Peer-reviewed publication of clinical trials completed for pediatric exclusivity
Peer-reviewed publication of clinical trials completed for pediatric exclusivity
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DOI：
10.1001/jama.296.10.1266
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发表时间：
2006-09-13
影响因子：
120.7
通讯作者：
Li, Jennifer S.
中科院分区：
文献类型：
--
作者：
Benjamin, Daniel K., Jr.;Smith, Philip Brian;Li, Jennifer S.
大部分儿科药物的使用是标签外的，因为没有进行适当的儿科研究，并且药物没有被美国食品和药物管理局（FDA）标记为用于儿童。1997年，国会授权FDA在进行FDA要求的儿科试验时，授予被称为“儿科排他性”的营销权扩展。因此，有100多个产品标签发生了变化。目的通过MEDLINE和EMBASE检索确定1998年至2004年期间所有儿科专用性试验的队列研究，并对随后的标签变更、并在同行评审的期刊上发表这些研究。我们将研究导致的任何标签变更分类为对研究药物的阳性或阴性。然后，我们评估了与随后在同行评审的医学期刊上发表相关的研究和产品标签变更的方面。主要结果测量在同行评审的期刊上发表的试验数据。结果在1998年至2004年期间，有253项研究提交给FDA进行儿科独占：125项（50%）评价疗效，51项（20%）为多次给药药代动力学，34项（13%）为单次给药药代动力学，43项（17%）为安全性研究。127/253（50%）项研究的标签变更为阳性;仅113/253（45%）项研究发表。疗效研究和那些具有积极的标签变化更有可能被published.Conclusions儿科排他性计划已成功地鼓励儿童药物研究。然而，这些结果在同行评审文献中的传播是有限的。通过出版物更广泛地传播这一信息的机制值得进一步评价。
Context Much of pediatric drug use is off-label because appropriate pediatric studies have not been conducted and the drugs have not been labeled by the US Food and Drug Administration (FDA) for use in children. In 1997, Congress authorized the FDA to grant extensions of marketing rights known as "pediatric exclusivity" if FDA-requested pediatric trials were conducted. As a result, there have been over 100 product labeling changes. The publication status of studies completed for pediatric exclusivity has not been evaluated.Objective To quantify the dissemination of results of studies conducted for pediatric exclusivity into the peer-review literature.Design Cohort study of all trials conducted for pediatric exclusivity between 1998 and 2004 as determined by MEDLINE and EMBASE searches through 2005, the subsequent labeling changes, and the publication of those studies in peer-reviewed journals. We categorized any labeling changes resulting from the studies as positive or negative for the drug under study. We then evaluated aspects of the studies and product label changes that were associated with subsequent publication in peer-reviewed medical journals.Main Outcome Measures Publication of the trial data in peer-reviewed journals.Results Between 1998 and 2004, 253 studies were submitted to the FDA for pediatric exclusivity: 125 (50%) evaluated efficacy, 51 (20%) were multi-dose pharmacokinetic, 34 (13%) were single-dose pharmacokinetic, and 43 (17%) were safety studies. Labeling changes were positive for 127/253 (50%) of studies; only 113/253 (45%) were published. Efficacy studies and those with a positive labeling change were more likely to be published.Conclusions The pediatric exclusivity program has been successful in encouraging drug studies in children. However, the dissemination of these results in the peer-reviewed literature is limited. Mechanisms to more widely disperse this information through publication warrant further evaluation.