{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,29]],"date-time":"2026-10-29T03:15:04Z","timestamp":1793243704638,"version":"4.5.0"},"reference-count":41,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2025,11,1]],"date-time":"2025-11-01T00:00:00Z","timestamp":1761955200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2025,11,1]],"date-time":"2025-11-01T00:00:00Z","timestamp":1761955200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,8,22]],"date-time":"2025-08-22T00:00:00Z","timestamp":1755820800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100000780","name":"European Commission","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100000780","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100008017","name":"Institut Gustave-Roussy","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100008017","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cancertreatmentreviews.com","clinicalkey.com","clinicalkey.com.au","clinicalkey.es","clinicalkey.fr","clinicalkey.jp","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Cancer Treatment Reviews"],"published-print":{"date-parts":[[2025,11]]},"DOI":"10.1016\/j.ctrv.2025.103013","type":"journal-article","created":{"date-parts":[[2025,8,15]],"date-time":"2025-08-15T06:17:48Z","timestamp":1755238668000},"page":"103013","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":12,"special_numbering":"C","title":["Overcoming the barriers to treatment of rare cancer patients in the era of precision oncology: A call to action"],"prefix":"10.1016","volume":"140","author":[{"given":"Winette T.A.","family":"van der Graaf","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nina S.","family":"Heiss","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carolyn L.","family":"Hynes","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Simone P.","family":"Keller","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ariane","family":"Weinman","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jean-Yves","family":"Blay","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pedro","family":"Franco","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rachel H.","family":"Giles","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Denis","family":"Lacombe","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Philipp","family":"Schlatter","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David M.","family":"Thomas","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sahar Barjesteh","family":"van Waalwijk van Doorn-Khosrovani","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Max","family":"Williamson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ruth","family":"Plummer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ctrv.2025.103013_b0005","unstructured":"International Agency for Research on Cancer. IARC highlights rare cancers to mark Rare Disease Day 2023, 29 February 2024. Available at: https:\/\/www.iarc.who.int\/video\/rare-disease-day-2024\/ (last accessed 13 May 2025)."},{"issue":"12","key":"10.1016\/j.ctrv.2025.103013_b0010","first-page":"807","article-title":"Diagnostic trajectories of rare cancer patients in the Netherlands: results from a nationwide cross-sectional survey","volume":"32","author":"Padilla","year":"2024","journal-title":"Support Care Cancer"},{"key":"10.1016\/j.ctrv.2025.103013_b0015","article-title":"Psychological outcomes in patients with rare cancers: a systematic review and meta-analysis","volume":"72","author":"Low","year":"2024","journal-title":"EClin Med"},{"issue":"14","key":"10.1016\/j.ctrv.2025.103013_b0020","doi-asserted-by":"crossref","first-page":"2529","DOI":"10.3390\/cancers16142529","article-title":"Target-driven tissue-agnostic drug approvals \u2013 a new path of drug development","volume":"16","author":"Thein","year":"2024","journal-title":"Cancers (Basel)"},{"issue":"9","key":"10.1016\/j.ctrv.2025.103013_b0025","doi-asserted-by":"crossref","first-page":"1255","DOI":"10.1038\/bjc.2017.321","article-title":"Rare cancers the greatest inequality in cancer research and oncology treatment","volume":"117","author":"Alvi","year":"2017","journal-title":"Br J Cancer"},{"issue":"32","key":"10.1016\/j.ctrv.2025.103013_b0030","doi-asserted-by":"crossref","first-page":"3817","DOI":"10.1200\/JCO.2015.61.5997","article-title":"Impact