AbstractEmbedding precision medicine in paediatric oncology shows promise to have a positive impact on how children with cancer will be treated in the future. While there are a number of studies of precision medicine in childhood cancer, there is limited evidence available on the cost of implementing the related testing.

摘要将精准医学嵌入儿科肿瘤学有望对未来癌症儿童的治疗产生积极影响。虽然有许多关于儿童癌症精准医学的研究，但关于实施相关测试的成本的证据有限。

This is the first Australian study that systematically measures the cost of using precision medicine in the care of high-risk childhood cancers, through the Zero Childhood Cancer Precision Medicine Programme. In 2021 Australian dollars, the estimated costs inclusive of genomic and preclinical testing were: (A) $12,743 per patient for access; (B) $14,262 per identification of molecular cause; and (C) $21,769 per MTB recommendation.

这是澳大利亚第一项通过零儿童癌症精准医学计划系统地衡量使用精准医学治疗高危儿童癌症的成本的研究。在2021澳元中，包括基因组和临床前测试在内的估计费用为：（A）每位患者12743澳元；（B） 每次鉴定分子原因14262美元；（C）每个MTB建议21769美元。

The information gained supports the understanding of the cost of reporting clinically significant outcomes relevant to the biology of the tumour, diagnosis, prognosis and potentially improving clinical management for a child..

获得的信息有助于理解报告与肿瘤生物学，诊断，预后和潜在改善儿童临床管理相关的临床重要结果的成本。。

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Delivering precision oncology to patients with cancer

为癌症患者提供精准肿瘤学

Article

文章

19 April 2022

2022年4月19日

Feasibility of functional precision medicine for guiding treatment of relapsed or refractory pediatric cancers

功能精准医学指导小儿复发或难治性癌症治疗的可行性

Article

文章

Open access

开放存取

11 April 2024

2024年4月11日

Precision medicine: affording the successes of science

精准医学：提供科学的成功

Article

文章

Open access

开放存取

04 January 2023

2023年1月4日

IntroductionCancer is the leading cause of disease-related death in children in high-income countries, including Australia1. While some higher-incidence cancers have seen dramatic improvements in survival rates, children and young adults (≤21 years of age) with a histologic diagnosis of a high-risk malignancy, defined as expected overall survival of <30%, accounts for ~20–25% of those diagnosed with cancer each year in Australia2.

引言癌症是包括澳大利亚在内的高收入国家儿童疾病相关死亡的主要原因1。虽然一些高发病率的癌症的生存率有了显着提高，但组织学诊断为高风险恶性肿瘤（定义为预期总生存率低于30%）的儿童和年轻人（≤21岁）占澳大利亚每年被诊断患有癌症的人的约20-25%2。

For surviving children, conventional chemotherapies and radiotherapy have non-specific toxicity to both normal and cancer cells, resulting in acute and chronic side effects. Around 75% of childhood cancer survivors will suffer from chronic health conditions, of which 30% will be severe, life-threatening, or disabling, including second cancers, cardiac disease, learning difficulties and growth abnormalities3.

对于存活的儿童，常规化疗和放疗对正常细胞和癌细胞都具有非特异性毒性，导致急性和慢性副作用。大约75%的儿童癌症幸存者将患有慢性健康状况，其中30%将严重，危及生命或致残，包括第二种癌症，心脏病，学习困难和生长异常3。

Acute adverse effects and long-term sequelae contribute significant burden on the health care system. Novel, less toxic therapeutics and models of care, such as precision medicine are thus urgently needed to improve outcomes and reduce the incidence of later adverse effects in childhood cancer survivors.Paediatric precision oncology recognises that every child and every cancer is unique4.

急性不良反应和长期后遗症给医疗保健系统带来了沉重负担。因此，迫切需要新型毒性较小的治疗方法和护理模式，如精准医学，以改善预后并减少儿童癌症幸存者后期不良反应的发生率。儿科精准肿瘤学认识到每个孩子和每个癌症都是独一无二的4。

Precise characterisation of a child’s individual tumour improves the potential for the identification of tailored treatment regiments5,6,7. Further, knowledge that facilitates avoidance of ineffective or unnecessarily harmful treatments can have added health benefits and cost savings4,8. In recent years, a number of paediatric precision oncology research programmes have been established globally that use innovative emerging technologies to generate rich data sets that increasingly allow us to identify new targets that drive a child’s individual cancer, ma.

儿童个体肿瘤的精确表征提高了确定定制治疗方案的潜力5,6,7。此外，有助于避免无效或不必要有害治疗的知识可以增加健康益处和节省成本4,8。近年来，全球已经建立了许多儿科精确肿瘤学研究计划，这些计划使用创新的新兴技术生成丰富的数据集，使我们能够越来越多地确定驱动儿童个体癌症的新目标，ma。

Data availability

数据可用性

Study data are available from the authors upon request for a methodologically sound project approved by an independent review committee.

