2023-12-14 09:38:51
The report “Current challenges in access to treatments for
rare diseases (EERR)” shows the main obstacles to be overcome to guarantee access to the medications necessary for these pathologies in Spain, while offering proposals for improvement.
EFE/ARCHIVE/Villar López
The average waiting time in Spain to access medicines aimed at rare diseases (EERR) is 786 days. Furthermore, of the total number of those approved throughout Europe, only 44% are approved in the country.
The Annual Report on Indications for Access to Innovative Therapies in Europe (WAIT Indicator) shows that Spain does not have a specific financing and pricing procedure for medicines aimed at rare diseases (EERR). This is shown in the report “Current challenges in access to treatments for rare diseases”.
This document has been prepared by a committee of experts composed of doctors Francesc Palau Martínez, Jesús Díez Manglano, José Luis Poveda Andrés, Álvaro Hidalgo Vega and Antonio Cabrera Cantero. As well as with the coordination of Dr. Enrique Castellón.
Likewise, it has been discussed and reviewed, in a second phase, by representatives of the field of health management and administration of Andalusia, Catalonia, the Valencian Community, Galicia and the Region of Murcia.
The document has the endorsement of the Spanish Society of Internal Medicine (SEMI), the ObserveMHe (Orphan Medicines Observatory), the GIES (Economy and Health Research Group of the University of Castilla La Mancha), as well as with the participation of the Spanish Federation of Rare Diseases (FEDER). He has also had the collaboration of Boehringer Ingelheim Spain.
Rapid access to treatments for REE is one of the objectives of the report. EFE/Paco Torrente
Report recommendations
The report “Current challenges in access to treatments for rare diseases” advocates the creation of a centralized body. This would make it easier for patients to access the medications they need. In addition, it would be equipped with an Advisory Council, which has its own financing and is autonomous in nature.
This centralized body is based on models already in use for transplants or CAR-T therapies. It seeks to guarantee the financing, availability and access of new treatments for RRD, betting on equity in the process.
Some of its functions would be the promotion of agile access to treatments, referring each patient to the most appropriate circuit for their disease or ensuring compliance with the objectives set in the Rare Diseases Strategy of the National Health System (SNS).
New financing methods
One of the barriers identified in the report is budget uncertainty. In addition to the delay in the evaluation and financing processes or lack of equity.
The need for financing is one of the problems that must be overcome to guarantee access to the necessary treatments for RRD.
For this reason, the report proposes innovative financing agreements such as payment by results, population-based or with a spending ceiling. The objective is to accelerate the transfer of innovation to the patient in Spain, where in 2022 one of every four clinical trials that were carried out was to combat a rare disease.
“The shortage of treatments for rare or ultra-rare diseases should encourage us to continue working to eliminate barriers to access without delay. To achieve this objective, we must understand the scientific, clinical and economic particularities of the RREs and commit to renewed structures and procedures that speed up the arrival of treatments to patients,” says the coordinator of the report, Dr. Enrique Castellón.
Cover of the rare diseases report. Image provided
Improve diagnosis
Another of the The objectives that are reflected in the report on rare diseases is the improvement in the diagnosis of these pathologies. Thus, the importance of Primary Care (PC) in the SNS is emphasized, since it is a key actor in ensuring a quick and accurate diagnosis.
To streamline these processes and improve the field of action of the PC, the protocols must arise from joint work between the Autonomous Communities, Scientific Societies and Patient Associations.
In addition, different improvements are reflected that can be very beneficial throughout this process:
Form and have bidirectional systems in the care circuits between Primary Care, Specialized Care and Reference Centers. Promote Integrated Assistance programs (PAI) in the field of rare diseases. Include training plans around RR for health professionals and promote information and training programs for patients and families in the health, socio-labor and educational fields. Guarantee a multidisciplinary socio-health approach through the promotion of EERR coordination plans. These would be done in collaboration with scientific societies and patient associations. Facilitate patients’ access to reference units and European networks for their pathologies. In addition, it is also desired to implement virtual visit systems with the specialists of these networks. Develop information campaigns aimed at the general population to raise awareness and publicize these types of diseases.
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