dc.contributor.author | Zozaya, N. | |
dc.contributor.author | Villaseca, J. | |
dc.contributor.author | Abdalla, F. | |
dc.contributor.author | Ancochea, A. | |
dc.contributor.author | Málaga, I. | |
dc.contributor.author | Trapero-Bertran, Marta | |
dc.contributor.author | Martín-Sobrino, N. | |
dc.contributor.author | Delgado, O. | |
dc.contributor.author | Ferré, P. | |
dc.contributor.author | Hidalgo-Vega, A. | |
dc.date.accessioned | 2023-03-21T14:17:10Z | |
dc.date.available | 2023-03-21T14:17:10Z | |
dc.date.issued | 2023 | |
dc.identifier.citation | Zozaya, N.; Villaseca, J.; Abdalla, F. [et al.]. Strategic discussion on funding and access to therapies targeting rare diseases in Spain: an expert consensus paper. Orphanet Journal of Rare Diseases, 2023, 18, 41. Disponible en: <https://ojrd.biomedcentral.com/articles/10.1186/s13023-023-02635-3>. Fecha de acceso: 21 mar. 2023. DOI: 10.1186/s13023-023-02635-3 | ca |
dc.identifier.issn | 1750-1172 | ca |
dc.identifier.uri | http://hdl.handle.net/20.500.12328/3628 | |
dc.description.abstract | Background: In recent years, significant advances have been made in the field of rare diseases (RDs). However, there is a large number of RDs without specific treatment and half of these treatments have public funding in Spain. The aim of the FINEERR project was to carry out a multidisciplinary strategic discussion on the challenge of funding and access to RD-targeted drugs in Spain, in order to agree on specific proposals for medium-term improvement and hence support decision-making in the Spanish National Healthcare System (SNHS). Results: The FINEERR Project was organized around a CORE Advisory Committee, which provided an overview, agreed on the design and scope of the project, and selected the members within each of four working groups (WG). Overall, 40 experts discussed and reached a consensus on different relevant aspects, such as conditioning factors for initial funding and access, evaluation and access to RD-targeted therapies, funding of these therapies, and implementation of a new funding and access model. From these meetings, 50 proposals were defined and classified by their level of relevance according to the experts. A descriptive analysis of responses was performed for each proposal. Thereafter, experts completed another questionnaire where they ranked the 25 most relevant proposals according to their level of feasibility of being implemented in the SNHS. The most relevant and feasible proposals were to improve: process of referral of patients with RDs, control over monitoring mechanisms, and communication between healthcare professionals and patients. Conclusions: The FINEERR project may provide a starting point for stakeholders involved in the process of funding and access to RD-targeted therapies in Spain to provide the necessary resources and implement measures to improve both the quality of life and life expectancy of patients with RDs. | en |
dc.format.extent | 14 | ca |
dc.language.iso | eng | ca |
dc.publisher | Springer Nature | ca |
dc.relation.ispartof | Orphanet Journal of Rare Diseases | ca |
dc.relation.ispartofseries | 18 | |
dc.relation.uri | https://ojrd.biomedcentral.com/articles/10.1186/s13023-023-02635-3 | ca |
dc.rights | This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated in a credit line to the data. | en |
dc.rights.uri | https://creativecommons.org/licenses/by/4.0/ | |
dc.subject.other | Reemborsament | ca |
dc.subject.other | Finançament | ca |
dc.subject.other | Accés | ca |
dc.subject.other | Malalties rares | ca |
dc.subject.other | Medicaments orfes | ca |
dc.subject.other | Espanya | ca |
dc.subject.other | Refecció | ca |
dc.subject.other | Mesures | ca |
dc.subject.other | Regulatòria ciència | ca |
dc.subject.other | Reembolso | es |
dc.subject.other | Financiación | es |
dc.subject.other | Acceso | es |
dc.subject.other | Enfermedades raras | es |
dc.subject.other | Medicamentos huérfanos | es |
dc.subject.other | España | es |
dc.subject.other | Reflexión | es |
dc.subject.other | Medidas | es |
dc.subject.other | Regulatorio ciencia | es |
dc.subject.other | Reimbursement | en |
dc.subject.other | Funding | en |
dc.subject.other | Access | en |
dc.subject.other | Rare diseases | en |
dc.subject.other | Orphan drugs | en |
dc.subject.other | Spain | en |
dc.subject.other | Refection | en |
dc.subject.other | Measures | en |
dc.subject.other | Regulatory science | en |
dc.title | Strategic discussion on funding and access to therapies targeting rare diseases in Spain: an expert consensus paper | en |
dc.type | info:eu-repo/semantics/article | ca |
dc.description.version | info:eu-repo/semantics/publishedVersion | ca |
dc.rights.accessLevel | info:eu-repo/semantics/openAccess | |
dc.embargo.terms | cap | ca |
dc.subject.udc | 61 | ca |
dc.subject.udc | 615 | ca |
dc.identifier.doi | https://doi.org/10.1186/s13023-023-02635-3 | ca |