of precision medicine in diverse cancers: a meta-analysis of phase II clinical trials","volume":"33","author":"Schwaederle","year":"2015","journal-title":"J Clin Oncol"},{"issue":"11","key":"10.1016\/j.ctrv.2025.103013_b0035","article-title":"Impact of a biomarker-based strategy on oncology drug development: a meta-analysis of clinical trials leading to FDA approval","volume":"107","author":"Jardim","year":"2015","journal-title":"J Natl Cancer Inst"},{"issue":"11","key":"10.1016\/j.ctrv.2025.103013_b0040","doi-asserted-by":"crossref","first-page":"1452","DOI":"10.1001\/jamaoncol.2016.2129","article-title":"Association of biomarker-based treatment strategies with response rates and progression-free survival in refractory malignant neoplasms: a meta-analysis","volume":"2","author":"Schwaederle","year":"2016","journal-title":"JAMA Oncol"},{"issue":"Suppl 16","key":"10.1016\/j.ctrv.2025.103013_b0045","article-title":"Increasing targeted therapy options for patients with relapsed cancer with broader somatic gene panel analysis from the primary tumor: the Profiler02 randomized phase II trial","volume":"40","author":"Tredan","year":"2022","journal-title":"J Clin Oncol"},{"issue":"4","key":"10.1016\/j.ctrv.2025.103013_b0050","doi-asserted-by":"crossref","first-page":"531","DOI":"10.1016\/S1470-2045(19)30856-3","article-title":"Larotrectinib in patients with TRK fusion-positive solid tumours: a pooled analysis of three phase 1\/2 clinical trials","volume":"21","author":"Hong","year":"2020","journal-title":"Lancet Oncol"},{"key":"10.1016\/j.ctrv.2025.103013_b0055","doi-asserted-by":"crossref","first-page":"731","DOI":"10.1056\/NEJMoa1714448","article-title":"Efficacy of larotrectinib in TRK fusion\u2013positive cancers in adults and children","volume":"378","author":"Drilon","year":"2018","journal-title":"N Engl J Med"},{"issue":"8","key":"10.1016\/j.ctrv.2025.103013_b0060","doi-asserted-by":"crossref","first-page":"726","DOI":"10.1056\/NEJMoa1502309","article-title":"Vemurafenib in multiple nonmelanoma cancers with BRAF V600 mutations","volume":"373","author":"Hyman","year":"2015","journal-title":"N Engl J Med"},{"issue":"Suppl 16","key":"10.1016\/j.ctrv.2025.103013_b0065","doi-asserted-by":"crossref","first-page":"1540","DOI":"10.1200\/JCO.2023.41.16_suppl.1540","article-title":"Genomic therapy matching in rare and refractory cancers: Updated results from a retrospective cohort study in the Molecular Screening and Therapeutic (MoST) program","volume":"41","author":"Lin","year":"2023","journal-title":"J Clin Oncol"},{"issue":"1","key":"10.1016\/j.ctrv.2025.103013_b0070","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1016\/S1470-2045(21)00581-7","article-title":"The global burden of adolescent and young adult cancer in 2019: a systematic analysis for the Global Burden of Disease Study 2019","volume":"23","author":"Alvarez","year":"2022","journal-title":"Lancet Oncol"},{"issue":"4","key":"10.1016\/j.ctrv.2025.103013_b0075","article-title":"Access to clinical trials for adolescents and young adults with cancer: a meta-research analysis","volume":"3","author":"De Rojas","year":"2019","journal-title":"JNCI Cancer Spectr"},{"key":"10.1016\/j.ctrv.2025.103013_b0080","doi-asserted-by":"crossref","first-page":"49","DOI":"10.1016\/j.ejca.2019.02.011","article-title":"Timing of first-in-child trials of FDA-approved oncology drugs","volume":"112","author":"Neel","year":"2019","journal-title":"Eur J Cancer"},{"issue":"13","key":"10.1016\/j.ctrv.2025.103013_b0085","doi-asserted-by":"crossref","first-page":"4495","DOI":"10.1002\/cam4.3083","article-title":"Sponsorship of oncology clinical trials in the United States according to age of eligibility","volume":"9","author":"Neel","year":"2020","journal-title":"Cancer Med"},{"issue":"2","key":"10.1016\/j.ctrv.2025.103013_b0090","doi-asserted-by":"crossref","DOI":"10.1016\/j.esmoop.2021.100096","article-title":"Adolescents and young adults (AYA) with cancer: a position paper from the AYA Working Group of the European Society for Medical Oncology (ESMO) and the European Society for Paediatric Oncology (SIOPE)","volume":"6","author":"Ferrari","year":"2021","journal-title":"ESMO Open"},{"key":"10.1016\/j.ctrv.2025.103013_b0095","doi-asserted-by":"crossref","first-page":"145","DOI":"10.1016\/j.ejca.2022.01.033","article-title":"ACCELERATE \u2013 five years accelerating cancer drug development for children and adolescents","volume":"166","author":"Pearson","year":"2022","journal-title":"Eur J Cancer"},{"issue":"Suppl 16","key":"10.1016\/j.ctrv.2025.103013_b0100","article-title":"Growing wiser with age: results from the ACCELERATE Fostering Age Inclusive Research Group clinical trial survey","volume":"40","author":"Williamson","year":"2023","journal-title":"J Clin Oncol"},{"issue":"3","key":"10.1016\/j.ctrv.2025.103013_b0105","doi-asserted-by":"crossref","first-page":"766","DOI":"10.1093\/annonc\/mdy002","article-title":"Joint adolescent\u2013adult early phase clinical trials to improve access to new drugs for adolescents with cancer: proposals from the multi-stakeholder platform\u2014ACCELERATE","volume":"29","author":"Gaspar","year":"2018","journal-title":"Ann Oncol"},{"issue":"7","key":"10.1016\/j.ctrv.2025.103013_b0110","doi-asserted-by":"crossref","DOI":"10.1001\/jamanetworkopen.2023.21568","article-title":"Measuring safety and outcomes for the use of compassionate and off-label therapies for children, adolescents, and young adults with cancer in the SACHA-France study","volume":"6","author":"Berlanga","year":"2023","journal-title":"JAMA Netw Open"},{"key":"10.1016\/j.ctrv.2025.103013_b0115","doi-asserted-by":"crossref","first-page":"127","DOI":"10.1038\/s41586-019-1600-x","article-title":"The Drug Rediscovery protocol facilitates the expanded use of existing anticancer drugs","volume":"574","author":"Van der Velden","year":"2019","journal-title":"Nature"},{"issue":"5","key":"10.1016\/j.ctrv.2025.103013_b0120","doi-asserted-by":"crossref","first-page":"663","DOI":"10.1093\/annonc\/mdz119","article-title":"Personalised reimbursement: a risk-sharing model for biomarker-driven treatment of rare subgroups of cancer patients","volume":"30","author":"Van Waalwijk van Doorn-Khosrovani","year":"2019","journal-title":"Ann. Oncol."},{"issue":"7","key":"10.1016\/j.ctrv.2025.103013_b0125","doi-asserted-by":"crossref","first-page":"1402","DOI":"10.1158\/1078-0432.CCR-21-3752","article-title":"Patients with rare cancers in the Drug Rediscovery Protocol (DRUP) benefit from genomics-guided treatment","volume":"28","author":"Hoes","year":"2022","journal-title":"Clin Cancer Res"},{"key":"10.1016\/j.ctrv.2025.103013_b0130","doi-asserted-by":"crossref","DOI":"10.1016\/j.ejca.2024.114225","article-title":"Empowering effective biomarker-driven precision oncology: a call to action","volume":"209","author":"Lawler","year":"2024","journal-title":"Eur J Cancer"},{"key":"10.1016\/j.ctrv.2025.103013_b0135","doi-asserted-by":"crossref","first-page":"368","DOI":"10.2340\/1651-226X.2024.34885","article-title":"The evolution of precision oncology: the ongoing impact of the Drug Rediscovery Protocol (DRUP)","volume":"63","author":"Mohammad","year":"2024","journal-title":"Acta Oncol"},{"issue":"7","key":"10.1016\/j.ctrv.2025.103013_b0140","doi-asserted-by":"crossref","first-page":"1413","DOI":"10.1002\/ijc.34649","article-title":"Limited clinical activity of palbociclib and ribociclib monotherapy in advanced cancers with cyclin D-CDK4\/6 pathway alterations in the Dutch DRUP and Australian MoST trials","volume":"153","author":"Zeverijn","year":"2023","journal-title":"Int J Cancer"},{"issue":"Suppl 2","key":"10.1016\/j.ctrv.2025.103013_b0145","first-page":"S925","article-title":"The DRUP-like clinical trials family: a distributed European trial network for equitable access to precision medicine","volume":"34","author":"Gelderblom","year":"2023","journal-title":"Ann Oncol"},{"issue":"Suppl 2","key":"10.1016\/j.ctrv.2025.103013_b0150","doi-asserted-by":"crossref","first-page":"S294","DOI":"10.1016\/j.annonc.2024.08.2202","article-title":"PRIME-ROSE: Merging clinical outcome data from DRUP-like clinical trials","volume":"35","author":"Kringelbach","year":"2024","journal-title":"Ann Oncol"},{"key":"10.1016\/j.ctrv.2025.103013_b0155","doi-asserted-by":"crossref","first-page":"385","DOI":"10.2340\/1651-226X.2024.34791","article-title":"PCM4EU and PRIME-ROSE: collaboration for implementation of precision cancer medicine in Europe","volume":"63","author":"Task\u00e9n","year":"2024","journal-title":"Acta