研究数据可根据独立审查委员会批准的方法合理的项目的要求从作者那里获得。

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The future of precision medicine in Australia. (Australian Council of Learned Academies, 2018).Download referencesAcknowledgementsZero Childhood Cancer Precision Medicine Programme (ZERO) is a joint initiative led by the Children’s Cancer Institute and Kids Cancer Centre, Sydney Children’s Hospital, Randwick.

澳大利亚精准医学的未来。（澳大利亚学术学院理事会，2018年）。Download ReferencesAcknowlementsZero儿童癌症精准医学计划（ZERO）是由兰德威克悉尼儿童医院儿童癌症研究所和儿童癌症中心领导的联合倡议。

The following are acknowledged for providing information and advice on the Zero Childhood Cancer Precision Medicine Programme. Andrijana Padovan, Angela Lin, Angela Xie, Biljana Dumevska, Chelsea Mayoh, Emilio Caspanello, Emmy Dolman, Gabor Tax, Kimberly Dias, Loretta M. S. Lau, Louise Cui, Marie Wong, Mark J.

。安德莉亚娜·帕多瓦（AndrijanaPadovan）、安吉拉·林（AngelaLin）、安吉拉·谢（AngelaXie）、比尔雅娜·杜梅夫斯卡（BiljanaDumevska）、切尔西·梅奥（ChelseaMayoh）、埃米利奥·卡斯帕内罗（EmilioCaspanello）、艾美·多尔曼（EmmyDolman）、加博·塔克斯（Gabor Tax）、金伯利·迪亚斯（Ki。

Cowley, Moiz Bandukwala, Pamela Ajuyah, Patrick A. Strong, Paulette Barahona, Peter Trebilcock and Vishal Bishnoi from Children’s Cancer Institute. Laura Woodrow from the Kids Cancer Centre, Sydney Children’s Hospital. Dong-Anh Khuong-Quang from the Children’s Cancer Centre, Royal Children’s Hospital.

儿童癌症研究所的考利、莫伊斯·班杜瓦拉、帕梅拉·阿朱亚、帕特里克·斯特朗、保莱特·巴拉霍纳、彼得·特雷比尔科克和维沙尔·比什诺伊。来自悉尼儿童医院儿童癌症中心的劳拉·伍德罗。皇家儿童医院儿童癌症中心的Dong Anh Khuong Quang。

Sarah West from the Centre for Economic Impacts of Genomic Medicine, Macquarie University, is acknowledged for providing research support. We thank the Kinghorn Centre for Clinical Genomics (Garvan Institute of Medical Research), Murdoch Children’s Research Institute/Victorian Comprehensive Genetics Service for sequencing services.

麦格理大学基因组医学经济影响中心的SarahWest因提供研究支持而受到认可。我们感谢金霍恩临床基因组学中心（Garvan医学研究所），默多克儿童研究所/维多利亚州综合遗传学服务中心提供的测序服务。

The authors acknowledge the use of the clinical services and facilities of the Australian Genome Research Facility (AGRF). This work was funded by Luminesce Alliance—Innovation for Children’s Health. Luminesce Alliance—Innovation for.

作者承认使用了澳大利亚基因组研究机构（AGRF）的临床服务和设施。这项工作由Luminesce儿童健康创新联盟资助。Luminesce联盟创新。

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PubMed Google ScholarContributionsC.O., O.T. and R.S. performed statistical analysis and prepared the draft of the manuscript. C.O. and O.T. contributed equally to the manuscript. J.K. collated and prepared the data. T.O., V.T. and D.S. conceived the project. All authors interpreted the results, read and approved the final manuscript.Corresponding authorsCorrespondence to.

PubMed谷歌学术贡献中心。O、 ，O.T.和R.S.进行了统计分析并准备了手稿草稿。C、 O.和O.T.对手稿的贡献相同。J、 K.整理并准备了数据。T、 O.，V.T.和D.S.构思了这个项目。所有作者都解释了结果，阅读并批准了最终稿件。通讯作者通讯。

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Reprints and permissionsAbout this articleCite this articleOwens, C.E.L., Tan, O., Kuroiwa-Trzmielina, J. et al. The economic costs of precision medicine for clinical translational research among children with high-risk cancer.

转载和许可本文引用本文Owens，C.E.L.，Tan，O.，Kuroiwa-Trzmielina，J。等人。精准医学用于高危癌症儿童临床转化研究的经济成本。

npj Precis. Onc. 8, 224 (2024). https://doi.org/10.1038/s41698-024-00711-wDownload citationReceived: 22 January 2024Accepted: 17 September 2024Published: 05 October 2024DOI: https://doi.org/10.1038/s41698-024-00711-wShare this articleAnyone you share the following link with will be able to read this content:Get shareable linkSorry, a shareable link is not currently available for this article.Copy to clipboard.

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Cancer genomicsGenetic testingHealth care economicsPaediatric cancer

癌症基因组遗传学检测卫生保健经济学儿科癌症