Lasala Ruggero, Santoleri Fiorenzo, Zanon Davide, Romagnoli Alessia, Abrate Paolo, Costantini Alberto, Musicco Felice
Hospital Pharmacy of Corato, 26178ASL Bari, Bari, Italy.
Hospital Pharmacy of "Santo Spirito" General Hospital, Pescara, Italy.
J Oncol Pharm Pract. 2023 Jan;29(1):83-87. doi: 10.1177/10781552211053251. Epub 2021 Oct 23.
The treatment options available to children with cancer are limited. This is why for more than 10 years, the European Medicine Agency (EMA) has stated that all drugs to be marketed must be tested on the paediatric population in accordance with the Paediatric Investigation Plan (PIP). The objective of this study is to make a cross sectional analysis of the information related to the use of cancer drugs authorised on the European market in the paediatric population.
The European Public Assessment Reports and PIPs have been considered. The following data were extracted for onco-haematological drugs approved since 2016: paediatric indications, information about the paediatric population in the Summary of Product Characteristics (SmPC) and presence and characteristics of PIPs. A descriptive analysis of the characteristics of the drugs was made from the point of view of the paediatric population.
Forty-eight drugs with onco-haematological indications have been authorised for marketing since 2016, 7 (15%) of these have paediatric indications. Two (4%) drugs have no paediatric indication but have information related to the paediatric population within SmPC. Forty-one (85%) drugs have no reference to the paediatric population in SmPC. Seventeen (35%) drugs out of 48 do not have PIPs and 11 have been granted a waiver to present the results of paediatric studies. The other 19 active ingredients have a total of 28 PIPs.
Most of the onco-haematological drugs approved by EMA since 2016 have neither paediatric indications nor mentions about paediatric use in SmPC. PIPs represent an opportunity, but demand for the paediatric population is still huge.
癌症患儿可用的治疗选择有限。这就是为什么十多年来，欧洲药品管理局（EMA）一直规定，所有上市药物都必须根据儿科研究计划（PIP）在儿科人群中进行测试。本研究的目的是对欧洲市场上已获批的癌症药物在儿科人群中的使用相关信息进行横断面分析。
参考了欧洲公共评估报告和儿科研究计划。提取了自2016年以来获批的血液肿瘤学药物的以下数据：儿科适应症、产品特性摘要（SmPC）中有关儿科人群的信息以及儿科研究计划的存在情况和特点。从儿科人群的角度对药物特性进行了描述性分析。
自2016年以来，有48种具有血液肿瘤学适应症的药物获批上市，其中7种（15%）有儿科适应症。2种（4%）药物没有儿科适应症，但在产品特性摘要中有与儿科人群相关的信息。41种（85%）药物在产品特性摘要中未提及儿科人群。48种药物中有17种（35%）没有儿科研究计划，11种已获得豁免提交儿科研究结果。其他19种活性成分共有28项儿科研究计划。
自2016年以来EMA批准的大多数血液肿瘤学药物既没有儿科适应症，在产品特性摘要中也没有提及儿科用途。儿科研究计划是一个契机，但对儿科人群的需求仍然巨大。
