On World Rare Disease Day, the Defensor del Pueblo reiterates its commitment to the defence of the rights of people affected by these diseases and to the need of supporting increased means of medical research.
The institution welcomes the fact that -in 2015- the Ministry of Health pushed a procedure for the identification, reception and publication of good practices in the treatment of rare diseases in the National Health System.
In 2014, that same Ministry already approved the creation of the “Strategy for Rare Diseases in the National Health system” to promote recognition, prevention, diagnosis, treatment and investigation in this area.
Within the line of work of the institution in this area, the Defensor is paying special attention to the progressive designation of centres, services and reference units (CSUR) for pathologies in this category.
Highlighted actions
* The Defensor del Pueblo took action before the Ministry of Health in reaction to the delays and difficulties in the effective commercialization of medicines designated as “orphan drugs” or “ultra-orphan drugs,” which are used in the treatment of rare diseases. Following the initial guaranteed authorization by the European Medical Agency, the process of inclusion of this type of drugs into the pharmaceutical regimen of the National Health Agency could take months, pending reaching a financing agreement. During this period the demand will accentuate the impossibility of patients in the majority of cases to access these drugs for their own treatment. According to a report from the Ministry, Spain will commercialize 52 pharmaceuticals of this type out of the 67 that exist in Europe.
* The financing of the medicines used to fight these diseases has prompted complaints. The action of the Defensor has allowed that the use of certain drugs be evaluated and in some cases has sped up the commercialization process.
* Throughout 2015, the institution took concrete action in relation to two pharmaceutical companies, indicated respectively in the treatment of Morquio Syndrome and Duchenne Muscular Dystrophy. The relevant health administrations responded positively.
* The institution has also worked to ensure that people affected by these diseases are referred to specialized centres. Just as, in Andalucía, following the actions of the Defensor, a minor was referred to a specialized centre to receive a second clinical opinion.
* Moreover, the Defensor has intervened so that specific and structural measures to diagnose and treat these diseases are adopted.
* The institution has taken interest in the situation and need for educational support of students with rare diseases.
