Program on Regulation, Therapeutics, and Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, Massachusetts.
Pediatrics. 2024 Oct 1;154(4). doi: 10.1542/peds.2024-066920.
Adult cancer drugs have historically been exempted from pediatric testing requirements. In 2017, Congress passed the Research to Accelerate Cures and Equity (RACE) for Children Act to expand mandatory pediatric testing to cancer drugs; the law took effect in 2020. With this study, we sought to evaluate how the pediatric testing of molecularly targeted adult cancer drugs changed after the RACE Act.
In this retrospective cohort study, we used publicly available Food and Drug Administration data to compare pediatric post-approval requirements, trials, and trial characteristics, including timing, in adult cancer drugs before and after the RACE Act.
Between 2017 and 2024, the Food and Drug Administration approved 61 adult cancer drugs with molecular targets relevant to pediatric cancer; 40 were submitted before 2020, and 21 were submitted after 2020. The 40 pre-RACE Act drugs were associated with no pediatric post-approval requirements, whereas the 21 post-RACE Act drugs were associated with 15 pediatric post-approval testing requirements. Approximately two-thirds (26/40, 65%) of pre-RACE Act drugs and 57% (12/21) of post-RACE Act drugs were evaluated in pediatric trials. Among pre-RACE Act cancer drugs, pediatric trials were initiated a median of 0.04 years after approval (interquartile range: -3.3 to 1.9 years), whereas post-RACE Act trials were initiated a median of 2.8 years before approval (interquartile range: -4.3 to 0.3 years).
The RACE Act has been associated with greater numbers of pediatric post- approval testing requirements and the earlier initiation of pediatric trials, although early pediatric trial rates appear unchanged. Formalizing pediatric testing requirements may lead to the timely completion of pediatric studies to the benefit of pediatric patients with cancer.
成人癌症药物历来免除儿科测试要求。2017 年，国会通过了《为儿童加速治愈和公平（RACE）法案》，将强制性儿科测试扩大到癌症药物；该法律于 2020 年生效。本研究旨在评估 RACE 法案通过后，分子靶向成人癌症药物的儿科测试情况发生了怎样的变化。
在这项回顾性队列研究中，我们使用公开的美国食品和药物管理局（FDA）数据，比较了 RACE 法案前后分子靶向成人癌症药物的儿科批准后要求、试验和试验特征，包括时间。
在 2017 年至 2024 年期间，FDA 批准了 61 种具有儿科癌症相关分子靶点的成人癌症药物；其中 40 种在 2020 年前提交，21 种在 2020 年后提交。40 种 RACE 法案前药物与无儿科批准后要求相关，而 21 种 RACE 法案后药物与 15 种儿科批准后测试要求相关。大约三分之二（26/40，65%）的 RACE 法案前药物和 57%（12/21）的 RACE 法案后药物在儿科试验中进行了评估。在 RACE 法案前的癌症药物中，儿科试验在批准后中位数 0.04 年启动（四分位距：-3.3 至 1.9 年），而 RACE 法案后的试验在批准前中位数 2.8 年启动（四分位距：-4.3 至 0.3 年）。
RACE 法案与更多儿科批准后测试要求和更早启动儿科试验相关，尽管儿科早期试验率似乎保持不变。将儿科测试要求正式化可能会及时完成儿科研究，使癌症患儿受益。
