Division of Oncology-Hematology, Department of Pediatrics, Kantonsspital Aarau, Aarau, Switzerland; Department of Oncology, Haematology, Immunology, Stem Cell Transplantation and Somatic Gene Therapy, University Children's Hospital Zurich, Zurich, Switzerland.
Division of Pediatric Hematology and Oncology, University Children's Hospital Bern, Bern, Switzerland.
Lancet Oncol. 2022 Dec;23(12):1537-1546. doi: 10.1016/S1470-2045(22)00623-4. Epub 2022 Nov 1.
Shortages and unequal access to anticancer medicines for children and adolescents are a reality in Europe. The aim of the European Society for Paediatric Oncology (SIOPE) Essential Anticancer Medicines Project was to provide a list of anticancer medicines that are considered essential in the treatment of paediatric cancers to help ensure their continuous access to all children and adolescents with cancer across Europe.
This pan-European project, done between Jan 20, 2020, and Feb 18, 2022, was designed to be a systematic collection and review of treatment protocols and strategies that are used to treat childhood cancer in Europe. We formed 16 working groups on the basis of paediatric cancer types, and which were based on the existing SIOPE Clinical Trial Groups. Workings groups consisted of representatives from the SIOPE Clinical Trial Groups, Young SIOPE members, and senior paediatric oncology experts. Each group collected existing treatment protocols that are used to treat the respective cancer types in Europe. Medicines from the standard group of each protocol were extracted. For medicines not on the WHO Essential Medicines List for children (EMLc) 2017, working groups did a literature search to determine whether the medicines should be defined as essential, promising, or neither essential nor promising. Each group provided an individual summary, and all medicines that were considered essential by at least one group were combined in a joint list.
The working groups identified 73 treatment protocols used in Europe and defined 66 medicines as essential. For several newer medicines, such as kinase inhibitors or tisagenlecleucel, the supporting evidence was insufficient to consider them essential, so these medicines were defined as promising. 25 medicines were considered promising by at least one working group. 22 (33%) of the 66 essential and none of the promising medicines were included in the WHO EMLc 2017. The WHO EMLc 2021 included two new medicines (everolimus and vinorelbine) following applications we made as a result of this project.
Medicines that were defined as essential within this project should be available for the treatment of childhood and adolescent cancer continuously and across Europe. This list can be used to support and guide stakeholders and policy makers in negotiations on a national and European level regarding shortages, accessibility, and affordability of these medicines.
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儿童和青少年抗癌药物的短缺和获取不平等在欧洲是一个现实问题。欧洲小儿肿瘤学会（SIOPE）基本抗癌药物项目的目的是提供一份被认为是治疗儿科癌症基本药物的清单，以帮助确保所有欧洲儿童和青少年癌症患者都能持续获得这些药物。
这项泛欧项目于 2020 年 1 月 20 日至 2022 年 2 月 18 日进行，旨在对欧洲治疗儿童癌症的治疗方案和策略进行系统收集和审查。我们根据小儿癌症类型成立了 16 个工作组，这些工作组基于现有的 SIOPE 临床试验组。工作组由 SIOPE 临床试验组的代表、年轻的 SIOPE 成员和资深儿科肿瘤专家组成。每个小组都收集了在欧洲用于治疗各自癌症类型的现有治疗方案。从每个方案的标准组中提取药物。对于不在世界卫生组织 2017 年儿童基本药物清单（EMLc）上的药物，工作组进行了文献检索，以确定这些药物是否应被定义为基本、有前途还是既非基本也非有前途。每个小组都提供了一份单独的总结，至少有一个小组认为基本的所有药物都被组合在一个联合清单中。
工作组确定了欧洲使用的 73 种治疗方案，并将 66 种药物定义为基本药物。对于一些较新的药物，如激酶抑制剂或 tisagenlecleucel，支持其被认为是基本药物的证据不足，因此这些药物被定义为有前途。至少有一个工作组认为 25 种药物有前途。在 66 种基本药物中，没有一种有前途的药物被列入世界卫生组织 2017 年 EMLc。世界卫生组织 2021 年 EMLc 列入了两种新的药物（依维莫司和长春瑞滨），这是我们根据该项目的申请而列入的。
在本项目中被定义为基本药物的药物应在整个欧洲持续用于治疗儿童和青少年癌症。该清单可用于支持和指导利益攸关方和决策者在国家和欧洲一级就这些药物的短缺、可及性和可负担性进行谈判。
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