Ethics and access in pediatric CAR T-cell therapy

Advanced gene and cell therapies, including CAR T-celltreatments such as tisagenlecleucel (also known as Kymriah), have significantlyimproved outcomes for pediatric patients with relapsed or refractory B-cellAcute Lymphoblastic Leukemia (ALL). However, access, in the form of clinicaleligibility criteria, institutional capacity, and financial barriers, continuesto impede families and patients from pursuing these options. Currentqualifications require refractory disease or multiple relapses following theexhaustion of conventional treatment options before a patient becomes eligiblefor CAR T-cell therapy, delaying access to treatments with demonstratedefficacy. While designed to save lives, health policy implementations andprotocols limit the maximum impact of these therapies, while potentiallycontributing to disease progression for patients and higher financial burdenson families. This poster applies a bioethical approach to the currentaccessibility standards of pediatric CAR T-cell therapy, addressing the fourmain bioethical imperatives: autonomy, beneficence, non-maleficence, andjustice. From a patient advocacy perspective, this poster highlightsopportunities for improvement specifically aligned with patient-centered needsin advanced gene cell therapy development, implementation, and practice.