Constança Bradell, the 24-year-old who has cystic fibrosis and who asked on social media for access to Katfrio, an innovative drug used to treat this rare disease, has already received the innovative drug.
In a post on social media, the young woman appears in a photo with a box of “Kaftrio” and thanks the wave of solidarity.
“I have imagined so many times what I would feel if that day came and the truth is that no matter how hard I try, I cannot put into words what I am feeling. First of all I would like to thank everyone from the bottom of my heart who made this possible. The wave of solidarity that has arisen since the moment I shared my desperate situation, ”writes the young woman, adding that without her I would not have the medicine in my hand. “It’s surreal to have the miraculous ‘life changing’ pill with me and remember that I was between life and death a week ago,” he says. And he appeals: “Together we will fight until it is approved by Infarmed and accepted into the NHS.”
The drug that made Constança Bradell catch on on social media has several peculiarities. Kaftrio is the only drug available that has been shown to be highly effective in 80% of cystic fibrosis patients, but has not yet been approved by Infarmed in Portugal.
Infarmed approved 14 applications for a special permit (AUE) from Kaftrio for the treatment of cystic fibrosis, including applications from the Centro Hospitalar Universitário Lisboa Norte (CHULN). The approvals included Constança Bradell’s application to use the drug, who exposed on social media the delay in approval of the treatment for cystic fibrosis, a genetic, hereditary and rare disease in Portugal. The AUE are provided by the pharmaceutical company, while the national agency for the drug, in this case Infarmed, does not approve them.