Office of Clinical Pharmacology, Office of Translational Sciences, U.S. Food and Drug Administration, Silver Spring, Maryland 20993, USA
Office of Clinical Pharmacology, Office of Translational Sciences, U.S. Food and Drug Administration, Silver Spring, Maryland 20993, USA.
Cold Spring Harb Perspect Med. 2024 Apr 1;14(4):a041656. doi: 10.1101/cshperspect.a041656.
The discovery and development of anticancer drugs for pediatric patients have historically languished when compared to both past and recent activity in drug development for adult patients, notably the dramatic spike of targeted and immune-oncology therapies. The reasons for this difference are multifactorial. Recent changes in the regulatory landscape surrounding pediatric cancer drug development and the understanding that some pediatric cancers are driven by genetic perturbations that also drive disparate adult cancers afford new opportunities. The unique cancer-initiating events and dependencies of many pediatric cancers, however, require additional pediatric-specific strategies. Research efforts to unravel the underlying biology of pediatric cancers, innovative clinical trial designs, model-informed drug development, extrapolation from adult data, addressing the unique considerations in pediatric patients, and use of pediatric appropriate formulations, should all be considered for efficient development and dosage optimization of anticancer drugs for pediatric patients.
与过去和最近成人患者药物开发的活动相比，儿童患者抗癌药物的发现和开发一直滞后，特别是靶向治疗和肿瘤免疫治疗的显著增加。造成这种差异的原因是多方面的。围绕儿科癌症药物开发的监管环境的最新变化，以及认识到一些儿科癌症是由驱动不同成人癌症的遗传扰动驱动的，这为新的机会提供了条件。然而，许多儿科癌症的独特致癌事件和依赖性需要额外的儿科特异性策略。为了解儿科癌症的基础生物学、创新临床试验设计、模型指导药物开发、从成人数据推断、解决儿科患者的独特考虑因素以及使用儿科适当制剂而进行的研究工作，都应该被考虑用于提高儿童患者抗癌药物的开发效率和剂量优化。
