Catalonia is one of the four regions that already administers Vyjuvek, the gene treatment for epidermolysis bullosa, while the Interministerial Pricing Commission has yet to approve its state funding.
The epidermolysis bullosa, known as butterfly skin, now has treatment in Catalonia. The Health Department offers Vyjuvek, a gene therapy that until now was only available through the exceptional route of medications in special situations. According to elespanol.com, three other regions have joined this initiative: Andalusia, Castile and León, and Canary Islands.
The drug is applied as a cream, but it contains modified viruses that introduce corrected copies of the COL7A1 gene into skin cells. This allows chronic wounds suffered by these patients to heal. The therapy is not yet funded at the state level because the Interministerial Pricing Commission is still negotiating with the pharmaceutical company.
The head of Research at the patient association DEBRA, Núria Tarrats, explained that negotiations are progressing: "The Ministry's negotiation with the pharmaceutical company is going well. They are gradually finding that point of agreement for it to enter the National Health System (SNS). There is good willingness, and work is being done to achieve this as soon as possible."
Meanwhile, the situation is uneven. Aragon and Balearic Islands have already begun the procedures to offer the treatment, but two other autonomous communities have rejected it, according to this newspaper. Tarrats insists on the urgency: "The urgency of accelerating the inclusion of this innovative treatment in the SNS service portfolio must be understood. We need to end this inequity of access; it is essential that anyone who may need it can access it smoothly and quickly."
In Spain, there are around 500 people with this disease, of whom about 200 would be candidates for the drug. These are those with mutations in the COL7A1 gene and in advanced stages, with chronic wounds and risk of infection.
The most publicised case is that of Leo, a 12-year-old boy from Seville who has become the face of the disease. He has received more than 20 doses, and his progress is favourable: he has gained two kilos and has grown two centimetres, and the wound on his chest has improved significantly. The treatment has also been received by Adrián, a 22-year-old from Malaga, and two people in the Canary Islands.
To access Vyjuvek in Catalonia, the dermatologist must submit the request to the hospital pharmacy service, which, if approved, forwards it to the Health Department. It is a process that is activated case by case and, for now, is only available to patients in the communities that have decided to fund it.
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