Cooner Freda, Ye Jingjing, Reaman Gregory
Global Biostatistics, Amgen Inc, Thousand Oaks, CA, USA.
Global Statistics and Data Sciences (GSDS), BeiGene USA, Fulton, MD, USA.
J Biopharm Stat. 2023 Nov 2;33(6):859-874. doi: 10.1080/10543406.2023.2172424. Epub 2023 Feb 7.
Oncology has been one of the most active therapeutic areas in medicinal products development. Despite this fact, few drugs have been approved for use in pediatric cancer patients when compared to the number approved for adults with cancer. This disparity could be attributed to the fact that many oncology drugs have had orphan drug designation and were exempt from Pediatric Research Equity Act (PREA) requirements. On August 18, 2017, the RACE for Children Act, i.e. Research to Accelerate Cures and Equity Act, was signed into law as Title V of the 2017 FDA Reauthorization Act (FDARA) to amend the PREA. Pediatric investigation is now required if the drug or biological product is intended for the treatment of an adult cancer and directed at a molecular target that FDA determines to be "substantially relevant to the growth or progression of a pediatric cancer." This paper discusses the specific considerations in clinical trial designs and statistical methodologies to be implemented in oncology pediatric clinical programs.
肿瘤学一直是药品开发中最活跃的治疗领域之一。尽管如此，与获批用于成年癌症患者的药物数量相比，获批用于儿科癌症患者的药物却很少。这种差异可能归因于许多肿瘤学药物都有孤儿药指定，并且免于《儿科研究公平法案》（PREA）的要求。2017年8月18日，《儿童研究加速治愈与公平法案》（即《加速治愈与公平研究法案》）作为2017年《美国食品药品监督管理局再授权法案》（FDARA）的第五章签署成为法律，以修订PREA。如果药物或生物制品旨在治疗成人癌症且针对美国食品药品监督管理局确定为“与儿科癌症的生长或进展基本相关”的分子靶点，则现在需要进行儿科研究。本文讨论了肿瘤学儿科临床项目中临床试验设计和统计方法的具体考虑因素。
