Out-of-Pocket Expenditures of Families Affected by Epidermolysis Bullosa in Europe
Author(s)
Renata Linertová1, Christine Bodemer, PhD2, May El Hachem, PhD3, Cristina Has, PhD4, Gudrun Salamon, PhD5, Marta Pentek, PhD6, Georgi Iskrov, PhD7, Olivia Mullins, Ms8, Verónica Alonso-Ferreira, PhD9, Benjamin Rodríguez-Díaz, Msc10, Aránzazu Hernández Yumar, PhD11, Lidia García-Pérez, PhD10.
1Senior researcher, Fundación Canaria Instituto de Investigación Sanitaria de Canarias (FIISC), Las Palmas de Gran Canaria, Spain, 2Necker hospital, Paris, France, 3Ospedale Pediatrico Bambino Gesù, Rome, Italy, 4Universitätsklinikum Freiburg, Freiburg, Germany, 5Sigmund Freud University, Vienna, Austria, 6Óbuda University, Budapest, Hungary, 7Institute for Rare Diseases, Plovdiv, Bulgaria, 8DEBRA International, Vienna, Austria, 9Instituto de Salud Carlos III, Instituto de Investigación de Enfermedades Raras, Madrid, Spain, 10Fundación Canaria Instituto de Investigación Sanitaria de Canarias, Las Palmas de Gran Canaria, Spain, 11Servicio de Evaluación del Servicio Canario de la Salud, El Rosario, Spain.
1Senior researcher, Fundación Canaria Instituto de Investigación Sanitaria de Canarias (FIISC), Las Palmas de Gran Canaria, Spain, 2Necker hospital, Paris, France, 3Ospedale Pediatrico Bambino Gesù, Rome, Italy, 4Universitätsklinikum Freiburg, Freiburg, Germany, 5Sigmund Freud University, Vienna, Austria, 6Óbuda University, Budapest, Hungary, 7Institute for Rare Diseases, Plovdiv, Bulgaria, 8DEBRA International, Vienna, Austria, 9Instituto de Salud Carlos III, Instituto de Investigación de Enfermedades Raras, Madrid, Spain, 10Fundación Canaria Instituto de Investigación Sanitaria de Canarias, Las Palmas de Gran Canaria, Spain, 11Servicio de Evaluación del Servicio Canario de la Salud, El Rosario, Spain.
OBJECTIVES: Epidermolysis Bullosa (EB) is a rare, severe genetic skin disorder often associated with substantial healthcare costs, a significant portion of which may be borne by families as out-of-pocket (OOP) expenditures. Understanding the inequality and progressivity of these expenditures is crucial for equitable health policy. This is a preliminary analysis of the financial burden and its distribution among families affected by EB in seven EU countries (Austria, Bulgaria, France, Germany, Hungary, Italy, Spain).
METHODS: We analyzed survey data from 348 families, focusing on equivalised monthly income and monthly health OOP expenditures. Income-related inequality in OOP expenditures was assessed using Concentration Curves and the Concentration Index (CI) for the whole sample, and separately for adult and child patients. The progressivity of OOP expenditures was further examined using the Kakwani Index. Additionally, the incidence of catastrophic health expenditure (CHE) was calculated using the 25% income threshold.
RESULTS: The overall Concentration Index for OOP expenditures was -0.020, suggesting a very slight pro-poor distribution. However, disaggregated analysis revealed subtle differences: CI for adult patients was −0.080 (pro-poor), and for child patients, it was 0.061 (pro-rich). Despite this, the Kakwani Index consistently showed OOP expenditures to be highly regressive for the whole sample (K=−0.330), adult patients (K=−0.400), and child patients (K=−0.231). Furthermore, a substantial proportion of families experienced CHE, with incidence disproportionately higher in lower-income quintiles (48% for Q1 vs. 5% for Q5 at 25% threshold).
CONCLUSIONS: While the overall distribution of OOP expenditures might appear near-proportional, a deeper analysis reveals significant regressivity. OOP expenditures impose a disproportionately heavy financial burden on lower-income families, particularly leading to catastrophic health expenditures. These findings highlight the need for policy interventions aimed at protecting vulnerable households from the regressive financial impact of EB-related healthcare costs.
METHODS: We analyzed survey data from 348 families, focusing on equivalised monthly income and monthly health OOP expenditures. Income-related inequality in OOP expenditures was assessed using Concentration Curves and the Concentration Index (CI) for the whole sample, and separately for adult and child patients. The progressivity of OOP expenditures was further examined using the Kakwani Index. Additionally, the incidence of catastrophic health expenditure (CHE) was calculated using the 25% income threshold.
RESULTS: The overall Concentration Index for OOP expenditures was -0.020, suggesting a very slight pro-poor distribution. However, disaggregated analysis revealed subtle differences: CI for adult patients was −0.080 (pro-poor), and for child patients, it was 0.061 (pro-rich). Despite this, the Kakwani Index consistently showed OOP expenditures to be highly regressive for the whole sample (K=−0.330), adult patients (K=−0.400), and child patients (K=−0.231). Furthermore, a substantial proportion of families experienced CHE, with incidence disproportionately higher in lower-income quintiles (48% for Q1 vs. 5% for Q5 at 25% threshold).
CONCLUSIONS: While the overall distribution of OOP expenditures might appear near-proportional, a deeper analysis reveals significant regressivity. OOP expenditures impose a disproportionately heavy financial burden on lower-income families, particularly leading to catastrophic health expenditures. These findings highlight the need for policy interventions aimed at protecting vulnerable households from the regressive financial impact of EB-related healthcare costs.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE605
Topic
Economic Evaluation, Health Policy & Regulatory, Study Approaches
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases