Cystic Fibrosis Association (Kifder) Marks Its 10th Anniversary

The Cystic Fibrosis Association (Kifder), which has been operating since 2012 to raise awareness about cystic fibrosis—a fatal genetic disease affecting multiple systems including the respiratory and digestive systems and causing infections and damage—and to work with physicians, public authorities and other relevant stakeholders to identify needs during treatment and take necessary steps, is celebrating its 10th anniversary. Kifder Board Chairman İlknur Görgün: "In countries where access to modulator medications is provided, the average life expectancy and quality of life of cystic fibrosis patients increases significantly." In a statement marking the association's 10th anniversary, Kifder Board Chairman İlknur Görgün stated that they will henceforth celebrate 8 September as "Cystic Fibrosis Awareness Day" and bring current issues faced during the treatment process to public attention. Görgün noted that while the precise prevalence in Turkey is unknown, cystic fibrosis—a genetic and fatal disease—occurs in approximately 1 out of every 6,000 people.

Görgün, noting that the association founded by patients and their relatives has achieved many important successes over the past 10 years, including having the disease added to the newborn screening program beginning in 2012, explained as follows:

"The treatment process for babies born with this disease continues throughout their lifetime. We know that the number of patients currently undergoing treatment is around 3,500. Nevertheless, we are witnessing increases in average life expectancy and quality of life in cystic fibrosis globally with each passing year. The most important reason for this increase is access to drugs classified as modulators, which have revolutionized cystic fibrosis treatment."

According to cystic fibrosis patient registries maintained globally, Görgün drew attention to the fact that while the adult patient ratio is 54.6% in the United States and 51.3% in Europe, it remains at only 7% in Turkey, with the most important reason being that patients in our country do not have access to next-generation medications, and continued as follows:

"Modulator medications used abroad for cystic fibrosis have become a great hope for our patients and their families. The triple combination modulator treatment Elexacaftor + Tezacaftor + Ivacaftor, approved by the FDA on 21 October 2019, was received with excitement by the medical community and ourselves. We witnessed significant improvements in respiratory function tests, substantial reductions in sweat salt concentrations and hospitalizations for pulmonary exacerbations, and remarkable improvements in quality of life in cystic fibrosis patients aged six and over carrying the DeltaF508 mutation on one allele."  

"Every moment without medication brings our children one step closer to death!"

"However, since there is no reimbursement coverage for cystic fibrosis modulator medications in our country, we were forced to file lawsuits to gain access to these drugs. Because every moment without medication brings our children one step closer to death. Although we have gained the right to access the drug through court decisions, this is not a method we prefer. Our request is that these medications be covered by reimbursement as soon as possible. We are concerned about the health of our patients whose medication use has been interrupted due to drug supply problems. Another problem is the issues we have encountered during the legal process. One of these is the debt notices sent by the Social Security Institution to those who filed lawsuits recently. We wish to emphasize that none of us have the financial capacity to make such payments. We will continue our discussions with lawmakers and efforts to place ivacaftor, the first modulator drug made available in 2012, and the combination of Elexacaftor + Tezacaftor + Ivacaftor, approved in 2019, on the foreign drug list Annex 4C, which is a patient-based payment system, and to ensure access to these drugs for patients with matching mutations."