为了描述儿童癌症临床试验起始的延迟及其与FDA批准的关系，我们使用了ClinicalTrials.gov创建了一个样本，该样本包括了从2006年到2011年开始在成年人中进行有效性测试的儿童相关癌症药物。我们描述了成年人有效性试验与儿童试验之间的延迟。我们还评估了在儿童中评估未获得批准的药物的比例，未获得批准的药物中在获得食品和药物管理局（FDA）批准之前未在儿童中进行评估的比例，以及较短的延迟是否与更大的效果大小或更高的批准概率相关联。在我们的样本中，185种药物中有34%在儿童中进行了评估；到达临床试验的中位延迟为4.16年。在所有药物中，有17%获得了FDA批准，其中41%在获得许可之前从未在儿童中进行过测试。在未获得批准的153种药物中，78%在儿童中未进行过评估。较早的测试并没有显著预测更高的反应率（p = 0.13）。未获得监管批准的药物较早进行了评估（未获得批准药物的延迟测试为3.0年，而获得批准药物的延迟测试为5.4年，p = 0.019）。癌症新药通常在经过成年人有效性评估后数年才在儿童中进行评估。这种延迟可能在儿童测试之前淘汰了一些缺乏良好药理学特征的药物。但是，一些被淘汰的药物可能在儿童适应症中有活性。需要进一步考虑用于优先考虑儿童测试的方法。Copyright © 2023 Elsevier Inc. All rights reserved.

To describe the delay for first-in-minor cancer clinical trials and its relationship with FDA approval.We used ClinicalTrials.gov to create a sample of pediatric-relevant cancer drugs starting efficacy testing in adults from 2006 through 2011. We characterized the delay between first-in-adult efficacy trials and first-in-minor trials. We also assessed the proportion of drugs evaluated in minors that failed to gain approval, the proportion that were not evaluated in minors before receiving Food and Drug Administration (FDA) approval, and whether shorter delay was associated with larger effect sizes or greater probability of regulatory approval.34% of the 185 drugs in our cohort were evaluated in minors; the median delay to clinical trials was 4.16 years. Of all drugs, 17% received FDA approval, 41% of which were never tested in minors before licensing. Of the 153 drugs not attaining approval, 78% were not evaluated in minors. Earlier testing did not significantly predict greater response rates (p = 0.13). Drugs not attaining regulatory approval were evaluated significantly earlier (3.0 for drugs not approved vs. 5.4 years delayed testing for approved drugs, p = 0.019).New cancer drugs were typically evaluated in minors years after adult efficacy evaluation. This delay likely eliminated some drugs lacking desirable pharmacology before pediatric testing. However, some drugs that were eliminated may have had activity in pediatric indications. Approaches for prioritizing drugs for pediatric testing warrants further consideration.Copyright © 2023 Elsevier Inc. All rights reserved.