Significant disparities exist in the availability and funding of CAR-T therapy for pediatric patients with B-ALL across Europe, highlighting the need for a standardized regional referral framework.
More than one-quarter of surveyed European countries did not have access to CD19 chimeric antigen receptor (CAR) T-cell therapy for pediatric patients with B-cell acute lymphoblastic leukemia (B-ALL), according to the result of the IMPACT study published in JCO Global Oncology.
“Gaps in clinical trials, collaboration, and referral systems limit equitable access,” the <a href="https://absafricatv.com/new-mission-hits-right-note-with-boost-for-chip-design-research/” title=”New mission hits right note with boost for chip design, research”>researchers wrote in their report. “Efforts to improve infrastructure and establish referral networks are essential to enhance care for patients with pediatric B-ALL.”
As part of the cross-sectional IMPACT study, researchers surveyed 36 WHO-defined European countries with a questionnaire developed by the European Group for Blood and Marrow Transplantation Pediatric Diseases Working Party, St Jude Children’s Research Hospital, and IBFM. Of the countries, 27 were high-income and the remaining 9 were upper-middle-income.
There were a median of 5 pediatric hematology/oncology centers present in each country (range, 0.05-1.83). There were 32 (89%) countries with hematopoietic stem cell transplantation (HSCT) facilities, with the median number of annual B-ALL transplants of 5 (range, 1-80).
There were 26 (72%) countries that reported availability of CD19 CAR-T therapy; however, 3 countries (Italy, Belgium, and Switzerland) reported limited patient access. Commercially purchased product was the most commone manufacturing facilities. Open CD19 CAR-T therapy trials were reported in 11 countries (31%)
Acceptance of international patients varied; 10 countries always or almost always, 10 sometimes, 3 rarely, and 2 (Sweden and Russia) did not accept any foreign patients. The primary reason for declining international patients was lack of funding. The most commonor the referring country
“Most countries accepted foreign patients, but referrals remained limited, with 1-2 foreign patients treated annually per country,” the researchers explained in their report.
An established referral network for patients without access to a CD19 CAR T-cell treatment center was present in 6 countries, with an additional 18 expressing interest in participating in such a network.
The questionnaire also queried about their B-ALL treatments. In general, there was greater availability to B-ALL therapies in high-income countries. The most commonly used regimens overall were ALLTogether, AIEOP-BEM ALL 2017, and ALL-IC across the surveyed countries.
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Despite the European Union’s strong focus on rare diseases, no standardized pathway for CAR-T referral currently exists and access remains variable across the region.
Monitoring of disease response by minimal residual disease (MRD) was common in high-income countries, where it was government-funded. Blinatumomab and asparaginase were also funded, though access to blinatumomab was reported as variable.
In middle-income countries, MRD testing was not government-funded, and coverage was variable. For example, coverage ranged from 0-25% for Moldova, Kazakhstan, and Albania and 50%-75% for Turkey. Availability and funding were highly variable for blinatumomab, with some countries including Armenia, Romania, Serbia, Turkey, and Belarus reporting wide availability, whereas others such as Moldova, Albania, Kazakhstan, and Ukraine reported no or limited availability. The availability of asparaginase was also variable.
“Despite the European Union’s strong focus on rare diseases, no standardized pathway for CAR-T referral currently exists and access remains variable across the region,” the researchers concluded. “A coordinated regional framework is urgently needed to promote equitable access, sustainable funding, and patient referral coordination.”
Disclosures: Some of the study authors declared affiliations with biotech, pharmaceutical, or device companies. Please see the original reference for a full list of disclosures.
Oszer A, Galimard J-E, Wardell JR, et al. Landscape assessment to characterize baseline access and multilevel barriers to IMProve Access to Chimeric Antigen Receptor T-Cell CD19 Therapy for Pediatric B-ALL (IMPACT study) across Europe. JCO Glob Oncol. 2026;12:e2600050. doi:10.1200/GO-26-00050
