Peer-reviewed publication of clinical trials completed for pediatric exclusivity.
BACKGROUND
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.


METHODS
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.


METHODS
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.
Benjamin
Daniel K
DK
Office of Pediatric Therapeutics, Office of the Commissioner, US Food and Drug Administration, Rockville, Md, USA. danny.benjamin@duke.edu
Smith
Philip Brian
PB
Murphy
M Dianne
MD
Roberts
Rosemary
R
Mathis
Lisa
L
Avant
Debbie
D
Califf
Robert M
RM
Li
Jennifer S
JS
D002986:Clinical Trials as Topic; D004348:Drug Labeling; D010372:Pediatrics; D017712:Peer Review, Research; D004364:Pharmaceutical Preparations; D014481:United States; D014486:United States Food and Drug Administration
D004364:Pharmaceutical Preparations