Background/Aims:

The Pediatric Research Equity Act and Best Pharmaceuticals for Children Act are intended to promote the conduct of clinical trials that generate pediatric-specific evidence about drug safety and efficacy. This study assesses the quality of evidence generated through Pediatric Research Equity Act–mandated and Best Pharmaceuticals for Children Act–incentivized clinical trials of hematology/oncology drugs and characterizes subsequent changes in pediatric drug utilization rates.

Methods:

Trial characteristics (blinding, randomization, and comparator group) were determined for clinical trials that supported pediatric label changes. Using data from OptumLabs^® Data Warehouse, a de-identified administrative claims database, we calculated pediatric utilization rates for each drug. We calculated monthly utilization rates from January 2003 (or from the first month in which data were available) to December 2018.

Results:

We identified 11 hematology/oncology drugs that underwent pediatric label changes under the Pediatric Research Equity Act Pediatric Research Equity Act and/or Best Pharmaceuticals for Children Act, and we identified 15 trials supporting these changes. Of these trials, 36% (5/14) were randomized, 31% (4/13) were blinded, and 36% (5/14) used a comparator group. A median of 49 children (interquartile range 29.5) received the drug under investigation across these trials. Pediatric label changes were not associated with subsequent changes in pediatric drug utilization. Although some drugs saw increased pediatric use after gaining new pediatric indications, this pattern was not consistently observed. In addition, there was no evidence to suggest that drugs were utilized less frequently after they failed to receive pediatric indications.

Conclusions:

Clinical trials of hematology/oncology drugs conducted under the Pediatric Research Equity Act Pediatric Research Equity Act and Best Pharmaceuticals for Children Act generally have low methodological rigor, and the resulting label changes are not consistently associated with changes in pediatric utilization. Alternative regulatory strategies and study designs may be necessary to maximize the impact of newly generated knowledge on drug utilization.

中文翻译：

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药品标签变化和儿科血液学/肿瘤学处方：衡量美国立法的影响

背景/目标：

《儿科研究公平法》和《儿童最佳药品法》旨在促进临床试验的进行，生成有关药物安全性和有效性的儿科特定证据。本研究评估了通过《儿科研究公平法》规定和《儿童最佳药品法》激励的血液学/肿瘤学药物临床试验产生的证据质量，并描述了儿科药物利用率的后续变化。

方法：

确定了支持儿科标签变更的临床试验的试验特征（盲法、随机化和比较组）。使用来自 OptumLabs ^®数据仓库（一个去识别化的行政索赔数据库）的数据，我们计算了每种药物的儿科利用率。我们计算了从 2003 年 1 月（或从有数据可用的第一个月）到 2018 年 12 月的月度利用率。

结果：

我们确定了 11 种血液学/肿瘤学药物根据《儿科研究公平法》和/或《最佳儿童药物法》进行了儿科标签变更，并且我们确定了 15 项支持这些变化的试验。在这些试验中，36% (5/14) 是随机试验，31% (4/13) 是盲法，36% (5/14) 使用比较组。在这些试验中，中位数为 49 名儿童（四分位数间距 29.5）接受了所研究的药物。儿科标签的变化与儿科药物使用的后续变化无关。尽管一些药物在获得新的儿科适应症后儿童使用量有所增加，但这种模式并未得到一致观察。此外，没有证据表明药物在未获得儿科适应症后会减少使用频率。

结论：

根据《儿科研究公平法》和《儿童最佳药品法》进行的血液学/肿瘤学药物临床试验通常方法严谨性较低，并且由此产生的标签变化与儿科利用的变化并不总是相关。为了最大限度地发挥新产生的知识对药物利用的影响，可能需要替代的监管策略和研究设计。