Understanding access to novel high-cost therapies across Canada: A survey of pediatric oncology providers.

e13500 Background: In Canada’s publicly funded healthcare system, access to novel cancer therapies is often inequitable. Despite compelling evidence for therapies including targeted drugs, proton therapy, and cellular therapy, many are high-cost and not publicly funded. Provinces differ in their funding review processes, resulting in variable reimbursement, despite national assessment strategies. To explore disparities in access to these therapies for children with cancer, we created an online survey to examine access to select evidence-informed but not universally funded treatments. Methods: This online vignette-based cross-sectional survey of pediatric oncology providers across 16 Canadian centres explored blinatumomab for low-risk relapse of ALL, larotrectinib for TRK-fused soft tissue sarcoma, proton therapy for unresectable head and neck sarcoma, and CT for first relapse of ALL in a patient with Down Syndrome (DS). The primary outcome was provider-reported accessibility to each therapy, compared across centres. Secondary outcomes included time to accessing therapies, funding source, and perceived barriers to access. Results: 70 respondents participated, with 68 (97%) completing at least one question. Respondents' primary roles were pediatric medical oncologist (45.6%), oncology pharmacist (14.7%), nurse practitioner (10.3%), radiation oncologist (10.3%), and hematopoietic stem cell transplant/CT physician (4.4%). Blinatumomab was accessible for low-risk relapse of ALL over 80% of the time, except in Nova Scotia (75%) and Quebec (70%). Reported access to proton therapy was highly variable, ranging from 33.3% in Nova Scotia to 100% in Manitoba. CT for first relapse in patients with DS was deemed accessible 94% of the time, and larotrectinib was deemed accessible 79% of the time. Barriers to accessing blinatumomab, proton therapy, and CT included prolonged time to obtain therapy, patient/family unwillingness to travel for therapy, and economic and psychosocial impact of travelling for therapy. Additional barriers to accessing proton therapy included challenges managing complex patients through travel, and immigration and visa restrictions. Barriers to accessing larotrectinib included being cost-prohibitive for the treating centre and patient, and other effective therapies being faster to obtain. Conclusions: Our findings demonstrate inequitable access to evidence-informed therapies in Canada. The direct costs of these therapies must be addressed to make them less prohibitive for families; however, strategies must also be developed to mitigate psychosocial and economic impacts of travelling to obtain therapies. Universal funding of these therapies, and simplified access to proton therapy centres, including the development of Canadian proton therapy facilities, would increase equitable access, ultimately improving outcomes for children with cancer.

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Publication Details

Journal
Journal of Clinical Oncology
Published
2025-05-28
DOI
https://doi.org/10.1200/jco.2025.43.16_suppl.e13500
Primary Topic
Pharmaceutical studies and practices
Type
article
Field-Weighted Citation Impact
0.00
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article

Understanding access to novel high-cost therapies across Canada: A survey of pediatric oncology providers.

Céline Cressman, Derek S. Tsang, Marcel Romanick, Paul James Gibson et al.
Journal of Clinical Oncology
Pharmaceutical studies and practices
article

Understanding access to novel high-cost therapies across Canada: A survey of pediatric oncology providers.

Céline Cressman, Derek S. Tsang, Marcel Romanick, Paul James Gibson, Avram Denburg, Kriti Kumar
article en

Abstract

e13500 Background: In Canada’s publicly funded healthcare system, access to novel cancer therapies is often inequitable. Despite compelling evidence for therapies including targeted drugs, proton therapy, and cellular therapy, many are high-cost and not publicly funded. Provinces differ in their funding review processes, resulting in variable reimbursement, despite national assessment strategies. To explore disparities in access to these therapies for children with cancer, we created an online survey to examine access to select evidence-informed but not universally funded treatments. Methods: This online vignette-based cross-sectional survey of pediatric oncology providers across 16 Canadian centres explored blinatumomab for low-risk relapse of ALL, larotrectinib for TRK-fused soft tissue sarcoma, proton therapy for unresectable head and neck sarcoma, and CT for first relapse of ALL in a patient with Down Syndrome (DS). The primary outcome was provider-reported accessibility to each therapy, compared across centres. Secondary outcomes included time to accessing therapies, funding source, and perceived barriers to access. Results: 70 respondents participated, with 68 (97%) completing at least one question. Respondents' primary roles were pediatric medical oncologist (45.6%), oncology pharmacist (14.7%), nurse practitioner (10.3%), radiation oncologist (10.3%), and hematopoietic stem cell transplant/CT physician (4.4%). Blinatumomab was accessible for low-risk relapse of ALL over 80% of the time, except in Nova Scotia (75%) and Quebec (70%). Reported access to proton therapy was highly variable, ranging from 33.3% in Nova Scotia to 100% in Manitoba. CT for first relapse in patients with DS was deemed accessible 94% of the time, and larotrectinib was deemed accessible 79% of the time. Barriers to accessing blinatumomab, proton therapy, and CT included prolonged time to obtain therapy, patient/family unwillingness to travel for therapy, and economic and psychosocial impact of travelling for therapy. Additional barriers to accessing proton therapy included challenges managing complex patients through travel, and immigration and visa restrictions. Barriers to accessing larotrectinib included being cost-prohibitive for the treating centre and patient, and other effective therapies being faster to obtain. Conclusions: Our findings demonstrate inequitable access to evidence-informed therapies in Canada. The direct costs of these therapies must be addressed to make them less prohibitive for families; however, strategies must also be developed to mitigate psychosocial and economic impacts of travelling to obtain therapies. Universal funding of these therapies, and simplified access to proton therapy centres, including the development of Canadian proton therapy facilities, would increase equitable access, ultimately improving outcomes for children with cancer.

Journal of Clinical OncologyVol. 43(16_suppl)
University Health Network (CA), Hospital for Sick Children (CA), McMaster Children's Hospital (CA), Princess Margaret Cancer Centre (CA), Stollery Children's Hospital (CA)
Openalex Percentile: Top 83%
Pharmaceutical studies and practices
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