Identification of strengths and weaknesses of the healthcare system for persons living with rare diseases in Catalonia (Spain), and recommendations to improve its comprehensive attention

Background: Rare diseases (RDs) are a heterogeneous group of complex and low-prevalence conditions in which the time to establish a definitive diagnosis is often too long. In addition, for most RDs, few to no treatments are available and it is often difficult to find a specialized care team. Objecti...

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Autores: Hernández Rodríguez, José|||0000-0002-2357-2015, Martinez Valle, Fernando|||0000-0003-2673-2034, Acebes, X.|||0000-0001-6449-938X, Alerany, Carmen, Antón, Jordi|||0000-0002-8792-4219, Calvo Rojas, Gonzalo|||0000-0001-8860-6653, Corral, M., Cruz, J., Mangues, Ma Antonia|||0000-0002-1438-8515, Mateo, Jose|||0000-0002-4227-3256, Rivera, Josefa, Salazar, Albert, Francisco, R., Mallol, Cristina|||0000-0002-2920-9612, Reig-Viader, Rita|||0000-0002-6893-6177, Tigri-Santiña, Ariadna|||0000-0003-2765-0065, Ricart, A., Palau, F.
Tipo de recurso: artículo
Fecha de publicación:2025
País:España
Institución:Universitat Autònoma de Barcelona
Repositorio:Dipòsit Digital de Documents de la UAB
Idioma:inglés
OAI Identifier:oai:ddd.uab.cat:320385
Acceso en línea:https://ddd.uab.cat/record/320385
https://dx.doi.org/urn:doi:10.1186/s13023-024-03518-x
Access Level:acceso abierto
Palabra clave:Comprehensive care
Focus group
Healthcare system
Patient journey
Person living with a rare disease
Rare diseases
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network_acronym_str ES
network_name_str España
repository_id_str
dc.title.none.fl_str_mv Identification of strengths and weaknesses of the healthcare system for persons living with rare diseases in Catalonia (Spain), and recommendations to improve its comprehensive attention
the "acERca las enfermedades raras" project
title Identification of strengths and weaknesses of the healthcare system for persons living with rare diseases in Catalonia (Spain), and recommendations to improve its comprehensive attention
spellingShingle Identification of strengths and weaknesses of the healthcare system for persons living with rare diseases in Catalonia (Spain), and recommendations to improve its comprehensive attention
Hernández Rodríguez, José|||0000-0002-2357-2015
Comprehensive care
Focus group
Healthcare system
Patient journey
Person living with a rare disease
Rare diseases
title_short Identification of strengths and weaknesses of the healthcare system for persons living with rare diseases in Catalonia (Spain), and recommendations to improve its comprehensive attention
title_full Identification of strengths and weaknesses of the healthcare system for persons living with rare diseases in Catalonia (Spain), and recommendations to improve its comprehensive attention
title_fullStr Identification of strengths and weaknesses of the healthcare system for persons living with rare diseases in Catalonia (Spain), and recommendations to improve its comprehensive attention
title_full_unstemmed Identification of strengths and weaknesses of the healthcare system for persons living with rare diseases in Catalonia (Spain), and recommendations to improve its comprehensive attention
title_sort Identification of strengths and weaknesses of the healthcare system for persons living with rare diseases in Catalonia (Spain), and recommendations to improve its comprehensive attention
dc.creator.none.fl_str_mv Hernández Rodríguez, José|||0000-0002-2357-2015
Martinez Valle, Fernando|||0000-0003-2673-2034
Acebes, X.|||0000-0001-6449-938X
Alerany, Carmen
Antón, Jordi|||0000-0002-8792-4219
Calvo Rojas, Gonzalo|||0000-0001-8860-6653
Corral, M.
Cruz, J.
Mangues, Ma Antonia|||0000-0002-1438-8515
Mateo, Jose|||0000-0002-4227-3256
Rivera, Josefa
Salazar, Albert
Francisco, R.
Mallol, Cristina|||0000-0002-2920-9612
Reig-Viader, Rita|||0000-0002-6893-6177
Tigri-Santiña, Ariadna|||0000-0003-2765-0065
Ricart, A.
Palau, F.
author Hernández Rodríguez, José|||0000-0002-2357-2015
author_facet Hernández Rodríguez, José|||0000-0002-2357-2015
Martinez Valle, Fernando|||0000-0003-2673-2034
Acebes, X.|||0000-0001-6449-938X
Alerany, Carmen
Antón, Jordi|||0000-0002-8792-4219
Calvo Rojas, Gonzalo|||0000-0001-8860-6653
Corral, M.
