) and of children was 0.71 (0.2). These values are substantially lower than of the age and sex matched values of the general population of Hungary.
CONCLUSIONS: This first EB study with the EQ-5D instruments in Hungary show a significant burden of EB on patients’ health-related quality of life. Our data can be used to estimate utility scores but further studies involving larger samples of different EB subtypes are encouraged to confirm and refine our results.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR121
Topic
Patient-Centered Research
Topic Subcategory
Health State Utilities, Patient-reported Outcomes & Quality of Life Outcomes
Disease
Rare & Orphan Diseases, Sensory System Disorders (Ear, Eye, Dental, Skin)