CELL INTELLIGENCE · 肿瘤细胞治疗研究

INTRODUCTION: Many evidence-informed high-cost therapies for childhood cancer, including targeted drugs, proton beam therapy (PBT), and cellular therapy (CT) are not publicly funded in Canada. This study aimed to identify disparities and barriers to access, and key health policy changes to improve access to high-cost therapies for children. METHODS: We conducted an online cross-sectional vignette-based survey among pediatric oncology providers at 16 Canadian pediatric oncology centres. Vignettes explored access to evidence-informed but not universally funded therapies: blinatumomab for low-risk relapse of B-cell acute lymphoblastic leukemia (B-ALL), larotrectinib for TRK-fused soft tissue sarcoma, PBT for unresectable head-and-neck sarcoma, and tisagenlecleucel for first relapse of B-ALL in a patient with Down syndrome. The primary outcome was access to each therapy, defined as the patient's ability to receive the specified therapy; secondary outcomes included time to access, funding sources, and perceived barriers. RESULTS: Seventy participants were enrolled from 15 centres, with 68 (response rate = 97%) completing at least one survey question. Most respondents were pediatric medical oncologists (31/68, 45.6%). Provider-reported access rates were 89% for blinatumomab ( n = 35), 79% for larotrectinib ( n = 27), 59.2% for PBT ( n = 30), and 94% for tisagenlecleucel ( n = 30). Key barriers to accessing blinatumomab, PBT, and tisagenlecleucel included patient/family inability to travel, and the economic and psychosocial impact of travel. High cost of larotrectinib was a notable barrier. CONCLUSIONS: Access to evidence-informed cancer therapies for Canadian children remains variable. Universal funding, simplified approval processes, and the establishment of Canadian PBT centres to reduce travel burden, would ensure timely, equitable access to high-cost therapies.