土耳其学术性 CAR-T 细胞治疗的国家视角：土耳其血液学会细胞与基因治疗科学小组委员会（TSH-CGT SSC）报告

This study evaluated the current status, clinical capacity, and key barriers related to chimeric antigen receptor T-cell (CAR-T) therapy in T rkiye with the aim of guiding national policy development. From June 2023 to March 2024, the Scientific Subcommittee on Cell and Gene Therapies of the Turkish Society of Hematology conducted two nationwide online surveys and held three multidisciplinary meetings with contributions from hematologists, basic scientists, and key stakeholders. Survey findings were integrated with the meeting outputs and recent regulatory developments. Surveys indicated a marked gap between the estimated annual need (about 507 patients) and actual access to CAR-T therapy. Between 2019 and March 2024, only 23 patients in T rkiye received CAR-T therapy. Major barriers included high treatment costs, insufficient infrastructure, and a limited number of trained personnel. Expert meetings further highlighted challenges with regulatory pathways, reimbursement, and the need for standardized production and clinical protocols. Despite these limitations, participants demonstrated strong clinical readiness and consensus on the need to improve access. The implementation of CAR-T therapy in T rkiye continues to be impeded by structural and economic limitations. It is imperative to develop a strategic national roadmap that encompasses: 1) the establishment of a national academic CAR-T network, 2) the securing of sustainable funding and legal frameworks, 3) the implementation of hospital-exemption pathways, 4) the development of a centralized registry, 5) the expansion of structured training programs, and 6) the enhancement of international collaborations. These measures are fundamental for the sustainable integration of this therapy into clinical practice. Bu al man n amac , T rkiye de kimerik antijen resept rl T-h cre (CAR-T) tedavisinin mevcut durumunu, klinik kapasitesini ve ba l ca k s tlay c fakt rleri de erlendirmek ve ulusal politika geli tirme s re lerine bilimsel veri sa lamakt r.

Haziran 2023-Mart 2024 d neminde, T rk Hematoloji Derne i H cre ve Gen Tedavileri Bilimsel Alt Komitesi taraf ndan lke ap nda iki evrimi i anket uygulanm ve hematologlar, temel bilimciler ile ilgili payda lar n kat l m yla multidisipliner toplant ger ekle tirilmi tir. Elde edilen anket verileri, toplant kt lar ve g ncel d zenleyici geli melerle b t nc l olarak analiz edilmi tir. Anket bulgular , y ll k tahmini ihtiya (yakla k 507 hasta) ile CAR-T tedavisine eri im aras nda belirgin bir uyumsuzluk oldu unu ortaya koymaktad r. 2019 ile Mart 2024 tarihleri aras nda T rkiye de yaln zca 23 hastaya CAR-T tedavisi uygulanm t r. Ba l ca k s tlay c fakt rler; y ksek tedavi maliyetleri, yetersiz kurumsal altyap ve s n rl say da e itimli insan kayna olarak belirlenmi tir. Uzman toplant lar nda ayr ca d zenleyici s re lerdeki belirsizlikler, geri deme mekanizmalar ndaki yetersizlikler ve standartla t r lm retim ile klinik uygulama protokollerine duyulan gereksinim vurgulanm t r.

T m bu s n rl l klara kar n, kat l mc lar g l bir klinik haz rl k d zeyine sahip olduklar n ve eri imin art r lmas gereklili i konusunda y ksek d zeyde g r birli i bulundu unu ifade etmi tir. T rkiye de CAR-T tedavisinin klinik uygulamaya entegrasyonu, yap sal ve ekonomik s n rl l klar nedeniyle halen k s tl d r. Bu ba lamda, s rd r lebilir bir ulusal stratejik yol haritas n n geli tirilmesi kritik nem ta maktad r.

Bu yol haritas kapsam nda; 1) ulusal akademik CAR-T a n n olu turulmas , 2) s rd r lebilir finansman modelleri ve destekleyici yasal d zenlemelerin hayata ge irilmesi, 3) hastane muafiyeti (hospital exemption) mekanizmalar n n uygulanmas , 4) merkezi bir ulusal kay t sisteminin kurulmas , 5) yap land r lm e itim programlar n n yayg nla t r lmas ve 6) uluslararas i birliklerinin g lendirilmesi temel ncelikler olarak ne kmaktad r.