Cleveland Celeste, Ralaidovy Ambinintsoa H, Jallo Chrys, Frank Felicia, Adam Taghreed, Ilbawi Andre, Boklan Jessica
Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ, USA; Department of Child Health, University of Arizona College of Medicine, Phoenix, AZ, USA.
Strategic Planning and Performance Monitoring Unit, WHO, Geneva, Switzerland.
Lancet Child Adolesc Health. 2025 Aug;9(8):544-552. doi: 10.1016/S2352-4642(25)00142-7. Epub 2025 Jun 25.
Although childhood cancer survival outcomes have markedly improved over the past several decades due to therapeutic advancements, significant gaps remain in accessibility to cancer medicines for children. We aimed to analyse the global paediatric oncology landscape and pipeline of all childhood cancer medicines that are in common use or development.
We searched the International Clinical Trials Registry Platform (ICTRP) for paediatric oncology trials registered between Jan 1, 2007 and Aug 2, 2022, to identify childhood cancer medicines. From each trial's full entry and further online search across various sources, we obtained information on mechanism of action, molecular target, most recent development phase, malignancy inclusion in trials, administration route, paediatric-friendly oral formulation availability, storage requirements, and regulatory approval status for each cancer medicine. Cellular therapies were analysed separately by target, most recent development phase, malignancy inclusion in trials, and regulatory approval status. We summarise attributes of childhood cancer medicines and paediatric oncology trial conduct.
Of 5068 clinical trials downloaded from the ICTRP, 2160 met inclusion criteria for full review and data extraction. The highest clinical trial participation was in high-income countries, the majority being in the Americas (1006 trials), the Western Pacific (843 trials), and Europe (588 trials). We identified 440 unique childhood cancer medicines, excluding cellular therapy; of these, 243 (55%) medicines were either molecular targeted therapies or immunotherapies. Of 212 medicines with available information, 79 (37%) required cold storage, and 112 (55%) of 204 required light protection. Paediatric-friendly formulations were available for 57 (45%) of 126 orally administered medicines. Of the 440 cancer medicines used in paediatric cancer trials, 37 (8%) and 85 (19%) are approved for children by the European Medicines Agency (EMA) and US Food and Drug Administration (FDA), respectively, with a median approval lag time between adult and paediatric approvals of 2 years (IQR 0-7) for the EMA and 3 years (0-10) for the FDA. 274 (62%) of 440 cancer medicines were in phase 1 or 2 of development. Most cellular therapies in clinical trials were chimeric antigen receptor (CAR) T-cell therapies, targeting 48 unique antigens.
This overview of the global paediatric oncology landscape and pipeline of childhood cancer medicines highlights important barriers to improving effective treatment access for all children. Further analyses of these data, which are now publicly available on an online dashboard hosted by the WHO Global Observatory on Health Research and Development, should guide stakeholders in further investigation of clinical trial results to inform drug prioritisation for clinical development, paediatric-friendly formulations, expedited regulatory approvals, essential medicine designation, and local capacity building.
St Jude Children's Research Hospital.
尽管由于治疗进展，儿童癌症的生存结果在过去几十年中有了显著改善，但儿童获得癌症药物的机会仍存在巨大差距。我们旨在分析全球儿科肿瘤学格局以及所有常用或正在研发的儿童癌症药物的研发进程。
我们在国际临床试验注册平台（ICTRP）上搜索了2007年1月1日至2022年8月2日期间注册的儿科肿瘤学试验，以识别儿童癌症药物。从每个试验的完整条目以及通过对各种来源的进一步在线搜索，我们获得了每种癌症药物的作用机制、分子靶点、最新研发阶段、试验中纳入的恶性肿瘤、给药途径、儿科友好型口服制剂的可用性、储存要求以及监管批准状态。细胞疗法按靶点、最新研发阶段、试验中纳入的恶性肿瘤以及监管批准状态进行单独分析。我们总结了儿童癌症药物的属性以及儿科肿瘤学试验的开展情况。
从ICTRP下载的5068项临床试验中，2160项符合全面审查和数据提取的纳入标准。临床试验参与度最高的是高收入国家，其中大部分在美洲（1006项试验）、西太平洋地区（843项试验）和欧洲（588项试验）。我们识别出440种独特的儿童癌症药物，不包括细胞疗法；其中，243种（55%）药物为分子靶向疗法或免疫疗法。在有可用信息的212种药物中，79种（37%）需要冷藏，204种中的112种（55%）需要避光保存。126种口服药物中有57种（45%）有儿科友好型制剂。在儿科癌症试验中使用的440种癌症药物中，分别有37种（8%）和85种（19%）获得了欧洲药品管理局（EMA）和美国食品药品监督管理局（FDA）的儿童用药批准，EMA批准的成人与儿童批准之间的中位滞后时间为2年（四分位间距0 - 7），FDA为3年（0 - 10）。440种癌症药物中有274种（62%）处于1期或2期研发阶段。临床试验中的大多数细胞疗法是嵌合抗原受体（CAR）T细胞疗法，靶向48种独特抗原。
对全球儿科肿瘤学格局以及儿童癌症药物研发进程的概述凸显了改善所有儿童有效治疗可及性的重要障碍。对这些现已在世卫组织全球卫生研究与发展观察站托管的在线仪表板上公开提供的数据进行进一步分析，应指导利益相关者进一步研究临床试验结果，以为临床开发的药物优先级排序、儿科友好型制剂、加快监管批准、基本药物指定以及当地能力建设提供信息。
圣裘德儿童研究医院。
