Vassal Gilles, Kearns Pam, Blanc Patricia, Scobie Nicole, Heenen Delphine, Pearson Andy
Department of Clinical Research, Gustave Roussy, Paris-Sud University, Paris, France; Innovative Therapy for Children with Cancer, Villejuif, France.
Innovative Therapy for Children with Cancer, Villejuif, France; Institute of Cancer and Genomic Sciences, University of Birmingham, Birmingham, UK.
Eur J Cancer. 2017 Oct;84:149-158. doi: 10.1016/j.ejca.2017.07.021. Epub 2017 Sep 4.
Oncology represents a major sector in the field of orphan drug development in Europe. The objective was to evaluate whether children and adolescents with cancer benefited from the Orphan Drug Regulation.
Data on orphan drug designations (ODDs) and registered orphan drugs from 8th August 2000 to 10th September 2016 were collected from the Community Register of medicinal products for human use. Assessment history, product information and existence of paediatric investigation plans were searched and retrieved from the European Medicine Agency website.
Over 16 years, 272 of 657 oncology ODDs (41%) concerned a malignant condition occurring both in adults and children. The five most common were acute myeloid leukaemia, high-grade glioma, acute lymphoblastic leukaemia, graft-versus-host disease and soft-tissue sarcomas. 74% of 31 marketing authorisations (MAs) for an indication both in adults and children (26 medicines) had no information for paediatric use in their Summary of Product Characteristics (SmPC) at the time of the first MA. Furthermore, 68% still have no paediatric information in their most recently updated SmPC, at a median of 7 years after. Only 15 ODDs (2%) pertained to a malignancy occurring specifically in children and only two drugs received an MA: Unituxin for high-risk neuroblastoma and Votubia for sub-ependymal giant-cell astrocytoma.
The Orphan Drug Regulation failed to promote the development of innovative therapies for malignancies occurring in children. Major delays and waivers occurred through the application of the Paediatric Medicines Regulation. The European regulatory environment needs to be improved to accelerate innovation for children and adolescents dying of cancer.
肿瘤学是欧洲罕见病药物研发领域的一个主要部门。目的是评估癌症儿童和青少年是否从《罕见病药物法规》中受益。
从人用药品共同体注册登记处收集了2000年8月8日至2016年9月10日期间的罕见病药物指定（ODD）和已注册罕见病药物的数据。从欧洲药品管理局网站搜索并检索了评估历史、产品信息和儿科研究计划的存在情况。
在16年期间，657项肿瘤学ODD中有272项（41%）涉及成人和儿童中均出现的恶性疾病。最常见的五种疾病是急性髓系白血病、高级别胶质瘤、急性淋巴细胞白血病、移植物抗宿主病和软组织肉瘤。在31项成人和儿童适应症的上市许可（MA）中（26种药物），74%在首次获得MA时，其产品特性总结（SmPC）中没有儿科用药信息。此外，68%在其最新更新的SmPC中仍然没有儿科信息，中位数时间为7年后。只有15项ODD（2%）涉及专门发生在儿童中的恶性肿瘤，只有两种药物获得了MA：用于高危神经母细胞瘤的Unituxin和用于室管膜下巨细胞星形细胞瘤的Votubia。
《罕见病药物法规》未能促进针对儿童期发生的恶性肿瘤的创新疗法的研发。通过应用《儿科药物法规》出现了重大延误和豁免。欧洲的监管环境需要改善，以加速为死于癌症的儿童和青少年进行创新。
