Strategic discussion on funding and access to therapies targeting rare diseases in Spain: an expert consensus paper

Strategic discussion on funding and access to therapies targeting rare diseases in Spain: an expert consensus paper

Year: 2023

Publisher: Orphanet Journal of Rare Diseases

Abstract: In recent years there have been important advances in the field of rare diseases (RDD). However, there is a large number of rare diseases without specific treatment and half of these treatments are publicly funded in Spain. The aim of the FINEERR project was to carry out a multidisciplinary strategic discussion on the challenge of funding and access to medicines for RD in Spain, in order to reach a consensus on specific proposals for improvement in the medium term and thus support decision-making in the National Health System (NHS).

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 of each of the four working groups (WGs). In total, 40 experts discussed the conditioning factors of financing, evaluation and access to therapies targeting RDD and agreed on 50 improvement measures, which they ranked according to their level of relevance. Subsequently, the experts ranked the 25 most relevant proposals by their level of feasibility of implementation in the NHS. The most relevant and feasible proposals were to improve the process of referring patients with RRD, the control of follow-up mechanisms and communication between health professionals and patients.

The FINEERR project can be a starting point for the agents involved in the process of financing and access to therapies for RD in Spain to provide the necessary resources and implement measures to improve both the quality of life and life expectancy of patients with RD.

Keywords: Reimbursement, Financing, Access, Rare diseases, Rare diseases, Orphan drugs, Spain, Reflection, Measures, Regulatory science.

How to cite: 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 J Rare Dis 18, 41 (2023). https://doi.org/10.1186/s13023-023-02635-3