Carmack Mary, Hwang Thomas, Bourgeois Florence T
Mary Carmack is a researcher in the Pediatric Therapeutics and Regulatory Science Initiative in the Computational Health Informatics Program at Boston Children's Hospital, in Boston, Massachusetts.
Thomas Hwang is a researcher in the Pediatric Therapeutics and Regulatory Science Initiative in the Computational Health Informatics Program at Boston Children's Hospital.
Health Aff (Millwood). 2020 Oct;39(10):1799-1805. doi: 10.1377/hlthaff.2020.00198.
Several policies have been implemented in the US to promote the evidence-based use of therapeutics in pediatric populations. Under the Best Pharmaceuticals for Children Act of 2002 and the Pediatric Research Equity Act of 2003, the Food and Drug Administration (FDA) can request pediatric studies for new drug and biologic indications. The acts have been credited with generating pediatric information for hundreds of drugs. However, concerns have been raised that delays and noncompliance with study requests contribute to high rates of off-label and potentially unsafe or ineffective medication use in children. We used publicly available FDA documents to analyze all indications for new drugs and biologics approved by the FDA from 2002 through 2018. During this time the FDA issued 389 pediatric study requests (141 under the Best Pharmaceuticals for Children Act and 248 under the Pediatric Research Equity Act) for 274 new drugs and biologics, representing 320 indications. We found that as of December 31, 2018, fewer than a third of these study requests had been completed. Overall, 64 percent of new drug and biologic indications deemed relevant to pediatric patients lacked pediatric prescribing information at five years after FDA approval. Enforcement of pediatric drug study policies should be strengthened to reduce non-evidence-based medication use in pediatric patients.
美国已实施多项政策，以促进在儿科人群中循证使用治疗药物。根据2002年的《儿童最佳药物法案》和2003年的《儿科研究公平法案》，美国食品药品监督管理局（FDA）可要求针对新药和生物制品的适应症开展儿科研究。这些法案为数百种药物提供了儿科用药信息。然而，有人担心研究请求的延迟和不遵守导致儿童中标签外用药以及潜在不安全或无效用药的比例居高不下。我们利用FDA公开的文件，分析了2002年至2018年期间FDA批准的新药和生物制品的所有适应症。在此期间，FDA针对274种新药和生物制品发布了389项儿科研究请求（141项根据《儿童最佳药物法案》，248项根据《儿科研究公平法案》），涉及320个适应症。我们发现，截至2018年12月31日，这些研究请求中完成的不到三分之一。总体而言，在FDA批准五年后，64%被认为与儿科患者相关的新药和生物制品适应症缺乏儿科处方信息。应加强儿科药物研究政策的执行，以减少儿科患者中不基于证据的用药情况。
