期刊论文详细信息
Orphanet Journal of Rare Diseases
Strategic discussion on funding and access to therapies targeting rare diseases in Spain: an expert consensus paper
Research
N. Martín-Sobrino1  M. Trapero-Bertran2  A. Ancochea3  I. Málaga4  F. Abdalla5  J. Villaseca5  N. Zozaya5  O. Delgado6  A. Hidalgo-Vega7  P. Ferré8 
[1] Castilla y León Health Management Department, Technical Director of Pharmacy, Valladolid, Spain;Department of Basic Sciences, Universitat Internacional de Catalunya (UIC Barcelona), Barcelona, Spain;Federación Española de Enfermedades Raras (FEDER), Madrid, Spain;Head of the Neuropediatrics Unit, Asturias Central University Hospital, Asturias, Spain;Health Economics Department, Weber, C/ Moreto 17, 5D, 28014, Madrid, Spain;Pharmacy Service, Son Espases University Hospital, Palma, Balearic, Spain;President of Weber Foundation, Madrid, Spain;Technical Advisor On Temporal Leave, Ministry of Health, Madrid, Spain;
关键词: Reimbursement;    Funding;    Access;    Rare diseases;    Orphan drugs;    Spain;    Reflection;    Measures;    Regulatory science;   
DOI  :  10.1186/s13023-023-02635-3
 received in 2022-09-21, accepted in 2023-02-06,  发布年份 2023
来源: Springer
PDF
【 摘 要 】

BackgroundIn 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).ResultsThe 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.ConclusionsThe 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.

【 授权许可】

CC BY   
© The Author(s) 2023

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