Landscape Assessment To Characterize Baseline Access And Multilevel Barriers To Improve Access To CAR-T Cell Therapy (Impact Study) Across The Euro Region
Abstract
Introduction: CAR-T therapy has revolutionized the treatment of pediatric BCP–ALL, but multilevel health system barriers restrict availability outside of high-resourced countries. To develop tailored strategies to improve access to CAR-T, this study assesses potential needs, current access, and barriers to CAR-T cell therapy for children across Europe.
Methodology: Through a collaborative effort between EBMT, IBFM, and St. Jude Children’s Research Hospital, a Qualtrics survey was developed to assess factors impacting CAR-T access at a country level.
Results: Respondents included 16 WHO-defined European countries (14 high-income, 2 low- and middle-income), reporting a median of 3.5 (IQR: 1.5-5) pediatric oncology centers per country and a median of 57.5 new cases of BCP-ALL per year (IQR: 25-98.8). While 88% (n=14) had hematopoietic stem cell transplantation facilities, only 75% (n=12) of countries currently offer CD19 CAR-T cell therapy within their country. The cost of CAR-T therapy was fully reimbursed by the government in 75% (9/12) of these countries and almost always reimbursed in 25%. Tisagenlecleucel was the most utilized CAR-T source, available in 83% (n=10) of the countries.
Despite this, only 42% of countries reported active CAR-T clinical trial participation or international collaboration for children to receive CAR-T cell therapy, and only four countries had academic CAR-T clinical trials. While most countries (92%) indicated they accepted foreign patients, the mean number of foreign patients treated annually was 2.8 (SD = 1.8) per country. Additionally, 12 countries expressed interest in joining a referral network to enhance collaboration.
Conclusion: This interim analysis highlights gaps in clinical trial availability, international collaboration, and referral networks that impact CAR-T availability in Europe. However, early data from the survey demonstrates an opportunity to improve the representation of experience from LMICs in the region.
How to Cite
Oszer A, Rössig C, Galimard JE, Wardell J, Perez-Martinez A, Styczyński J, et al. Landscape Assessment To Characterize Baseline Access And Multilevel Barriers To Improve Access To CAR-T Cell Therapy (Impact Study) Across The Euro Region. OncoDaily Med J. 2025. doi:10.69690/ODMJ-018-0425-3980