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On National Cystic Fibrosis Day, commemorated today, April 28, 2021, the collective of people with Cystic Fibrosis demands the Covid Vaccine NOW.
Kaftrio / Trikafta, a necessary medicine along with the vaccine
In addition, the Respiralia Foundation and the Cystic Fibrosis Balearic Association request that the drug Kaftrio / Trikafta, which could benefit more than 75% of people with CF, be approved for funding and prioritized for vaccination against COVID-19.
Our claims
On the occasion of the National Cystic Fibrosis Day, commemorated today April 28 (fourth Wednesday of April), the Respiralia Foundation and the Cystic Fibrosis Balearic Association join the Cystic Fibrosis Spanish Federation to make visible the situation of people with Cystic Fibrosis in our country, who still do not have access to the drug Kaftrio; a treatment that, although not cure, paralyzes the deterioration caused by the disease and improves considerably their quality of life. This is especially important in the midst of this COVID-19 pandemic, which places people with Cystic Fibrosis among the risk groups, but without immediate access to the vaccine due to their younger profile.
Why
Cystic Fibrosis is one of the most frequent serious genetic diseases and its incidence in our country is estimated at around one in every 5,000 births (6,400 in the Balearic Islands), while one in every 35 people are healthy carriers of the disease. It is a chronic disease of genetic origin that affects different organs and, above all, has important consequences in the lungs, which makes them more vulnerable to a possible infection by COVID-19.
In recent years much progress has been made in the knowledge and treatment of the disease, but, in spite of that, it is still a pathology without cure. Therefore, it is very important to have access to the latest treatments, such as CFTR protein modulators, which represent a considerable improvement in the quality of life of people with Cystic Fibrosis, since, although they do not cure, they slow down the deterioration caused by the disease.
The latest of these modulatory treatments is the drug Kaftrio (ivacaftor / tezacaftor/ elexacaftor), which was approved by the European Medicines Agency (EMA) in June 2020 and authorized by the European Commission for marketing in August 2020. At that time, it was indicated only for people with Cystic Fibrosis aged 12 years and older with one F508del mutation and one minimal function (MF) mutation or two F508del mutations in the CFTR gene. But recently, in March 2021, the EMA has issued a positive opinion to extend the indication of the drug to people with Cystic Fibrosis 12 years and older with at least one copy of the F508del mutation, regardless of what their other mutation is.
With this new indication, Europe is on a par with that approved by the FDA in the United States and expands access to Kaftrio to thousands more people, as it is the most common mutation in Cystic Fibrosis. In Spain, specifically, according to the “Spanish Cystic Fibrosis Registry” prepared by the Spanish CF Society, more than 75% of people with Cystic Fibrosis have at least one F508del mutation and, therefore, would be eligible for this treatment.
EMA, an example to follow
The urgency and agility with which EMA has carried out the entire process of evaluation and approval of this drug, as well as its new indication, denote the effort made, both by the Agency and CF Europe, so that people with Cystic Fibrosis throughout Europe can have rapid access to this innovative treatment at a particularly sensitive time for them, due to the COVID-19 pandemic.
Therefore, the Respiralia Foundation and the Cystic Fibrosis Balearic Association request that Spain follows the example of other European countries, in which the drug Kaftrio is already available for all those who need it, as is the case of the United Kingdom, Ireland, Denmark, Finland, Switzerland, Luxembourg, Slovenia, or which have a positive evaluation pending approval for its commercialization, as is the case in Germany, France and Portugal. We ask the Spanish Agency of Medicines and Health Products (AEMPS) to make an effort to speed up the process of preparing the Therapeutic Positioning Report (TPR), as well as the General Directorate of Pharmacy and the Ministry of Health, to speed up negotiations so that an agreement on the financing of this drug can be reached as soon as possible. We also ask Vertex Pharmaceuticals, the laboratory that markets the drug, to make a special effort to take into account the current pandemic situation in which we find ourselves and the sustainability of our National Health System when setting their prices.
Claim of our President
Ana Amate, president of the Cystic Fibrosis Balearic Association asks for more understanding towards the situation of people with Cystic Fibrosis: “Europe and Spain have shown that it is possible to reduce bureaucratic times and streamline negotiation procedures in this pandemic year, where we are seeing record access to vaccines against COPD-19. We are concerned that the health of 75% of people with Cystic Fibrosis in Spain continues to deteriorate when there is an EMA-approved treatment that could prevent it. In Spain, we have the experience of drugs such as Kalydeco, Orkambi and Symkevi in which the results have been very good but which are aimed at a small group of people and have taken years to finance. Kaftrio is the first one that targets a larger number of people and shows very good results, even better than those mentioned above. Its access can radically change the lives of people with CF by alleviating the symptoms of their disease, improving their nutritional status and reducing the number of acute lung infections, which translates into fewer hospitalizations and may even prevent placement on the waiting list for a two-lung transplant.
We need Covid Vaccine NOW
This is a particularly high priority at this time of year when hospital pressure is so high. This added to the risk that people with Cystic Fibrosis have against a possible infection by COVID-19, and seeing that they are not currently prioritized in the vaccination strategy, is a great concern for us. There are more than 70 people with CF in the Balearic Islands and last year the Congress of Deputies issued an Institutional Declaration in support of all of them in which the House committed, among other things, to facilitate access to the best available treatment. This commitment was recently adopted by the Balearic Government through another institutional declaration. We ask the Government, the Ministry of Health and the Balearic Government to transform words into deeds, speeding up as much as possible the negotiation with the laboratory so that the access to Kaftrio is not delayed and vaccinating as a priority the Cystic Fibrosis collective, as it is being done in Catalonia since last week“.
