Przemysław Marszałek: We already have modern and effective therapies for cystic fibrosis. However, some groups of patients are still waiting for access to them

Cystic fibrosis patients already have access to revolutionary therapy, but this is not the case for all patients. Which of them are still waiting for changes?

Until a few years ago, the group of cystic fibrosis patients numbered (depending on the registry) from 1,500 to 1,700. They all had the same problems: there was no causal treatment, the disease progressed dramatically, and life expectancy averaged twenty-some years. Modern and highly effective therapies have been available for some time now. They have also been available in Poland for a year, but not for everyone. There remains a group of patients for whom a reimbursement process is currently underway at the Ministry of Health. We are still waiting for a decision on access to the most effective therapies for about two hundred patients.

Cystic fibrosis is called a disease of a thousand faces. There are said to be many gene mutations responsible for its occurrence. Do all of them respond to drugs?

Unfortunately, no. Due to the fact that these mutations are many, modern science, medicine does not have a solution for all of them. But we strive to ensure that all patients in Poland have access to the most modern therapies. On the other hand, however, we must not forget about those for whom these therapies do not exist. For them, we must maintain the highest possible quality of conservative treatment, because there is a great deal of research and programs on the horizon, which, in my opinion, in the near future will allow us to say that cystic fibrosis has become, from a fatal disease, a chronic disease.

Thank you for the interview.