The Ministry of Health announced today that treatment for cystic fibrosis with CFTR modulators has been significantly expanded. Currently, 126 patients are receiving the therapy, compared with just three in 2021.
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According to the Ministry of Health, all patients who meet the medical criteria are eligible, including children from the age of two and people with rare genetic mutations. Macedonia is among the countries in the region where the therapy is available from the age of two.
The ministry said that increased funding from the state budget and the Health Insurance Fund, together with successful negotiations with manufacturer Vertex, made it possible to expand the treatment.
An important step in early diagnosis was newborn screening. It began as a pilot project in 2018 and, since April 2019, has been implemented as a national program. This makes it possible to identify the disease at the earliest stages of life.
The fund enabled the first three patients to begin treatment with Kalydeco in 2021. Trikafta was subsequently made available, initially for patients with advanced lung damage. At the end of 2023, 12 people were receiving the therapy, and the National Guidelines for the Treatment of Cystic Fibrosis were published. During 2024, treatment was expanded to all eligible patients over the age of 12, and children aged 6 to 11 began to be included.
The Minister of Health said that the number of patients receiving therapy had risen from three in 2021 to 126 today. He stressed that behind each of these numbers are a child, a family and a new hope, attributing the achievement to continued institutional support and the goal of ensuring modern, targeted treatment for all patients who meet the medical criteria.
Macedonia’s cystic fibrosis centers are participating in the twinning project of the European Cystic Fibrosis Society (ECFS). The pediatric service is cooperating with Charité University Hospital in Berlin, while Papworth Hospital in Cambridge is supporting the development of care for adult patients.
Dr. Charles Haworth, an adult pulmonologist at Papworth Hospital, said that the new CFTR modulators had improved patients’ condition and life expectancy. According to him, the number of adults with cystic fibrosis is growing, which represents a major success. He said that on every visit to Skopje he observes clear progress in treatment conditions, doctors’ skills and health outcomes.
Dr. Miriam Stahl, a pediatric pulmonologist at Charité, explained that the teams hold monthly online meetings and consult on patients who experience difficulties with treatment. She said that care for people with cystic fibrosis in Skopje had made good progress.
Patient Fiki Gaspar is evidence of the change following treatment. Before receiving the therapy, he needed oxygen and spent almost half the year in hospital. Now, he says, he no longer requires oxygen support, and his lung function has increased from 15% to more than 55%. He has a family and a son, works full time and plays sports.
The Ministry of Health said that a total of 138 patients with cystic fibrosis have been registered in Macedonia. The country is part of the European Cystic Fibrosis Patient Registry, through which results are monitored and compared with European standards.