Cruz, J.
Mangues, Ma Antonia|||0000-0002-1438-8515
Mateo, Jose|||0000-0002-4227-3256
Rivera, Josefa
Salazar, Albert
Francisco, R.
Mallol, Cristina|||0000-0002-2920-9612
Reig-Viader, Rita|||0000-0002-6893-6177
Tigri-Santiña, Ariadna|||0000-0003-2765-0065
Ricart, A.
Palau, F.
author_role author
author2 Martinez Valle, Fernando|||0000-0003-2673-2034
Acebes, X.|||0000-0001-6449-938X
Alerany, Carmen
Antón, Jordi|||0000-0002-8792-4219
Calvo Rojas, Gonzalo|||0000-0001-8860-6653
Corral, M.
Cruz, J.
Mangues, Ma Antonia|||0000-0002-1438-8515
Mateo, Jose|||0000-0002-4227-3256
Rivera, Josefa
Salazar, Albert
Francisco, R.
Mallol, Cristina|||0000-0002-2920-9612
Reig-Viader, Rita|||0000-0002-6893-6177
Tigri-Santiña, Ariadna|||0000-0003-2765-0065
Ricart, A.
Palau, F.
author2_role author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
dc.contributor.none.fl_str_mv Universitat Autònoma de Barcelona
dc.subject.none.fl_str_mv Comprehensive care
Focus group
Healthcare system
Patient journey
Person living with a rare disease
Rare diseases
topic Comprehensive care
Focus group
Healthcare system
Patient journey
Person living with a rare disease
Rare diseases
description Background: Rare diseases (RDs) are a heterogeneous group of complex and low-prevalence conditions in which the time to establish a definitive diagnosis is often too long. In addition, for most RDs, few to no treatments are available and it is often difficult to find a specialized care team. Objectives: The project "acERca las enfermedades raras" (in English: "bringing RDs closer") is an initiative primary designed to generate a consensus by a multidisciplinary group of experts to detect the strengths and weaknesses in the public healthcare system concerning the comprehensive care of persons living with a RD (PLWRD) in the region of Catalonia, Spain, where a Network of Clinical Expert Units (Xarxa d'Unitats de Expertesa Clínica or XUEC) was created and is being implemented since 2015. The additional primary aim was to propose recommendations to solve or improve the limitations found. Methods: A task force of 13 participants with multidisciplinary expertise on RDs completed a questionnaire and participated in two focus groups. A document was drafted with an item series of strengths and weaknesses of the healthcare system regarding the care of PLWRD, and a set of proposals or recommendations to overcome the problems identified. Results: The Catalan Government healthcare model of XUECs for the comprehensive care for RDs is currently valid and adapted to the needs of PLWRD and their families since its strategic optimal and operational framework, and it is aligned with the European Reference Networks (ERNs) thematic areas. The problems found in the current healthcare model were grouped into ten main areas: (1) the healthcare model for RDs; (2) coordination with primary healthcare providers and other tertiary and secondary hospitals; (3) access to and coordination with non-medical services; (4) the role of case manager in the XUEC; (5) genetic diagnosis; (6) undiagnosed patients; (7) treatments; (8) referring process, continuous follow-up, and transition from pediatric to adult centers; (9) research and education for professionals; and (10) associations of PLWRD and their families (patients' advocacy). The need for more resources was currently detected as the common factor for most of them. Ten key recommendations to improve the healthcare system regarding RDs were postulated. Conclusions: Catalonia has established a unique healthcare model for RDs in Spain, with clear strengths and advantages. However, after analyzing them, the experts suggested that new governmental political and administrative decisions are needed to ensure the efficient implementation of a healthcare plan for PLWRD in Catalonia, which could be applied to other regions and nations worldwide.