Oncol"},{"issue":"11","key":"10.1016\/j.ctrv.2025.103013_b0160","doi-asserted-by":"crossref","first-page":"2852","DOI":"10.1093\/annonc\/mdx484","article-title":"Improved survival using specialized multidisciplinary board in sarcoma patients","volume":"28","author":"Blay","year":"2017","journal-title":"Ann Oncol"},{"issue":"7","key":"10.1016\/j.ctrv.2025.103013_b0165","doi-asserted-by":"crossref","first-page":"1143","DOI":"10.1093\/annonc\/mdz124","article-title":"Surgery in reference centers improves survival of sarcoma patients: a nationwide study","volume":"30","author":"Blay","year":"2019","journal-title":"Ann Oncol"},{"issue":"4","key":"10.1016\/j.ctrv.2025.103013_b0170","doi-asserted-by":"crossref","first-page":"351","DOI":"10.1016\/j.annonc.2024.01.001","article-title":"Improved nationwide survival of sarcoma patients with a network of reference centers","volume":"35","author":"Blay","year":"2024","journal-title":"Ann Oncol"},{"issue":"10","key":"10.1016\/j.ctrv.2025.103013_b0175","doi-asserted-by":"crossref","DOI":"10.2196\/22810","article-title":"The role of social media in enhancing clinical trial recruitment: scoping review","volume":"22","author":"Darmawan","year":"2020","journal-title":"J Med Internet Res"},{"issue":"2","key":"10.1016\/j.ctrv.2025.103013_b0180","doi-asserted-by":"crossref","first-page":"181","DOI":"10.1038\/s41591-019-0749-z","article-title":"The Angiosarcoma Project: enabling genomic and clinical discoveries in a rare cancer through patient-partnered research","volume":"26","author":"Painter","year":"2020","journal-title":"Nat Med"},{"issue":"10435","key":"10.1016\/j.ctrv.2025.103013_b0185","doi-asserted-by":"crossref","first-page":"1460","DOI":"10.1016\/S0140-6736(24)00319-2","article-title":"Afamitresgene autoleucel for advanced synovial sarcoma and myxoid round cell liposarcoma (SPEARHEAD-1): an international, open-label, phase 2 trial","volume":"403","author":"D\u2019Angelo","year":"2024","journal-title":"Lancet"},{"issue":"32","key":"10.1016\/j.ctrv.2025.103013_b0190","doi-asserted-by":"crossref","first-page":"3783","DOI":"10.1200\/JCO-24-01200","article-title":"Equipoise lost? Trial conduct challenges in an era of breakthrough therapies","volume":"42","author":"Fallah","year":"2024","journal-title":"J Clin Oncol"},{"issue":"12","key":"10.1016\/j.ctrv.2025.103013_b0195","doi-asserted-by":"crossref","first-page":"2740","DOI":"10.1002\/pon.4873","article-title":"Assessment of distress and quality of life in rare cancers","volume":"27","author":"Bergerot","year":"2018","journal-title":"Psychooncology"},{"issue":"3","key":"10.1016\/j.ctrv.2025.103013_b0200","doi-asserted-by":"crossref","first-page":"387","DOI":"10.3390\/cancers17030387","article-title":"Health-related quality of life (HRQoL) assessments in research on patients with adult rare solid cancers: a state-of-the-art review","volume":"17","author":"Padilla","year":"2025","journal-title":"Cancers (Basel)"},{"issue":"17","key":"10.1016\/j.ctrv.2025.103013_b0205","article-title":"Measuring health-related quality of life in solid rare cancer patients: a study protocol","volume":"2","author":"Padilla","year":"2024","journal-title":"Rare"}],"container-title":["Cancer Treatment Reviews"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0305737225001355?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0305737225001355?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,11,1]],"date-time":"2025-11-01T16:51:55Z","timestamp":1762015915000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0305737225001355"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,11]]},"references-count":41,"alternative-id":["S0305737225001355"],"URL":"https:\/\/doi.org\/10.1016\/j.ctrv.2025.103013","relation":{},"ISSN":["0305-7372"],"issn-type":[{"value":"0305-7372","type":"print"}],"subject":[],"published":{"date-parts":[[2025,11]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Overcoming the barriers to treatment of rare cancer patients in the era of precision oncology: A call to action","name":"articletitle","label":"Article Title"},{"value":"Cancer Treatment Reviews","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ctrv.2025.103013","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2025 The Authors. Published by Elsevier Ltd.","name":"copyright","label":"Copyright"}],"article-number":"103013"}}