Landscape assessment to characterize baseline access and multilevel barriers to IMProve Access to CAR-T CD19 therapy (IMPACT study) across Europe

BACKGROUND/PURPOSE: Chimeric Antigen Receptor-T Cell Therapy (CAR-T) has revolutionized the treatment of B-cell precursor acute lymphoblastic leukemia (B-ALL), but its global availability is limited. This study assessed current access and barriers to CAR-T CD19 cell therapy for children across Europe.

METHODS: A country questionnaire developed by the EBMT PDWP, St. Jude Children's Research Hospital, and IBFM assessed current access to advanced therapies for B-ALL in Europe using Qualtrics software.

RESULTS: Data from 35 WHO-defined European countries (26 high-income, 9 upper middle-income) revealed a median of 5 pediatric hematology-oncology (PHO) centers per country (0.55 PHO centers/1 million inhabitants, range: 0.05-1.83). Hematopoietic stem cell transplantation (HSCT) facilities were available in 89% of countries (31/35). Sixty B-ALL cases were diagnosed annually per country (4 B-ALL children/100,000 children, range: 0.4-8.4). CAR-T CD19 therapy was available in 71% of countries; however, more than 50% of countries lacked clinical trials or international collaborations for pediatric CAR-T CD19 therapy. Most countries accepted foreign patients, but referrals remained limited, with 1-2 foreign patients treated annually per country. Seventeen countries expressed interest in a referral network, but only six had established mechanisms for domestic or international referrals.

CONCLUSION: Substantial disparities exist in access to advanced therapies for pediatric B-ALL across Europe. Although CAR-T CD19 therapy is available in most countries, gaps in clinical trials, collaborations, and referral systems limit equitable access. Efforts to improve infrastructure and establish referral networks are essential to enhance care for pediatric B-ALL patients.