publishDate 2025
dc.date.none.fl_str_mv 2
2025-01-01
2025
2025-01-01
dc.type.none.fl_str_mv Article
http://purl.org/coar/resource_type/c_6501
VoR
http://purl.org/coar/version/c_970fb48d4fbd8a85
dc.type.openaire.fl_str_mv info:eu-repo/semantics/article
format article
dc.identifier.none.fl_str_mv https://ddd.uab.cat/record/320385
https://dx.doi.org/urn:doi:10.1186/s13023-024-03518-x
url https://ddd.uab.cat/record/320385
https://dx.doi.org/urn:doi:10.1186/s13023-024-03518-x
dc.language.none.fl_str_mv Inglés
eng
language_invalid_str_mv Inglés
language eng
dc.rights.none.fl_str_mv open access
http://purl.org/coar/access_right/c_abf2
https://creativecommons.org/licenses/by/4.0/
dc.rights.openaire.fl_str_mv info:eu-repo/semantics/openAccess
rights_invalid_str_mv open access
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eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.source.none.fl_str_mv reponame:Dipòsit Digital de Documents de la UAB
instname:Universitat Autònoma de Barcelona
instname_str Universitat Autònoma de Barcelona
reponame_str Dipòsit Digital de Documents de la UAB
collection Dipòsit Digital de Documents de la UAB
repository.name.fl_str_mv
repository.mail.fl_str_mv
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spelling Identification of strengths and weaknesses of the healthcare system for persons living with rare diseases in Catalonia (Spain), and recommendations to improve its comprehensive attentionthe "acERca las enfermedades raras" projectHernández Rodríguez, José|||0000-0002-2357-2015Martinez Valle, Fernando|||0000-0003-2673-2034Acebes, X.|||0000-0001-6449-938XAlerany, CarmenAntón, Jordi|||0000-0002-8792-4219Calvo Rojas, Gonzalo|||0000-0001-8860-6653Corral, M.Cruz, J.Mangues, Ma Antonia|||0000-0002-1438-8515Mateo, Jose|||0000-0002-4227-3256Rivera, JosefaSalazar, AlbertFrancisco, R.Mallol, Cristina|||0000-0002-2920-9612Reig-Viader, Rita|||0000-0002-6893-6177Tigri-Santiña, Ariadna|||0000-0003-2765-0065Ricart, A.Palau, F.Comprehensive careFocus groupHealthcare systemPatient journeyPerson living with a rare diseaseRare diseasesBackground: Rare diseases (RDs) are a heterogeneous group of complex and low-prevalence conditions in which the time to establish a definitive diagnosis is often too long. In addition, for most RDs, few to no treatments are available and it is often difficult to find a specialized care team. Objectives: The project "acERca las enfermedades raras" (in English: "bringing RDs closer") is an initiative primary designed to generate a consensus by a multidisciplinary group of experts to detect the strengths and weaknesses in the public healthcare system concerning the comprehensive care of persons living with a RD (PLWRD) in the region of Catalonia, Spain, where a Network of Clinical Expert Units (Xarxa d'Unitats de Expertesa Clínica or XUEC) was created and is being implemented since 2015. The additional primary aim was to propose recommendations to solve or improve the limitations found. Methods: A task force of 13 participants with multidisciplinary expertise on RDs completed a questionnaire and participated in two focus groups. A document was drafted with an item series of strengths and weaknesses of the healthcare system regarding the care of PLWRD, and a set of proposals or recommendations to overcome the problems identified. Results: The Catalan Government healthcare model of XUECs for the comprehensive care for RDs is currently valid and adapted to the needs of PLWRD and their families since its strategic optimal and operational framework, and it is aligned with the European Reference Networks (ERNs) thematic areas. The problems found in the current healthcare model were grouped into ten main areas: (1) the healthcare model for RDs; (2) coordination with primary healthcare providers and other tertiary and secondary hospitals; (3) access to and coordination with non-medical services; (4) the role of case manager in the XUEC; (5) genetic diagnosis; (6) undiagnosed patients; (7) treatments; (8) referring process, continuous follow-up, and transition from pediatric to adult centers; (9) research and education for professionals; and (10) associations of PLWRD and their families (patients' advocacy). The need for more resources was currently detected as the common factor for most of them. Ten key recommendations to improve the healthcare system regarding RDs were postulated. Conclusions: Catalonia has established a unique healthcare model for RDs in Spain, with clear strengths and advantages. However, after analyzing them, the experts suggested that new governmental political and administrative decisions are needed to ensure the efficient implementation of a healthcare plan for PLWRD in Catalonia, which could be applied to other regions and nations worldwide.Universitat Autònoma de Barcelona 22025-01-0120252025-01-01Articlehttp://purl.org/coar/resource_type/c_6501VoRhttp://purl.org/coar/version/c_970fb48d4fbd8a85info:eu-repo/semantics/articleapplication/pdfhttps://ddd.uab.cat/record/320385https://dx.doi.org/urn:doi:10.1186/s13023-024-03518-xreponame:Dipòsit Digital de Documents de la UABinstname:Universitat Autònoma de BarcelonaInglésengopen accesshttp://purl.org/coar/access_right/c_abf2Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, fins i tot amb finalitats comercials, sempre i quan es reconegui l'autoria de l'obra original.https://creativecommons.org/licenses/by/4.0/info:eu-repo/semantics/openAccessoai:ddd.uab.cat:3203852026-06-06T12:50:31Z
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