{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,31]],"date-time":"2026-08-31T02:35:31Z","timestamp":1788143731205,"version":"build-2803163510"},"reference-count":27,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2024,2,24]],"date-time":"2024-02-24T00:00:00Z","timestamp":1708732800000},"content-version":"vor","delay-in-days":23,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Cancer Medicine"],"published-print":{"date-parts":[[2024,2]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>The need for new therapies to improve survival and outcomes in pediatric oncology along with the lack of approval and accessible clinical trials has led to \u201cout\u2010of\u2010trial\u201d use of innovative therapies. We conducted a retrospective analysis of requests for innovative anticancer therapy in Canadian pediatric oncology tertiary centers for patients less than 30\u2009years old between 2013 and 2020.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Innovative therapies were defined as cancer\u2010directed drugs used (a) off\u2010label, (b) unlicensed drugs being used outside the context of a clinical trial, or (c) approved drugs with limited evidence in pediatrics. We excluded cytotoxic chemotherapy, cellular products, and cytokines.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>We retrieved data on 352 innovative therapy drug requests. Underlying diagnosis was primary CNS tumor 31%; extracranial solid tumor 37%, leukemia\/lymphoma 22%, LCH 2%, and plexiform neurofibroma 6%. RAS\/MAP kinase pathway inhibitors were the most frequently requested innovative therapies in 28% of all requests followed by multi\u2010targeted tyrosine kinase inhibitors (17%), inhibitors of the PIK3CA\u2010mTOR\u2010AKT pathway (8%), immune checkpoints inhibitors (8%), and antibody drug conjugates (8%). In 112 out of 352 requests, innovative therapies were used in combination with another anticancer agent. 48% of requests were motivated by the presence of an actionable molecular target. Compassionate access accounted for 52% of all requests while public insurance was used in 27%. Mechanisms of funding varied between provinces.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>This real\u2010world data collection illustrates an increasing use of \u201cout\u2010of\u2010trial\u201d innovative therapies in pediatric oncology. This new field of practice warrants further studies to understand the impact on patient trajectory and equity in access to innovative therapies.<\/jats:p><\/jats:sec>","DOI":"10.1002\/cam4.7033","type":"journal-article","created":{"date-parts":[[2024,2,24]],"date-time":"2024-02-24T09:04:49Z","timestamp":1708765489000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["Access to innovative therapies in pediatric oncology: Report of the nationwide experience in Canada"],"prefix":"10.1002","volume":"13","author":[{"given":"Sandra","family":"Judd","sequence":"first","affiliation":[{"name":"Department of Pharmacy Hospital for Sick Children  Toronto Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6512-1004","authenticated-orcid":false,"given":"Gabriel","family":"Revon\u2010Riviere","sequence":"additional","affiliation":[{"name":"Division of Haematology\/Oncology, Hospital for Sick Children, Department of Pediatrics University of Toronto  Toronto Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stephanie A.","family":"Grover","sequence":"additional","affiliation":[{"name":"Genetics and Genome Biology, Hospital for Sick Children  Toronto Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3315-4383","authenticated-orcid":false,"given":"Rebecca J.","family":"Deyell","sequence":"additional","affiliation":[{"name":"Division of Pediatric Hematology Oncology BMT BC Children's Hospital and Research Institute  Vancouver British Columbia Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2015-0615","authenticated-orcid":false,"given":"Magimairajan Issai","family":"Vanan","sequence":"additional","affiliation":[{"name":"Pediatric Neuro\u2010Oncology, Division of Pediatric Hematology\u2010Oncology, Cancer Care Manitoba University of Manitoba  Winnipeg Manitoba Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Victor A.","family":"Lewis","sequence":"additional","affiliation":[{"name":"University of Calgary  Alberta Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lucie","family":"Pecheux","sequence":"additional","affiliation":[{"name":"Stollery Children's Hospital University of Alberta  Edmonton Alberta Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexandra P.","family":"Zorzi","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Children's Hospital London Health Sciences Centre Western University  London Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Catherine","family":"Goudie","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Hematology\u2010Oncology, Montreal Children's Hospital McGill 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